tag:blogger.com,1999:blog-22760806945922777072024-03-12T19:48:42.907-07:00msemporda@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comBlogger27125tag:blogger.com,1999:blog-2276080694592277707.post-70722214690944627092022-11-12T11:03:00.001-08:002022-11-12T11:03:43.265-08:00<div style="text-align: center;"><b><u><span style="font-family: inherit; font-size: large;">Groundbreaking Organoid Vision Clinical Study: Takahashi Q&A by Paul Knoepfler</span></u></b></div><h1 style="background: rgb; clear: left; color: #0a0a03; margin: 0px; padding: 1px 0px; text-align: center;"><b><span><div style="color: black; font-weight: 400; text-align: left;"><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;"><a href="https://ipscell.com/?s=masayo+takahashi" target="_blank"><b><span style="color: #3a3a3a; text-decoration-line: none;">Masayo Takahashi (</span></b><b><span style="color: #3a3a3a; text-decoration-line: none;">高橋</span></b><b><span style="color: #3a3a3a; text-decoration-line: none;"> </span></b><b><span style="color: #3a3a3a; text-decoration-line: none;">政代</span></b><b><span style="color: #3a3a3a; text-decoration-line: none;">)</span></b><span style="color: #3a3a3a;"> </span></a>is a pioneer in stem cell-based clinical trial work and more specifically induced pluripotent stem cell (IPSC)-based trials. Her new clinical study is another groundbreaking step in that it is organoid based, in this case for <a href="https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/retinitis-pigmentosa" target="_blank"><b><span style="color: #0274be; text-decoration-line: none;">Retinitis pigmentosa</span></b></a>.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">This post is an informative Q&A interview with Dr. Takahashi on her new organoid clinical study. You can see an image below from their foundational mouse transplant work. Ref provided for researchers to <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5918611/" target="_blank"><b><span style="color: #0274be; text-decoration-line: none;">this pub</span></b></a> as well.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">Then later in the post Paul has included a timeline that Dr. Takahashi sent of the efforts and she noted that sub-team leader Dr. Michiko Mandai MD, PhD is doing a great deal of this work.</span></span></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgOlF7InL4uBFyR0gB2yOgYrhvvQTnYPAgeGNZMFP8FBirE_5H3Ki4ATDNG0nPgbS89IJa02601JA9QI9lqKF8SAY4e8vBnSN1rqb3ifc_o6gmFgjvB2Ah0bsMjFh-57NJIA6-nBpnyrDUFmno5cuciTc_gbZkFLm_HkQQBgS2SoOSLY2kxnRAHotPudA/s640/Takahashi1.JPG" style="margin-left: 1em; margin-right: 1em;"><span style="font-family: inherit; font-size: medium;"><img border="0" data-original-height="468" data-original-width="640" height="293" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgOlF7InL4uBFyR0gB2yOgYrhvvQTnYPAgeGNZMFP8FBirE_5H3Ki4ATDNG0nPgbS89IJa02601JA9QI9lqKF8SAY4e8vBnSN1rqb3ifc_o6gmFgjvB2Ah0bsMjFh-57NJIA6-nBpnyrDUFmno5cuciTc_gbZkFLm_HkQQBgS2SoOSLY2kxnRAHotPudA/w400-h293/Takahashi1.JPG" width="400" /></span></a></div><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><span style="color: green;">PK: This is the trial in the world to use a IPS cell derivative for Retinitis pigmentosa, correct? What preclinical work went into the foundation for this?</span></b><span style="color: #3a3a3a;"><o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">MT: Yes. First transplantation utilize organoids and first obvious reconstruction of CNS.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">Some past related work on retinal differentiation and organoids from pluripotent cells are below:<o:p></o:p></span></span></p><ul style="margin-top: 0in;" type="disc"><li class="MsoNormal" style="color: #3a3a3a; line-height: normal; margin-bottom: 0in; mso-list: l0 level1 lfo1; tab-stops: list .5in; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><i>Assawachananont et al Stem Cell Reports 2014 </i></b><o:p></o:p></span></li><li class="MsoNormal" style="color: #3a3a3a; line-height: normal; margin-bottom: 0in; mso-list: l0 level1 lfo1; tab-stops: list .5in; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><i>Fujii et al Sci Rep., 2016 </i></b><o:p></o:p></span></li><li class="MsoNormal" style="color: #3a3a3a; line-height: normal; margin-bottom: 0in; mso-list: l0 level1 lfo1; tab-stops: list .5in; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><i>Mandai et al Stem Cell Reports, 2017</i></b><o:p></o:p></span></li><li class="MsoNormal" style="color: #3a3a3a; line-height: normal; margin-bottom: 0in; mso-list: l0 level1 lfo1; tab-stops: list .5in; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><i>Iraha and Tu et al Stem Cell reports, 2018</i></b><o:p></o:p></span></li><li class="MsoNormal" style="color: #3a3a3a; line-height: normal; margin-bottom: 0in; mso-list: l0 level1 lfo1; tab-stops: list .5in; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><i>Tu and Watanabe et al EbioMedicine, 2019</i></b><o:p></o:p></span></li><li class="MsoNormal" style="color: #3a3a3a; line-height: normal; margin-bottom: 0in; mso-list: l0 level1 lfo1; tab-stops: list .5in; vertical-align: baseline;"><b><i><span style="font-family: inherit; font-size: medium;">Akiba et al. eCollection 2019 </span></i></b></li></ul><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><span style="color: green;">PK: What is the rationale for using a retinal organoid as opposed to a sheet or suspension of retinal cells?</span></b><span style="color: #3a3a3a;"><o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">MT: We will use tiny retinal progenitor cell sheets cut from retinal organids.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">For more than 10 years from the 2006 <i>Nature</i> paper, it had been believed that cell suspension transplant works well in the retina. But it was proved to be cytoplasmic transfer in 2016 or so.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">We believed from the beginning that tissue transplantation is necessary to reconstruct the photoreceptor layer. Our papers were right.<o:p></o:p></span></span></p><div class="separator" style="clear: both; text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhKGfmTU1-1_ZDRG7HWiRI5C0ZYPrIVmHNOjAWbJ6WInyBkic51_S-v9_ukLCM1Iusn1wLVEGcbDr675wdunRuIUeyTe69IqxGHJ3rN-UWvBbqEtdQuHls2e_K41T9HGPZBtvbuCo7fLucam_OEb8W4DH_gRc-VF4GF6o5izaeQE--pS1dOBqYWi30UzA/s633/Takahashi2.JPG" style="margin-left: 1em; margin-right: 1em;"><span style="font-family: inherit; font-size: medium;"><img border="0" data-original-height="322" data-original-width="633" height="204" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhKGfmTU1-1_ZDRG7HWiRI5C0ZYPrIVmHNOjAWbJ6WInyBkic51_S-v9_ukLCM1Iusn1wLVEGcbDr675wdunRuIUeyTe69IqxGHJ3rN-UWvBbqEtdQuHls2e_K41T9HGPZBtvbuCo7fLucam_OEb8W4DH_gRc-VF4GF6o5izaeQE--pS1dOBqYWi30UzA/w400-h204/Takahashi2.JPG" width="400" /></span></a></div><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><span style="color: green;">PK: Are there other cells present in the organoids besides photoreceptors? Does the purity matter? What organoid protocol is being used and how long are the organoids allowed to develop?</span></b><span style="color: #3a3a3a;"><o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">MT: We will transplant organoids that consists of only retinal progenitors. It will maturate in the patients eye for several months to generate mature photoreceptors. There are other types of retinal cells, but still it is OK to recover the visual function in the completely photoreceptor degenerated retinal<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><span style="color: green;">PK: How will the organoids be deployed? Are they surgical implanted in the retina? How organoids used per eye?</span></b><span style="color: #3a3a3a;"><o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">MT: Cut tiny sheets from the organdies and transplant into the subretinal space.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><span style="color: green;">PK: Do you predict the organoids will integrate into the eye and the cellular and neural network?</span></b><span style="color: #3a3a3a;"><o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">MT: We vigorously confirmed this for nearly 10 years using electrophysiology and behavior tests, which you can see in our papers.<o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="font-family: inherit; font-size: medium;"><b><span style="color: green;">PK: How many patients will be transplanted and how long will the follow-up be?</span></b><span style="color: #3a3a3a;"><o:p></o:p></span></span></p><p class="MsoNormal" style="line-height: normal; margin-bottom: 19.2pt; vertical-align: baseline;"><span style="color: #3a3a3a;"><span style="font-family: inherit; font-size: medium;">MT: The plan is 2 patients to start and 1 year of observation. A clinical trial is planned by a company after this study.<o:p></o:p></span></span></p><p class="MsoNormal"><span style="font-family: inherit; font-size: medium;">Source: <a href="https://ipscell.com/2020/07/groundbreaking-organoid-vision-trial-takahashi-qa/">The Niche</a></span></p></div></span></b></h1>@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-33566162373977933352019-08-30T19:57:00.002-07:002019-08-30T20:15:06.193-07:00<div style="text-align: center;">
<u><b><span style="font-size: large;">Osaka team pulls off cornea cell transplant using human iPS cells</span></b></u></div>
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In a world's first, a team of researchers here transplanted cornea cells created from human iPS cells into a patient whose visual acuity has been improved.</div>
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The transplant was conducted in July by a team led by Kohji Nishida, a professor of ophthalmology at Osaka University.</div>
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The team announced the achievement on Aug. 29 at a news conference at the university's campus in Suita, Osaka Prefecture.</div>
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The patient, a woman in her 40s, has made good progress since the operation and was released from the hospital on Aug. 23.</div>
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“It’s been just a month, but right now we see the operation as a success,” Nishida said.</div>
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The transplant is intended for patients with corneal epithelial stem cell impoverishment syndrome.<br />
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The cause of the syndrome is a loss of stem cells that produce a new cornea that can cover the surface of a pupil caused by injury or other reasons. The syndrome leads to poor eyesight and sometimes blindness.<br /><br />The team transformed iPS cells from a third party into cornea cells. They then turned them into a sheet 0.03 to 0.05 millimeters thick and transplanted them onto the patient’s left eye.<br /><br />If all goes well, the transplanted cells will enable a lasting production of cornea cells, maintain corneal transparency and help the patient regain lost vision.<br /><br />“After the operation, her clouded cornea became transparent and her vision has improved considerably. We'll continue to monitor her condition to see if it stays that way," Nishida said.<br /><br />The team said it has not observed an abnormal increase in the transplanted cornea cells, and the patient has regained vision to the extent that she can go about her daily life.<br /><br />Following the success, the team expects to perform a second transplant by the end of the year.<br /><br />Corneas of dead people are currently transplanted into patients with the particular syndrome, but the organs are chronically in short supply.<br /><br />According to the health ministry, organs from 720 people who were certified brain dead or in cardiac arrest were provided to patients with corneal diseases and 1,155 transplantation surgeries were performed in fiscal 2018.<br /><br />As of the end of March this year, 1,613 people were on a waiting list for a cornea transplant.<br /><br />In the team’s estimate, several hundred people in the country each year will become subjects for the cornea transplants using iPS cells.<br /><br />The operation was the third successful transplant using iPS cells, following the 2014 transplantation of retina cells by the government-affiliated Riken institute and Kyoto University researchers transplanting nerve cells into the brain of a Parkinson’s disease patient in 2018.<br /><br />Ref: The Asahi <a href="http://www.asahi.com/ajw/articles/AJ201908300056.html?utm_source=dlvr.it&utm_medium=twitter">PR</a></div>
@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-30321409085660587032019-04-02T11:07:00.001-07:002019-04-02T11:08:44.149-07:00Fate Therapeutics doses initial patient in 1st US trial of a iPSC-derived therapeutic<div style="text-align: center;">
<b><u>Fate Therapeutics Announces First Patient Treated with iPSC-derived NK Cell Cancer Immunotherapy FT500 Successfully Completes Initial Safety Assessment</u></b></div>
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<b>No Dose-Limiting Toxicities or Serious Adverse Events Reported following Three Once Weekly Doses of Universal, Off-the-Shelf NK Cell Product Candidate</b></div>
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<b>New Preclinical Data of Universal, Off-the-shelf, iPSC-derived NK Cell Product Candidates FT516 and FT596 Highlighted at 2019 AACR Annual Meeting</b></div>
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Fate Therapeutics, Inc., a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, today announced that the first patient treated with FT500 successfully completed an initial safety assessment. The patient received three once weekly doses of FT500, and the treatment cycle was well-tolerated with no dose-limiting toxicities or serious adverse events reported during the initial 28-day observation period. The universal, off-the-shelf natural killer (NK) cell product candidate is the first-ever cell therapy derived from an induced pluripotent stem cell (iPSC) administered to a patient in the U.S.</div>
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“The ability to effectively and efficiently deliver multiple doses of a cellular immunotherapy ‘on demand’ brings us closer to our goal of transforming the treatment of cancer for more patients. This initial observation of tolerability from the first-ever cancer patient to receive multiple doses of a universal, off-the-shelf cell product derived from a clonal master iPSC line provides early clinical validation of our proprietary iPSC product platform for off-the-shelf cancer immunotherapy,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “In addition to our clinical progress with FT500, our engineered iPSC-derived NK cell product candidates continue to exhibit a highly-differentiated therapeutic profile in preclinical models and we look forward to generating initial clinical data with FT516 and FT596 in 2019.”</div>
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Two additional patients have also been treated with FT500 as a monotherapy in the first dose cohort of 1x108 cells per dose and are currently within the initial 28-day observation period. The FT500 clinical trial is a two-arm study in up to 64 patients for the treatment of advanced solid tumors. The study is designed to assess the safety and activity of three once weekly doses of FT500 as a monotherapy and in combination with one of three FDA-approved checkpoint inhibitor therapies – nivolumab, pembrolizumab or atezolizumab – in patients that have failed or have confirmed disease progression on checkpoint inhibitor therapy. Patients that are clinically stable following the initial 28-day observation period are eligible to receive a second treatment cycle.</div>
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FT516 Novel CD16 Receptor Promotes High-Affinity Engagement with Monoclonal Antibody Therapy</div>
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Today the Company presented preclinical data for FT516, its universal, off-the-shelf NK cell product candidate derived from a clonal master iPSC line engineered to express a novel CD16 Fc (hnCD16) receptor, at the 2019 American Association for Cancer Research (AACR) in Atlanta, Georgia. FT516 is the first-ever cell therapy derived from a genetically engineered pluripotent stem cell cleared for clinical testing in the world, and the Company is preparing to initiate clinical investigation of FT516 in the U.S. in patients with certain relapsed/refractory hematologic malignancies, including acute myelogenous leukemia as a monotherapy, non-Hodgkin's lymphoma in combination with rituximab, and multiple myeloma in combination with elotuzumab.</div>
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While CD16 is naturally expressed on NK cells and mediates antibody-dependent cellular cytotoxicity, numerous clinical studies with FDA-approved tumor-targeting antibodies have demonstrated that patients with CD16 high-affinity variant 158V have improved clinical outcomes. However, only about 15% of humans are homozygous for 158V. Additionally, the expression of CD16 on NK cells in cancer patients can undergo considerable down-regulation, which significantly inhibits the cell’s anti-tumor activity. The novel CD16 Fc receptor expressed by FT516 has been designed to overcome these inherent deficiencies: it is comprised of the high-affinity 158V variant and is resistant to down-regulation.</div>
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In preclinical studies using a B-cell lymphoma line, the Company showed that approximately 70% of peripheral blood NK cells down-regulated CD16 expression upon co-culture with rituximab, while CD16 expression on FT516 remained resistant to down-regulation. These differences resulted in a significant anti-tumor benefit in vivo where, in a human lymphoma cancer model, mice treated with peripheral blood NK cells and rituximab had a median survival time of 39 days as compared to mice treated with FT516 and rituximab, where the median survival time was not yet reached at 100 days.</div>
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FT596 CAR and CD16 Modalities Exert Synergistic Anti-Tumor Activity</div>
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The Company also presented today at AACR new preclinical data for FT596, the Company’s first iPSC-derived chimeric antigen receptor (CAR) NK cell product candidate that is designed to concurrently target multiple tumor-associated antigens. FT596 is derived from a clonal master iPSC line engineered to express a proprietary CAR targeting CD19, a hnCD16 Fc receptor, and a novel IL-15 receptor fusion.</div>
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In a mixed co-culture assay, the Company showed that the concurrent activation of the CAR and hnCD16 targeting modalities of FT596 exert synergistic anti-tumor activity. Increased degranulation (CD107a) and cytokine release (interferon-gamma and TNF-alfa) were observed upon concurrent activation of both the CAR and CD16 receptors in CD19+CD20+ Raji cancer cells with rituximab as compared to activation of each receptor alone, suggestive that dual antigen engagement may elicit a deeper and more durable response. Additionally, in a cellular cytotoxicity assay designed to model CD19 antigen escape, FT596 combined with rituximab was able to effectively eliminate leukemia and lymphoma cancer cells that were positive for CD19 antigen expression as well as those that were null for CD19 antigen expression.</div>
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About FT500</div>
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FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. FT500 is being investigated in an open-label, repeat-dose Phase 1 clinical trial for the treatment of advanced solid tumors in up to 64 patients, both as a monotherapy and in combination with FDA-approved checkpoint inhibitor therapy. Despite the favorable response rates observed with checkpoint inhibitor therapy, the majority of patients do not respond and many responders relapse. One common mechanism of resistance to checkpoint inhibitor therapy is associated with loss-of-function mutations in genes critical for antigen presentation. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill tumor cells with these mutations.</div>
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About Fate Therapeutics’ iPSC Product Platform</div>
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The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. As a result, the Company’s platform is uniquely capable of addressing the limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is fraught with batch-to-batch and cell-to-cell variability that can affect safety and efficacy. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.</div>
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Source: Fate <a href="https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-announces-first-patient-treated-ipsc-derived" target="_blank">PR</a></div>
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Refs: <a href="https://clinicaltrials.gov/ct2/show/NCT03841110" target="_blank">Clinical Trial</a> </div>
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UC San Diego trial site <a href="https://health.ucsd.edu/news/releases/Pages/2019-04-01-UC-San-Diego-Health-Treats-Cancer-Patient-with-Stem-Cell-Derived-Natural-Killer-Cells.aspx" target="_blank">news</a></div>
@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-32989467996023923082018-11-30T12:54:00.000-08:002018-12-01T09:47:23.225-08:00<div style="text-align: center;">
<b><u>Fate Therapeutics Announces FDA Clearance of Landmark IND for FT500 iPSC-derived, Off-the-Shelf NK Cell Cancer Immunotherapy</u></b></div>
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<b>Company to Initiate First-ever U.S. Clinical Investigation of iPSC-derived Cell Product</b><br />
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Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, announced today that the U.S. Food and Drug Administration (FDA) has allowed its Investigational New Drug (IND) Application for FT500, the Company’s universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line. The clinical trial of FT500 is expected to be the first-ever clinical investigation in the U.S. of an iPSC-derived cell product.<br />
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“The clearance by the FDA of our FT500 IND is a significant milestone and marks the beginning of an exciting new era for the clinical development of cell products,” said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. “Clonal master iPSC lines are a renewable cell source that can uniquely produce cell products which are uniformly engineered and well characterized, can be mass produced in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. This revolutionary paradigm overcomes significant challenges that limit both patient- and donor-derived cell therapy, where heterogeneous populations of primary cells are repeatedly sourced, engineered, expanded and characterized on a batch-by-batch basis resulting in cell therapies with substantial variability in quality, consistency and potency.”<br />
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The Company plans to initiate first-in-human clinical testing of FT500 in combination with checkpoint inhibitor therapy for the treatment of advanced solid tumors. This study is expected to evaluate the safety and tolerability of multiple doses of FT500, in multiple dosing cycles with nivolumab, pembrolizumab or atezolizumab, in subjects that have progressed or failed on checkpoint inhibitor therapy.<br />
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Cheers!<br />
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IND Press Release <a href="https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-announces-fda-clearance-landmark-ind-ft500" target="_blank">here</a><br />
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See <b>Fate</b>'s dedicated Blog page <a href="https://msemporda.blogspot.com/p/usafate.html" target="_blank">here</a><br />
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Also recent news from <b>Fate</b> on their partnership with Japan for CAR-T Cell Cancer Immunotherapies based on <b>Fate</b>'s iPSC technology <a href="https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-announces-strategic-collaboration-ono" target="_blank">here</a>.<br />
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Further, <b>Fate</b> licenses <u>new</u> route to master iPS cell lines:<br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjen3rAPCprq4hOJUw6VJdh6Nz1Fskvet29GcZT_BHqtvQJA0a0Yiqzt5-4Qzik9yjwTyB3nS6L2720saXwcVfv65KQc3p05-ME-i6Lu3AChLS_DJ-lR2Bgn0T4WZvBv_rKEMrqTJFPEKc0/s1600/FATE+iPSC.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" data-original-height="724" data-original-width="904" height="256" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjen3rAPCprq4hOJUw6VJdh6Nz1Fskvet29GcZT_BHqtvQJA0a0Yiqzt5-4Qzik9yjwTyB3nS6L2720saXwcVfv65KQc3p05-ME-i6Lu3AChLS_DJ-lR2Bgn0T4WZvBv_rKEMrqTJFPEKc0/s320/FATE+iPSC.jpg" width="320" /></a></div>
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<b>Fate</b> PR announcement <a href="http://fatetherapeutics.com/pipeline/immuno-oncology-candidates/engineered-car-it-cell/" target="_blank">here</a>@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-36840821346892370632018-11-28T12:04:00.000-08:002018-12-16T20:47:54.327-08:00<h4 style="text-align: center;">
<u><b>First Babies Born Using Genome Editing Technology During IVF</b></u></h4>
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Further to the National Academies <a href="https://www.nap.edu/catalog/24623/human-genome-editing-science-ethics-and-governance" target="_blank">report</a> on human genome editing, published in 2017, the self-monitoring governance directive on the sicence to resist human clinical trials for the moment has been challenged by a Chinese scientist who has announced the birth of twins genetically modified to resist HIV infection.</div>
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See the following announcements and media coverage on the development:</div>
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<b><br /></b><a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=11262018" target="_blank">The National Academies</a> - organizing committee statement<br />
<a href="http://science.sciencemag.org/content/362/6420/1215.full" target="_blank">The National Academies</a> - President's & Chinese Academy editorial statement </div>
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<a href="https://www.nih.gov/about-nih/who-we-are/nih-director/statements/statement-claim-first-gene-edited-babies-chinese-researcher" target="_blank">NIH</a><br />
<a href="https://www.biocentury.com/bc-extra/clinical-news/2018-11-28/%E2%80%98tepid%E2%80%99-response-crispr-embryo-editing-could-lead-regulations-" target="_blank">FDA</a></div>
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<a href="https://www.nytimes.com/2018/11/28/world/asia/gene-editing-babies-he-jiankui.html" target="_blank">New York Times</a></div>
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<a href="https://www.bbc.com/news/world-asia-china-46368731" target="_blank">BBC</a></div>
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<a href="https://apnews.com/3dcd5d7c599e4694b2f13c6ab03ef399" target="_blank">Associated Press</a></div>
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<a href="https://www.technologyreview.com/s/612458/exclusive-chinese-scientists-are-creating-crispr-babies/?utm_campaign=site_visitor.unpaid.engagement&utm_source=linkedin&utm_medium=add_this&utm_content=2018-11-26" target="_blank">MIT Tech Review</a></div>
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<a href="https://ipscell.com/2018/11/why-crispr-baby-production-if-it-happened-was-unethical-dangerous/" target="_blank">The Niche</a><br />
<a href="https://www.sciencemag.org/news/2018/11/i-feel-obligation-be-balanced-noted-biologist-comes-defense-gene-editing-babies" target="_blank">Science</a> - George Church, geneticist, interview</div>
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At the conclusion of the 2nd Human Genome Editing Summit, where the above referred to announcement was debated, a <a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=11282018b&_ga=2.188205291.2140619369.1543541989-1851845224.1537921260" target="_blank">statement</a> by the Organizing Committee was made on the science (<a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=11282018b&_ga=2.188205291.2140619369.1543541989-1851845224.1537921260" target="_blank">here</a>).<br />
<b><br /></b>Related Blog posts on the developing technology can be found on the <a href="https://msemporda.blogspot.com/p/dna.html" target="_blank">Genetics</a> page - specifically <a href="https://msemporda.blogspot.com/p/nih-statement-on-editing-human-embryo.html" target="_blank">Germline Science & Embryo Use</a> and <a href="https://msemporda.blogspot.com/2015/03/the-rise-of-germline-science-human-dna.html" target="_blank">The Rise of Germline Science</a>. </div>
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Cheers</div>
@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-36084367940442833222018-08-06T12:46:00.000-07:002018-08-23T12:18:44.380-07:00<div style="text-align: center;">
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<span style="font-size: large;"><b>Kyoto Univ team eyes clinical test of platelets derived from iPS cells</b></span></div>
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A Kyoto University team plans to begin a clinical test using platelets grown from induced pluripotent stem cells to treat Aplastic anemia, a serious disease, sources with knowledge of the plan said Sunday.</div>
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The team led by professor Koji Eto is expected to begin the clinical test after getting approval from the Ministry of Health, Labor and Welfare. The university has already endorsed the plan.</div>
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Expectations are high in the field of regenerative medicine for iPS stem cells that can grow into any type of body tissue.</div>
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The world's first transplant of retinal cells grown from iPS cells into a patient has already been conducted and research is under way into the application of such cells to treat Parkinson's disease and heart failure.</div>
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So far, the team has established a method to produce high-quality platelets -- a key component of blood that can stem bleeding -- in large numbers through the use of iPS cells.</div>
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In the envisaged clinical test, the team plans to grow platelets from iPS cells from a patient. They will be injected into the patient to see the safety and effectiveness of the method, according to the sources.</div>
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Patients with Aplastic anemia are easy to bleed, get infections, and suffer from headaches as platelets and white blood cells decrease.</div>
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Patients with falling numbers of platelets are often treated with blood transfusions. But the patient in the upcoming clinical study has a special immune type and thus the use of platelets derived from the patient's own iPS cells can control rejection, the sources said.</div>
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Platelets are used in blood transfusions during surgery and to stem bleeding when people get injured.</div>
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Blood donations are one of the most useful ways to secure platelets for use in medical setting. But platelets can be used for a short period of time and ensuring stock is a challenge.</div>
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Source: <a href="https://japantoday.com/category/national/kyoto-univ.-team-eyes-clinical-test-of-platelets-derived-from-ips-cells" target="_blank">Japan Today</a> </div>
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<span style="font-family: inherit; font-size: large;"><b>Japan Announces Physician-initiated clinical trials for Parkinson's disease using iPSCs</b></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Kyoto University Hospital, in partnership with the Center for iPS Cell Research and Application (CiRA), Kyoto University, has planned physician-initiated clinical trials for Parkinson's disease that transplants dopaminergic progenitors1 generated from induced pluripotent stem (iPS) cells. The clinical trial notification was submitted to the Pharmaceutical and Medical Devices Agency (PMDA; the Japanese equivalent of the FDA) on June 4, 2018, and the clinical trials are scheduled to begin on August 1 this year.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Note: The candidate subjects are required to live in Japan, have Japanese public health insurance and can understand the Japanese informed consent form.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Outline of the Clinical Trials</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">1. Names</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Selected patients will participate in both of the following clinical trials.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">a) Kyoto Trial to Evaluate the Safety and Efficacy of iPSC-derived dopaminergic progenitors in the treatment of Parkinson's Disease (Phase I/II)</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">b) Kyoto Trial to Evaluate the Safety and Efficacy of Tacrolimus2 in the iPSC-based Therapy for Parkinson's Disease (Phase III)</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">2. Objectives</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">a) To evaluate the safety and efficacy of transplanting human iPS cell-derived dopaminergic progenitors into the putamen3 of Parkinson's disease patients.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">b) To evaluate the safety and efficacy of using tacrolimus for Parkinson's disease patients who received transplantation of human iPS cell-derived dopaminergic progenitors into their putamen.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">3. Strategy</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Dopaminergic progenitors are generated from iPS cells prepared at the iPS Cell Stock for Regenerative Medicine4 at CiRA and then transplanted into the bilateral putamen of seven subjects (Parkinson's disease patients) at the Kyoto University Hospital. </span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">The source iPS cells were generated from third-party donor blood cells, meaning the transplantations will be allogeneic. Because of a possible transplant rejection, patients will receive a standard immunosuppressant, tacrolimus. Each subject will be observed for two years post transplantation.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">4. The cell transplantation surgery</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Approximately 5 million iPS cell-derived dopaminergic progenitors will be transplanted by stereotaxic brain surgery5 into the left and right sides of the patient's putamen. </span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Glossary</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">1)Dopaminergic progenitors</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Dopaminergic neurons produce the neurotransmitter dopamine. In Parkinson's disease, these cells degenerate, resulting in decreased dopamine production. Dopaminergic progenitors differentiate into dopaminergic neurons. Animal studies have shown that transplanted progenitor cells will differentiate into mature dopaminergic neurons, resulting in efficient engraftment in the brain.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">2)Tacrolimus</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">An immunosuppressant commonly used following organ transplantation.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">3)Putamen</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">A region in the basal ganglia that is innervated by midbrain dopaminergic neurons.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">4)iPS Cell Stock for Regenerative Medicine</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Clinical-grade iPS cells are generated from healthy donors with specific cell types (HLA homozygosity) that are less likely to cause immune rejection in many people, and are stockpiled at CiRA following thorough quality check.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span></span><br />
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">5)Stereotaxic brain surgery</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">This neurosurgery involves drilling small holes on the patient's skull, through which a needle is entered or electrodes are embedded. One of the current therapies for Parkinson's disease, deep brain stimulation (DBS) surgery, is a type of the stereotaxic brain surgery.</span></span></span></div>
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<span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Sources: <a href="http://www.cira.kyoto-u.ac.jp/e/pressrelease/news/180730-170000.html" target="_blank">CiRA PR</a> </span></span><br />
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<span style="font-family: inherit; font-size: large;"><b>Japan approves clinical trials of stem cell treatment for Parkinson's disease - Kyoto University to conduct first-ever iPS-based test for incurable disease</b></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">KYOTO (Kyodo) -- A Kyoto University research team said Monday it will begin this week a clinical test using induced pluripotent stem cells to treat Parkinson's disease, in what will be the world's first application of iPS to the progressive neurological disorder.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">The team led by Jun Takahashi, a professor at the university's Center for iPS Cell Research and Application, has received government approval and is soliciting several patients to participate in the trial to be conducted at Kyoto University Hospital, according to sources close to the matter.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">The team is scheduled to hold a press conference to explain the details of the clinical test later in the day.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Parkinson's disease reduces dopamine-producing neurons in the brain and results in tremors in the hands and feet, and stiffness in the body. While there are treatments to relieve the symptoms, there is currently no cure for the disease.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">In the clinical test that will start on Wednesday, nerve cells derived from other people and stored at the university will be transplanted into the brains of patients to supplement damaged nerve cells.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">The team has already tested the process on monkeys in a preclinical study, in which the movement of the affected animals improved without seeing any tumors that could develop into cancer in the brain over a two-year observation period.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">The clinical trial will be led by doctors who will determine the safety and effectiveness of the test.</span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Among other clinical tests of iPS cells, the government-backed Riken institute conducted the world's first transplant of retinal cells grown from iPS cells to a patient suffering from a serious disease in 2014.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Osaka University is also planning a clinical test for treating heart failure by using a heart muscle cell sheet created from iPS cells.</span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Source: <a href="https://asia.nikkei.com/Business/Science/Japan-approves-clinical-trials-of-stem-cell-treatment-for-Parkinson-s-disease" target="_blank">Nikkei Asian Review</a> </span></span></span></div>
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<span style="font-family: inherit;"><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;"><br /></span></span><span style="font-family: inherit; font-size: small;"><span style="font-weight: normal;">Channel News Asia <a href="https://www.channelnewsasia.com/news/asia/japan-parkinsons-worlds-first-human-trial-using-stem-cell-10574720" target="_blank">Coverage of Pakinson's iPSC Trial Topic</a></span></span></span></div>
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-26586857072770445722018-01-24T17:25:00.004-08:002018-01-24T20:52:58.980-08:00Early Safety and Efficacy Data in Cynata’s Phase 1 Trial of CYP-001 in GvHD<div class="MsoNormal" style="text-align: justify;">
<b><i>Encouraging Early Safety and Efficacy Data in Cynata’s Phase
1 Trial of CYP-001 in GvHD; DSMB Recommendation to Initiate Enrolment
of Second Patient Cohort</i></b></div>
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Australian stem cell and regenerative medicine company, Cynata
Therapeutics Limited (ASX: CYP) announced that the independent
Data Safety Monitoring Board (DSMB) has recommended that Cynata’s clinical trial
of its lead Cymerus™ mesenchymal stem cell (MSC) product CYP-001 should
progress to the next stage as planned.<o:p></o:p></div>
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<b>Key Highlights:</b><o:p></o:p></div>
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• All eight participants in Cohort A (lower dose cohort)
have demonstrated at least a Partial<o:p></o:p></div>
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Response (defined as an improvement in the severity of GvHD
by at least one grade compared<o:p></o:p></div>
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to baseline)<o:p></o:p></div>
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• No treatment-related serious adverse events or safety
concerns have been identified<o:p></o:p></div>
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• DSMB recommendation to progress clinical trial to second
cohort (Cohort B)<o:p></o:p></div>
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• Patient enrolment in Cohort B (higher dose cohort) now
open at seven trial sites in the U.K.<o:p></o:p></div>
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and Australia<o:p></o:p></div>
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Cynata’s clinical trial, which is the first clinical trial
in which patients have been treated with an allogeneic, induced pluripotent stem cell (iPSC)-derived
therapeutic MSC product, consists of a planned total of 16 patients with steroid-resistant acute
graft-versus-host disease (GvHD). The recommendation to progress to the next stage (Cohort B) followed an
independent review by the DSMB of the eight participants in Cohort A. Recruitment for Cohort A commenced
in May 2017, and there are currently seven trial sites active and ready to enrol participants
into Cohort B.</div>
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Steroid-resistant GvHD patients today have a dismal
prognosis, where mortality rates are very high. At this time, seven of the eight participants in Cohort A are
alive. One participant died after developing pneumonia, which is a common finding in recipients of bone
marrow transplants and similar procedures.1 This death was not considered to be
treatment-related. Participants enrolled in Cohort A of the dose-escalation
trial received a dose of CYP-001 that was anticipated to be at the lower end of the effective dose
range (one million cells per kilogram of bodyweight, up to a maximum of 100 million cells per
infusion). In Cohort B, a further eight participants will receive two infusions of CYP-001 at a dose of two
million cells per kilogram of bodyweight, up to a maximum of 200 million cells per infusion.</div>
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Dr Ross Macdonald, CEO of Cynata Therapeutics, said, “We are
thrilled to report this encouraging early review of the Phase 1 trial of CYP-001, which marks the
first time that patients have been treated with an allogeneic, induced pluripotent stem cell-derived
therapeutic MSC product. The improvement in GvHD grade observed in 100% of these gravely ill people is
very promising, especially given the low dose administered in Cohort A. The positive DSMB recommendation
is an important milestone that enables us to begin enrolment in Cohort B, and advance toward our
goal of completing the trial later this year. A successful outcome will support the application of CYP-001
in many medically and commercially significant targets where therapeutic MSCs have shown
promising results.”</div>
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<b>Next Steps:</b><o:p></o:p></div>
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<br /></div>
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Patient enrolment into Cohort B is now open at seven active
sites across the U.K. and Australia. Cynata looks forward to providing further updates to the market as
the study progresses.</div>
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<br /></div>
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Ref: <a href="http://cynata.com/wp-content/uploads/2018/01/18.01.22.Encouraging-Early-Data-Cynata-Clinical-Trial-Proceeds.pdf" target="_blank">Cynata PR</a></div>
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-58248077646785023282017-01-30T08:22:00.001-08:002018-06-14T13:06:04.036-07:00Cynata & The Rubik of Cell Science<div style="text-align: justify;">
<span style="font-family: "helvetica neue" , "arial" , "helvetica" , sans-serif;">As with the sun rising in the East so have the first two pioneering programs using Induced Pluripotent Stem Cell (iPSC) technology for human clinical study. Independently initiated, but now strategic brethren, the <a href="https://msemporda.blogspot.com/p/rik-ip.html" target="_blank">Japanese</a> and Australian efforts to provide a next gen proof-of-concept foundation have all the makings of a new dawn in Stem Cell Science, one that many hope will deliver on unfulfilled expectations for the field.</span></div>
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<span style="font-family: "helvetica neue" , "arial" , "helvetica" , sans-serif; font-size: 16px; vertical-align: baseline;">Methods and mechanics, protocols and parameters - these are but a few of the myriad of defining characteristics that a commercial cell product needs to elucidate in the highly complex rubik of molecular innovation and translational steps. Barcodes if you will of cell product definitions underlying the very nature of scientific clarity and positioning that will pave the road moving forward and deliver safely on <a href="http://msemporda.blogspot.com/2017/01/the-n-factor_3.html" target="_blank">The N Factor</a> of human trials with data.</span></div>
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<span style="font-family: "helvetica neue" , "arial" , "helvetica" , sans-serif; font-size: 16px; vertical-align: baseline;">To highlight movement in this direction I’ve addressed a few questions to Dr. Ross MacDonald, CEO of the Australian stem cell company <a href="http://cynata.com/" target="_blank">Cynata Therapeutics</a>, on recent progress with their iPSC programs and business plan for this update review piece on the <a href="http://msemporda.blogspot.com/p/cynata.html" target="_blank">previous conversation</a> I posted last year. </span></div>
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<span style="font-family: "arial"; font-size: 16px; vertical-align: baseline;">Q&A:</span></div>
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<span style="font-family: "arial";">M - Cynata is poised to begin an important sector accomplishment with a first-in-man clinical trial involving a therapeutic product derived from Allogeneic Induced Pluripotent Stem Cells (iPSCs). Can you kindly provide a brief refresh for us on the cell type/technology being used, the target indication and the clinical trial sites prep/patient application/data timelines?</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - We are using a clinical grade, human iPSC as the starting material. The iPSC cells were obtained from Cellular Dynamics International (CDI). From these cells we manufacture our finished product, a therapeutic mesenchymal stem cell (MSC) preparation, using our proprietary Cymerus™ manufacturing process. The target indication is steroid-resistant acute Graft versus Host Disease (GvHD) and the Phase 1 clinical trial is entitled: “An Open-Label Phase 1 Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease”. The trial will aim to recruit approximately 16 participants who have undergone a bone marrow transplant or similar procedure, and were subsequently diagnosed with steroid-resistant Grade II-IV acute GvHD. The study centers are located in the United Kingdom and in Australia and we expect the study should conclude by the end of 2017. GvHD is a potentially fatal disease that often follows a bone marrow transplant procedure and occurs when the immune cells in the donor material (the graft) attack the recipient’s tissues (the host) as “foreign”.</span><span style="color: blue;"> </span></span></div>
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<span style="font-family: "arial";">M - Your recent validating announcement that Cynata has signed a definitive License Option and Equity Investment Agreement with FujiFilm reiterates your strategy to develop and partner development programs with leading sector players. Could you review for us this strategy, the determining factors involved in the decision to sign a partner to your lead program at this stage and the synergy with FujiFilm’s Regenerative Medicine divisions moving forward.</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - Cynata’s Cymerus technology enables the economic manufacture of a consistent and robust therapeutic MSC product. Given the very many potential therapeutic applications for MSCs (noting the >600 clinical trials underway using MSCs) it would not be possible for Cynata on its own to adequately exploit even a small fraction of the potential commercial opportunities. Moreover, the Australian environment for biotech companies is challenging for those seeking to become a fully integrated, sales and marketing enterprise. Accordingly, we believe the best path to ensure shareholders derive stellar returns is to partner our technology with those companies that have the resources, expertise, enthusiasm and global exposure to drive commercialisation of our products. Clearly Fujifilm is a very active participant in the regenerative medicine sector and has shown a willingness to invest very heavily to maximise the chances of success. They are an ideal partner for Cynata. We have however left open the opportunity to work with other companies as well.</span></span></div>
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<span style="font-family: "arial";">M - In your press release on the deal with FujiFilm you mentioned that the arrangement included “certain rights to other Cynata technology.” Are you at liberty to detail somewhat the nature of the technology and the rights granted.</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - No.</span></span></div>
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<span style="font-family: "arial";">M - Currently Cynata has targeted UK/Europe and Australia as its initial trial territories and has received favorable green light feedback from the respective regulators. Could you comment on the developing regulatory framework to commence these types of pioneering iPSC studies in the US and Cynata’s plans for clinical work in the US market.</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - We have already had very positive initial dialogue with the US FDA. We plan to progress these interactions this year, and we will certainly seek to include US centres in our clinical trials as we move forward. The Regenerative Advanced Therapy Designation process, which the FDA has recently introduced, is of great interest to us and potentially has numerous advantages, including accelerated approval, greater interaction with the FDA, assistance with study design, smaller trials, and ability to rely on real world evidence rather than solely data from formal clinical trials.</span></span></div>
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<span style="font-family: "arial";">M - As Cynata is a leader in the development of Pluripotent derived Allogeneic iPSC treatment technology is there a perspective you could share that puts into context the application specific potential for programs that seek personalized treatment options with Autologous iPSC technologies versus Allogeneic iPSC approaches. Is there a case for both, putting aside the cost issues for a moment, with regard to targeted therapeutic potential, safety and immunogenicity? </span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - I do think there is a case for both, but as always it will come down to (i) the weight of clinical evidence and (ii) cost. Autologous iPSCs may have a greater role to play in tissue engineering applications, where the aim is directly replace or repair damaged tissue at a site of injury or disease. With our Cymerus technology we have sought to provide a solution to the practical shortcomings associated with manufacturing an “off-the-shelf” allogeneic MSC product. We firmly believe that our approach, ie using iPSCs, will yield a huge cost and regulatory advantage, at least insofar as MSC therapeutics are concerned. The broader applications of iPSC-derived cell therapy products is being hotly debated and much research capital is being expended on developing the “super haplobanks” to make allogeneic products more practical. </span></span></div>
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<span style="font-family: "arial";">M - As previously reviewed, the IP surrounding your technology was invented by scientists at the University of Wisconsin and exclusively licensed to Cynata by the University’s IP management arm, WARF. You note that that these foundational patents are being expanded upon by the filing of additional Cynata patent applications related to progressive discoveries to the underlying technology. Could you provide some specifics at this stage as to the substance of the novel and unique aspects of the invention mentioned, given, I believe, these innovations are as yet unpublished.</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - Indeed we are seeking to build the IP platform with additional patents around new inventions in our Cymerus technology. As these applications are in the early stages of prosecution we are unable to provide details at this stage.</span></span></div>
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<span style="font-family: "arial";">M - In respect to your other program assets, could you highlight recent developments that pertain to Cynata’s pipeline and any news on initiated research into new indications, such as the positive impact Cymerus™ mesenchymal stem cells (MSCs) could have for Asthma suffers.</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - The data we reported late last year in the well-established chronic allergic airways disease model was certainly very exciting and accordingly we have been encouraged to further study the effects of our Cymerus MSCs in related models and potentially into the clinical setting. We also have active programs underway in cancer (through Harvard/MGH) and in cardiovascular disease (with Westmead Hospital/University of Sydney). With the success of our recent capital raising the company is now very well positioned to accelerate these ongoing programs as well as to consider other areas where we might be able to quickly build value.</span></span></div>
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<span style="font-family: "arial";">M - Finally, at present Cynata is a publicly traded entity on the Australian securities exchange (ASX). Do you have any plans in the near term to reach out to more investors through a UK or US listing?</span></div>
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<span style="font-family: "arial";"><span style="color: #073763;">R - This is a question that stimulates much discussion around the boardroom tables of Australian biotech companies. Certainly US and European investors have a very high level of understanding of the potential risks and rewards of biotech and the availability and quantum of capital is typically higher than in Australia. However, an offshore listing is not for the faint hearted and some recent spectacular fails have made Aussie’s very circumspect. We have an open mind, but would prefer to really build a solid and supportive shareholder base in Australia first and thus have a solid foundation beneath us before we would contemplate a foreign exchange.</span></span></div>
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<span style="font-family: "arial";">M - Thank you Ross for your time and all the best for the upcoming trial. Cheers</span><br />
<span style="font-family: "arial";"><br /></span>
<span style="font-family: "arial";">UPDATE: See latest Cynata program news at <a href="https://msemporda.blogspot.com/p/cynata.html">"Cynata's iMSCs" blog tab</a>.</span></div>
@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-91838195194440583012017-01-03T11:33:00.002-08:002017-06-29T20:58:56.076-07:00The N Factor<div class="separator" style="clear: both; text-align: center;">
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<span style="color: black; font-family: "arial"; font-size: 14.6667px; vertical-align: baseline; white-space: pre-wrap;">The mere mention of a possible stop-gap option to the grave medical realities of those that suffer, let alone a lifeline to a solution, is enough to get most patients and their families/friends talking. Clinical trials however are relatively few and far between for specific diseases and those that are available are currently akin to drawing the lottery where circumstances such as referral, timing, scale, proximity and onerous inclusion criteria set a very high bar to entry.</span><br />
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">The vast majority of patients don’t have any options.</span><br />
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">I’ll relay a representative case in point - a friend of mine was diagnosed recently with Stage 4 Melanoma, an advanced skin Cancer which is all but fatal in relatively short order. His doctors gave him the ticking clock and little hope. However, given his wish to live and determination to see his young son grow up he studied, turned over every rock and reached out. He landed a very rare spot as a tag along to a new immunotherapy trial. He was lucky, most are not, given he didn’t meet the strict criteria for official entry. The trial treatment saved his life.</span></div>
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<span style="font-size: 14.6667px; line-height: 20.24px; vertical-align: baseline; white-space: pre-wrap;"><span style="font-family: "arial";">Hope is a cruel bedfellow with its constant draw of energy and resources from the very reservoir it seeks to fill. Yet without that flicker of a flame gasping for oxygen the very medical system built upon empirical data derived from clinical trials designed to advance knowledge wouldn’t exist.</span></span></div>
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">The system needs patients and patients need the system - both rely on the other and as such require inclusive practices to further the agenda of progress towards real solutions.</span></div>
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<tr><td class="tr-caption" style="font-size: 12.8px; text-align: center;"><span style="font-size: xx-small;">Emily Whitehead</span></td></tr>
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<span style="color: black; font-family: "arial"; font-size: 14.6667px; vertical-align: baseline; white-space: pre-wrap;">N is a strong and decisive letter in our everyday language but in investigative medicine it’s a defining research statement which confers status and validity where quantitative analysis imbues significance and wields judgement on meaningfulness. For those not on the in, the N is translational science parlance for the number of patients treated and their data sets. The lower the N the more likely there will be variability in the eventual safety and benefit outcomes when applied to the patient population at large. While the larger the N the more significant the potential correlation in delivering a safe option and potentially treating the targeted disease indication. </span><br />
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">As fundamental as the N is to applying the scientific method to proving the safety and potential for wide scale application, it cannot be suggested to be devoid of meaning at any level, especially considering the human impact and nascent development of promising new therapeutic modalities for treating previously unmet medical needs.</span><br />
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">The mere fact that we will all suffer in one form or another from the debilitating conditions of age is enough to warrant more attention to the secondary benefits in N data given the lack of achieved endpoints of trialed drug interventions to-date. Combine the natural degenerative conditions of the aged with a population rife with chronic and acute medical issues that continue to overburden the system, leaving a significant population of those in need without a solution, and you have the argument for considered change.</span><br />
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">Breakthroughs in medical science indeed offer a real opportunity to effect change and the more that can be done to allow for inclusion and support for that transformative process to occur the more representative the N will be that correlates to real tangible wellbeing data logged for the betterment of the science as well as the patient. </span><br />
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<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">Cheers</span></div>
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<span style="font-family: "arial"; font-size: 14.6667px; vertical-align: baseline; white-space: pre-wrap;">Ref: <a href="http://msemporda.blogspot.com/p/cancer.html">Related Cancer Blog on Emily Whitehead</a></span>@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-10791051424985288222016-08-24T18:20:00.000-07:002016-08-28T12:02:36.631-07:00The Coming of Age of Pluripotent Science & Musings on a Sonogram<div class="MsoNormal" style="margin-bottom: 0.0001pt; text-align: justify;">
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<tr><td class="tr-caption" style="text-align: center;">usnews</td></tr>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">One of the most memorable images I can recall on the effect of catalytic dynamics for me during these years of scientific curiosity was the explosive result of sperm enzyme successfully impacting a human egg - almost Big Bang like in all its microscopic potential.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">One can now extend that impact phenomena analogy to the very pertinent research and translational effect Induced Pluripotent Stem Cell (iPSC) technology has had on the field of molecular biology and regenerative medicine.</span></span></div>
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<tr><td class="tr-caption" style="text-align: center;">Nature - Andy Potts</td></tr>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">It’s been ten years since Shinya Yamanaka and</span></span><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;"> Kazutoshi Takahashi opened the portal to a whole new way of thinking and practicing the Art of Stem Cell Science by announcing that embryonic like properties could be regained in adult cells through molecular reprogramming.</span></div>
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<tr><td class="tr-caption" style="text-align: center;">Royal Society</td></tr>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Much has been written about this apex</span></span><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;"> moment of foundational innovation which ushered in the era of mainstream adoption of reverse engineering techniques on human cells but as we celebrate the ten year anniversary of iPSCs the opportunity presents itself to reflect and celebrate the coming age of Pluripotent science, specifically iPSCs.</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgH1MFJhWUidlvtXHVsHVOH0qNj8fOv-Bdu69FfYkExBSk3BGtM6cxLEeygyWmfP5y6rwJAAtfhvlxobZ9t4LL1njoAYFE1VVh9w2fB5VJUqTUC-W5-PpiW-eaP05CDhcLf5nr3FEmMJg6L/s1600/signals.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><img border="0" height="82" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgH1MFJhWUidlvtXHVsHVOH0qNj8fOv-Bdu69FfYkExBSk3BGtM6cxLEeygyWmfP5y6rwJAAtfhvlxobZ9t4LL1njoAYFE1VVh9w2fB5VJUqTUC-W5-PpiW-eaP05CDhcLf5nr3FEmMJg6L/s200/signals.jpg" width="200" /></a><span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><a href="http://www.signalsblog.ca/" target="_blank">Signals</a> of Canada, a leading destination for "insiders’ perspectives on the world of regenerative medicine and stem cell research, written by scientists and professionals in the field" is hosting a "<a href="http://www.signalsblog.ca/a-complete-reprogram-to-recognize-the-10-year-anniversary-of-ipscs/" target="_blank">Blog Carnival</a>" of which this article is one of a number being written covering the iPSC anniversary topic. Please click <a href="http://www.signalsblog.ca/a-complete-reprogram-to-recognize-the-10-year-anniversary-of-ipscs/" target="_blank">here</a> to read what other bloggers think.</span></span><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgOrtxC6LmfeI_mABsgI-HB0H1ZBAfET3kvQrK_GKZ45B6ynUgRg34iJkqE-32-7MP2qs4GWxSEmezA05EzNEGwz935mkKPln4n3AdvtqNeicRqO39F2Vflq4P8f9ImHsvOMmZyNivutW89/s1600/Pluripotent-map.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" height="220" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgOrtxC6LmfeI_mABsgI-HB0H1ZBAfET3kvQrK_GKZ45B6ynUgRg34iJkqE-32-7MP2qs4GWxSEmezA05EzNEGwz935mkKPln4n3AdvtqNeicRqO39F2Vflq4P8f9ImHsvOMmZyNivutW89/s320/Pluripotent-map.jpg" width="320" /></a><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">I</span><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">nherently complex the various Pluripotent states and the multitude of their progressively differentiated descendants, as they relate to human biological microsystems, have only begun to be explored and understood. The inherent processes by which these interconnected derivative cells work and communicate are by and large just now being decoded and mapped. Although it’s been only ten years since the discovery of iPSCs and nearly 20 years since the human embryonic stem cells were first isolated the progress made to-date in translating Pluripotent science into real world clinical programs is very much the focus now of countless labs in the field, thanks in large part to the advent of “open source” iPSC technology. </span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">Prior to iPSCs the use of hESCs and the technology associated with its clinical translation was largely a specialty area limited in scope by funding, rules, regulations and IP. The advent of iPSCs changed that and with it the stem cell industry added a universal layer of potential. Whether it be using Pluripotent derived cells as tools or more notably to develop therapeutic cell candidates for clinical use those researching and developing applications using these cells are pioneering the way forward for the emerging era of next generation stem cell products.</span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">To put not too fine a point on it, we have only scratched the surface and in the next ten years I expect there will be a number of Pluripotent treatments on the market in various countries and many many more still in the clinic moving towards approval with positive results over standard of care or filling-in where there is currently nothing to offer patients in need.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">The topic of what can we expect to see on the frontline of the developing therapeutic market using Pluripotent derived cell products is often highlighted as a discussion point and rightly so given the limited public depth of awareness on the subject and the long standing promise by the sector as a potential basis for effective treatments.</span></span></div>
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<span style="font-family: "arial" , "helvetica" , sans-serif;"><span style="font-size: 18px;">Below are some of the targets and iPSC programs representative of the state of play in the field to look out for:</span></span></div>
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<table border="0" cellpadding="0" cellspacing="0" style="border-collapse: collapse; width: 582px;"><colgroup><col style="width: 38pt;" width="51"></col><col style="width: 89pt;" width="118"></col><col style="width: 80pt;" width="107"></col><col style="width: 17pt;" width="23"></col><col style="width: 48pt;" width="64"></col><col style="width: 81pt;" width="108"></col><col style="width: 83pt;" width="111"></col></colgroup><tbody>
<tr height="19" style="height: 14.4pt;"><td class="xl71" colspan="7" height="19" style="height: 14.4pt; width: 436pt;" width="582"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b>TARGET - COMPANY/INSTITUTION - LEAD SCIENTIST(S) - CELL - </b></span><br />
<span style="font-family: "arial" , "helvetica" , sans-serif;"><b>DISEASE - AUTO/ALLO*</b></span></td></tr>
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<tr height="19" style="height: 14.4pt;"><td class="xl68" height="19" style="height: 14.4pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b>EYE</b></span></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
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<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://msemporda.blogspot.com/p/rik-ip.html">Riken/Healios</a></td><td class="xl65" colspan="2"><a href="http://www.cdb.riken.jp/en/research/laboratory/takahashi.html">Masayo Takahashi</a></td><td class="xl72">iRPE++</td><td class="xl72">Wet/Dry AMD++</td><td class="xl72">Auto>Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="6" height="19" style="height: 14.4pt;"> Notes: Program will address many disease states of Retina/Eye</td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td class="xl65"></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="https://astellas.us/therapeutic/rnd/airm.aspx">Astellas RegMed</a></td><td class="xl65"><a href="http://www.nature.com/articles/srep29784#abstract">Lanza/MacLaren</a></td><td></td><td class="xl72">iRNP++</td><td class="xl72">Dry AMD/RP++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="6" height="19" style="height: 14.4pt;"> Notes: Program will address many disease states of Retina/Eye</td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td class="xl65"></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="https://www.waisman.wisc.edu/scrp-gamm-lab.htm">UWisconsin Waisman</a></td><td class="xl65"><a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3412675/#__ffn_sectitle">Gamm/Meyer</a></td><td></td><td class="xl72">iRNP/RPE</td><td class="xl72">Stargardt+</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="6" height="19" style="height: 14.4pt;"> Notes: Representative of next gen concept using eye "organoids" </td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td class="xl65"></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://www.genengnews.com/gen-news-highlights/cdi-receives-1-2m-nei-contract-to-develop-ipscs-for-amd-trial/81250519/">NEI/CDI-Fuji</a></td><td class="xl65"><a href="https://patentscope.wipo.int/search/en/detail.jsf;jsessionid=2049987DBB9CCFBFEC7448B7C32514BD.wapp2nC?docId=WO2014121077&recNum=1&maxRec=&office=&prevFilter=&sortOption=&queryString=&tab=PCT+Biblio">Bharti/Miller</a></td><td></td><td class="xl72">iRPE</td><td class="xl72">Dry AMD</td><td class="xl72">Auto</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="3" height="19" style="height: 14.4pt;"> Notes: US Govt backed program </td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td class="xl65"></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="https://www.cirm.ca.gov/our-progress/people/shaomei-wang">Cedars Sinai/CIRM</a></td><td class="xl65"><a href="https://www.cedars-sinai.edu/Research/Research-Labs/SWang-Lab/">Shaomei Wang</a></td><td></td><td class="xl72">iRNP</td><td class="xl72">RP</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="5" height="19" style="height: 14.4pt;"> Notes: Advanced status w/ IND enabling studies </td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td class="xl66"></td><td></td><td class="xl65"></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl68" height="19" style="height: 14.4pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b>BRAIN</b></span></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl68" height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://www.cira.kyoto-u.ac.jp/jtakahashi/eng/index.html">Kyoto Univ</a></td><td class="xl65"><a href="https://www.cira.kyoto-u.ac.jp/e/research/takahashi_summary.html">Jun Takahashi</a></td><td></td><td class="xl72">iNC</td><td class="xl72">Pakinsons</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="6" height="19" style="height: 14.4pt;"> Notes: Leading iPSC Parkinson program due to start in 2017</td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td class="xl65"></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="https://www.mskcc.org/research-areas/programs-centers/stem-cell-biology">Sloan Kettering</a></td><td class="xl65"><a href="https://www.mskcc.org/research-areas/labs/lorenz-studer">Lorenz Studer</a></td><td></td><td class="xl72">eNC/iNC</td><td class="xl72">Pakinsons</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="5" height="19" style="height: 14.4pt;"> Notes: Top tier US hESC/iPSC lab moving to clinic</td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td class="xl65"></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://www.scripps.edu/loring/">Scripps/CIRM</a></td><td class="xl65"><a href="http://msemporda.blogspot.com/p/jeanne-loring-of-scripps-research.html">Jeanne Loring</a></td><td></td><td class="xl72">iNC</td><td class="xl72">Pakinsons</td><td class="xl72">Auto</td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="5" height="19" style="height: 14.4pt;"> Notes: Bringing it home full circle w/ CIRM onboard </td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl68" colspan="2" height="19" style="height: 14.4pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b>IMMUNE</b></span></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl68" height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" height="19" style="height: 14.4pt;"><a href="http://msemporda.blogspot.com/p/cynata.html">Cynata</a></td><td></td><td class="xl65" colspan="2"><a href="http://www.stemcells.wisc.edu/node/209">Slukvin/U Wisconsin</a></td><td class="xl72">iMSC</td><td class="xl72">GvHD++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" colspan="6" height="19" style="height: 14.4pt;"> Notes: Entering clinic later in 2016 w/ solid pre-clinical data</td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl66" height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="https://astellas.us/therapeutic/rnd/airm.aspx">Astellas RegMed</a></td><td class="xl65"><a href="http://online.liebertpub.com/doi/abs/10.1089/scd.2013.0554">Robert Lanza</a></td><td></td><td class="xl72">iMSC</td><td class="xl72">Sepsis, Lupus++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="6" height="19" style="height: 14.4pt;"> Notes: Multiple targets across board w/ pre-clinical hPSC data</td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl69" height="19" style="height: 14.4pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b>BLOOD</b></span></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl69" height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://www.megakaryon.com/en/technology/">Megakaryon-Kyoto/Tokyo+</a></td><td class="xl65" colspan="2"><a href="http://www.cell.com/cell-stem-cell/abstract/S1934-5909%2814%2900012-5">Eto/Nakauchi/Daley</a></td><td class="xl72">iPlatelets</td><td class="xl72">Cancer/Surgery++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="6" height="19" style="height: 14.4pt;"> Notes: Leading Japanese program poised to enter clinic in 2017</td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://novosang.co.uk/">Novosang-Roslin/SNBTS++</a></td><td class="xl65"><a href="http://www.crm.ed.ac.uk/research/associate/snbts-cellular-therapy-development-centre">Marc Turner</a></td><td></td><td class="xl72">iRBC</td><td class="xl72">Thalassaemia++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="5" height="19" style="height: 14.4pt;"> Notes: Leading UK Consortia looking to clinic in 2017</td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="http://www.platod.fr/en/le-pipeline/">Inserm/PlatOD</a></td><td class="xl65" colspan="2"><a href="http://c.ymcdn.com/sites/www.celltherapysociety.org/resource/resmgr/2014_AnnualMtgPresentations/W4_D.Baruch.pdf">Dominique Baruch</a></td><td class="xl72">iPlatelets</td><td class="xl72">Cancer/Surgery++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="6" height="19" style="height: 14.4pt;"> Notes: Leading French program nearing clinic in 2017 </td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl65" colspan="2" height="19" style="height: 14.4pt;"><a href="https://astellas.us/therapeutic/rnd/airm.aspx">Astellas RegMed</a></td><td class="xl65"><a href="http://www.cell.com/stem-cell-reports/abstract/S2213-6711(14)00295-1">Robert Lanza</a></td><td></td><td class="xl72">iPlatelets</td><td class="xl72">Cancer/Surgery++</td><td class="xl72">Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="7" height="19" style="height: 14.4pt;"> Notes: Had a leading program using research grade iPSC line in 2013</td></tr>
<tr height="19" style="height: 14.4pt;"><td height="19" style="height: 14.4pt;"></td><td></td><td></td><td></td><td class="xl72"></td><td class="xl72"></td><td class="xl72"></td></tr>
<tr height="19" style="height: 14.4pt;"><td colspan="3" height="19" style="height: 14.4pt;">Players in CAR/Immuno Space</td><td></td><td class="xl72">iBloodCells</td><td class="xl72">Cancer/Immune</td><td class="xl72">Auto>Allo</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl73" colspan="7" height="19" style="height: 14.4pt; width: 436pt;" width="582"> Notes: Auto/Allo B, T, NK, DC+ benefits 4 immuno product requirements</td></tr>
<tr height="19" style="height: 14.4pt;"><td class="xl74" height="19" style="height: 14.4pt; width: 38pt;" width="51"></td><td class="xl74" style="width: 89pt;" width="118"></td><td class="xl74" style="width: 80pt;" width="107"></td><td class="xl74" style="width: 17pt;" width="23"></td><td class="xl74" style="width: 48pt;" width="64"></td><td class="xl74" style="width: 81pt;" width="108"><br /></td><td class="xl72"><br /></td></tr>
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: x-small;">*The above listing is representative of the sector and is not at all comprehensive. Apologies to the many great programs that should be there also.</span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">hiPSC science has industry wide support globally and is a mainstream technology acceptable in jurisdictions in which other ES methods face challenges. Translational hurdles for hiPSC are specific to their reprogramming and to the adult to youthful conversion which forms the basis of the applied technology. This presents an additional safety component to the already strict regulatory oversight applied to the clinical translation of Pluripotent programs now and in the future. </span></div>
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;"><b>Will they be highly successful and achieve revolutionary paradigm shifting status and establish new standards of care in their go-to-market quests?</b> </span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">That is a subjective question for each and every program and one which you could speculate on, yet it would be Hype to suggest definitively without established patient data. However, the indicative MOA and technology basis of those programs on the list point to a sound foundation to work from. </span></div>
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">In my opinion, Pluripotent science</span><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">, specifically hiPSCs as a universal technology,</span><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;"> has the very best chance to score across the board wins for the patient in areas of unmet medical needs. </span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">Why? </span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">Some of the reasons I have doggedly believed that lie in the very nature of the plasticity of the sources, youthful phenotypes, cell expression and innate modulatory properties. Other reasons specifically relate to the field’s capacity to precisely derive and modify them in-vitro while perfecting their required derivative purities and expandability to consistently replicate them indefinitely in volume under strict quality control for regulated mass market applications. </span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">This potential, if successfully delivered together, will usher in a new Pluripotent Era in the Stem Cell Story. </span></div>
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<span lang="EN-US" style="font-size: 13.5pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b><u>Commentary</u></b></span></span><br />
<span lang="EN-US" style="font-size: 13.5pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b><u><br /></u></b></span></span>
<span lang="EN-US" style="font-size: 13.5pt;"><span style="font-family: "arial" , "helvetica" , sans-serif;"><b>Are we at an inflection point?</b></span></span></div>
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjZbD6BZ8nSHiUscFJDAAmz3-QsjIMYQZwQ0dMd7OheFAbqYZ9d2ZkUVXvs7J_PkXE5NBuqb_SYmvnpgwBLHvD34Euzq5o-kFOFdOuD_W_3x67Z4IDjWjlt9eBHOo_2ivcYBIrr0Y-ncJ8k/s1600/pavasoni.JPG" imageanchor="1" style="clear: right; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><img border="0" height="212" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjZbD6BZ8nSHiUscFJDAAmz3-QsjIMYQZwQ0dMd7OheFAbqYZ9d2ZkUVXvs7J_PkXE5NBuqb_SYmvnpgwBLHvD34Euzq5o-kFOFdOuD_W_3x67Z4IDjWjlt9eBHOo_2ivcYBIrr0Y-ncJ8k/s320/pavasoni.JPG" width="320" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">pavasoni</td></tr>
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<span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">Certainly the perceived slow pace of translational activities has been a media drag on the sector, irrespective of the actual comparative timelines to move from bench to bedside. In retrospect there are still only a surprisingly small number of clinical programs in trials worldwide using Pluripotent derived cells, due in large part to stricter preclinical and regulatory standards applied to safety issues associated with these cell sources. So one would say we’re definitely due some momentum building inflection points…. </span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Human embryonic stem cell trials were initiated in the US some 6 years ago on the basis of only research cell lines after lengthy preclinical research and safety checks. Subsequent small studies in Europe and Korea using hESCs added additional safety data. These trials paved the way for iPSCs yet still today the only enrolled clinical trial for iPSC derived therapeutics is in Japan. This pioneering trial had been on hold for over a year as the cell source analysis flagged possible genetic instability issues and was switched out from an autologous approach to a recently approved allogeneic cell line and is due to resume in 2017. The one patient to-date receiving the iPSC based iRPE cell sheet for Wet AMD has been reported to be in good condition with no apparent safety issues associated with her procedure.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">The rigorous standards of the need to use an <a href="https://commonfund.nih.gov/stemcells/faq" target="_blank">NIH approved</a> clinical grade cell line in the US has delayed the start of US trials on iPSCs and only just recently has one been announced as available. Pre-clinical lab work one would assume would now need to be done using that line for the clinical trial programs wishing to enter the clinic sometime in the future (yrs) or approval sought and granted for proprietary lines already used for clinical prep on existing developmental programs. This safety issue, albeit necessary and prudent, has forestalled the advancement of Western work and raised the bar from where hESCs entered the clinic.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">When discussing timelines and where this segment of the sector is headed it’s important to factor in these types of regulatory hurdles one must overcome on the road to a Pluripotent IND, clinical trial approval and human phased testing. Trial design considerations require stringent oversight monitoring of Pluripotent trials and have and will slow down the translational best efforts of those academic and commercial players entering the space.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Generally if it takes longer that the average drug development process to see stem cell based products enter trials and progress through the Phases and a therapeutic emerge from any stem cell specific regulatory approval pathway people will continue to be disappointed. A scaled approach to the sector’s product entry and exit criteria seems reasonable given the variance of risks associated with different stem cell products under some form of <a href="http://www.ema.europa.eu/docs/en_GB/document_library/Report/2016/08/WC500211526.pdf" target="_blank">adaptive</a> umbrella.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Safety is of the essence and the nascent SC sector requires everyone err on the side of caution. This is the mantra I hear regularly and I can’t really say it’s not appropriate to a great extent. However, it is this writer’s opinion that the priority should not be overly weighted towards the slowest approach to protect the sector at the expense of patients willing to engage in regulated, open and comprehensive phase development programs designed to enroll and prove the science. Sufficient data can only be generated from a participatory system that is adaptive and accommodating not restrictive and burdensome by design or intent.</span></span><br />
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<b style="font-family: arial, helvetica, sans-serif; font-size: 18px;">A Quad Pregnancy demands care & attention.</b><br />
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg1pfZ6OSw5ejAOY8Yr9DAWBqIf-D9YGEgG5baeVXVX_w1KZbLTD1MioWRlpmvjLbFgphowhoieKD_9kHBFizvCPFcn5ep0AJ9_sXDJpItWszrxaYIojzuIU383ISmz6iJ_rom7Cck1Cgzw/s1600/quadruplets_ultrasound+www.lifesitenews.com.jpg" imageanchor="1" style="clear: left; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><img border="0" height="225" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg1pfZ6OSw5ejAOY8Yr9DAWBqIf-D9YGEgG5baeVXVX_w1KZbLTD1MioWRlpmvjLbFgphowhoieKD_9kHBFizvCPFcn5ep0AJ9_sXDJpItWszrxaYIojzuIU383ISmz6iJ_rom7Cck1Cgzw/s320/quadruplets_ultrasound+www.lifesitenews.com.jpg" width="320" /></a></td></tr>
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEixbo1fb3d-pLG1BF1AOquKHg5G8rgW2vsWEtoq9C9pYTWBqOlWVFaDy8uxeGA7e1yYSBKlSbT8ve0AlsJsEefEzlWJdFPoEhl8sGPynylbvTUqj0cFRm98NUwdAh8K2e96m43-vvIiNY6y/s1600/Ashley+Gardner+Quad+IVF+Reaction+cnn.com.jpg" imageanchor="1" style="clear: left; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><img border="0" height="179" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEixbo1fb3d-pLG1BF1AOquKHg5G8rgW2vsWEtoq9C9pYTWBqOlWVFaDy8uxeGA7e1yYSBKlSbT8ve0AlsJsEefEzlWJdFPoEhl8sGPynylbvTUqj0cFRm98NUwdAh8K2e96m43-vvIiNY6y/s320/Ashley+Gardner+Quad+IVF+Reaction+cnn.com.jpg" width="320" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">Ashley Gardner Quad IVF Reaction cnn</td></tr>
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<span style="font-family: "arial" , "helvetica" , sans-serif;"><span style="font-size: 18px;">When I recently scanned the Pluripotent sonogram I saw some Art where 4 heads appeared! Unlike our shock horror double take when 2 appeared in my wife’s scan, I was glad to see the 4 represented there, happily squished together and well. One seems to be growing bigger at the expense of a couple of the others but by and large it’s a Bridge Gang willing and able to take on the challenges when alive and kickin. I personally look forward to seeing them all born healthy and grow, in addition to their Olympic caliber Adult cousins. That would give us semi-oldies the best chance when it´s our turn to ask for help from those wise and experienced in the Jedi ways of healing. This is where Advocacy for Cures comes in. </span></span></div>
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh1Al37zHiv41gv6Zh9lsvvCV2-cJMPAAWt5Zwm09DqF_c16GGCYWuCRlrAqdIuvNXawTusZuGvCfG3wNqju84te_enx32YQEXjXyYcb_1HKKYC0PCrUQ4Xx9eH3p_b5pPKGT_yy4Wbkj1I/s1600/buzzfeed.jpg" imageanchor="1" style="clear: right; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><img border="0" height="238" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh1Al37zHiv41gv6Zh9lsvvCV2-cJMPAAWt5Zwm09DqF_c16GGCYWuCRlrAqdIuvNXawTusZuGvCfG3wNqju84te_enx32YQEXjXyYcb_1HKKYC0PCrUQ4Xx9eH3p_b5pPKGT_yy4Wbkj1I/s320/buzzfeed.jpg" width="320" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">buzzfeed</td></tr>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Hope is a powerful force and will always be there for patients in need. Unfortunately the reality is that most next generation Pluripotent cell solutions are still a few steps away, if not more, for those that suffer. High science, low science, no science - too many people continue to be excluded, lack alternatives, suffer & die from disease. Stem Cell Science offers potential solutions and requires stakeholders to rally around programs and data that deliver real world results, even marginal benefits over existing options while awaiting more advanced solutions.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">To conclude I have reported on the stem cell industry, specifically the Pluripotent segment for many years, and it has been easily influenced by sentiment and competitive currents rather than sharpening its aim on achieving sector growth. What we all care about most are real solutions for the long list of conditions that continue to ail us and for those that we love. Rather than future technology leading, current programs built on the foundation of pioneering efforts in both the Adult and Pluripotent fields need to be clinically accessible and then successfully delivered widely. Supporting and driving all </span></span><span style="font-family: "arial" , "helvetica" , sans-serif; font-size: 18px;">safe and effective stem cell solutions will propel the entire sector forward.</span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Safety with pace, open and inclusive. Driving patient centric solutions forward as a community, in a modernized regulatory environment, by design and for the people.</span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Advocacy for Cures. </span></span></div>
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Cheers</span></span><br />
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<span style="font-size: 18px;"><span style="font-family: "arial" , "helvetica" , sans-serif;">Refs:</span></span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif;">Cell Press Nucleus - "<a href="http://www.cell.com/nucleus-ipscs-decade-of-discovery" target="_blank">iPSCs: A Decade of Discovery</a>" (comprehensive review issue)</span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif;">Cell Stem Cell Editorial: "<a href="http://dx.doi.org/10.1016/j.stem.2016.01.023" target="_blank">10 Questions: Clinical Outlook for iPSCs</a>" Cell Stem Cell, Vol 18, Issue 2, 170-173, DOI: 10.1016/j.stem.2016.01.023 (included in review issue ref above)</span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif;">Ilic, D. and Ogilvie, C. (2016), "<a href="http://onlinelibrary.wiley.com/doi/10.1002/stem.2450/full" target="_blank">Human Embryonic Stem Cells — What Have We Done? What Are We Doing? Where Are We Going?</a>". Stem Cells. doi:10.1002/stem.2450</span><br />
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<span style="font-family: "arial" , "helvetica" , sans-serif;">The Niche, P.Knoepfler: "<a href="http://www.ipscell.com/2016/07/yamanakas-baby-turns-10-so-heres-top-10-ips-cell-hot-button-bullet-points/" target="_blank">Yamanaka's baby turns 10 so here's a top 10 list of IPS cell hot button bullet points</a>" </span><br />
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-7239285146448932312016-03-17T10:56:00.000-07:002017-01-05T08:54:26.636-08:00Balancing Paradigms with Mesenchymal Stromal Cells <div dir="ltr" style="margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-size: xx-small;">Steve Gschmeissner/Science Photo Library</span></td></tr>
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<span style="font-family: "calibri";"><span style="font-size: 14.6667px; line-height: 20.24px;">Innovation isn't uniquely devoid of commonality of adoption by discipline. Rather the </span></span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;">likelihood of </span><span style="font-family: "calibri";"><span style="font-size: 14.6667px; line-height: 20.24px;">acceptance generally tracks evenly to historical norms in parallel with society's openness to progress and the search for solutions. However, the impact of technological change is variable and dependent on societal factors related to income and health. One could argue the greatest benefit comes when change drives both economic prosperity and improved health standards. </span></span><br />
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<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;">While the average pace of technological innovation slowed some decades ago the recent rapid rise of medical science has taken on the mantle of sustainability for growth. The dramatic impact potential of fundamentally transformative practices in healthcare is being fueled by access to new knowledge and a greater sharing of insight. </span><br />
<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;"><br /></span>
<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;">Today, due to the convergence of various technology led disciplines, t</span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;">here are many important catalysts for paradigm shifting change. A </span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;">key criteria common to all are the Drivers - fundamental products or processes that opens up the gates to new realms of understanding and acceptance. At each juncture a bridge must span the divide and a stake ground into new terrain. </span><br />
<span style="font-family: "calibri";"><span style="font-size: 14.6667px; line-height: 20.24px;"><br /></span></span><span style="font-family: "calibri";"><span style="line-height: 20.24px;"><i><b>Are MSCs a Driver that can forge a paradigm shift in stem cell healthcare & how did we get here?</b></i></span></span><br />
<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;"> </span><span style="font-family: "calibri";"><span style="font-size: 14.6667px; line-height: 20.24px;"> </span></span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;"> </span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;"> </span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px;"> </span><br />
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<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.38;">The investigation of bone marrow (“BM”) stem cells led to the establishment and widespread clinical practice using cells of the mesodermal blood lineage via bone marrow transplantation – known as hematopoietic cells (“HSCs”). </span></div>
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<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">The first use of these BM stem cells as therapy was pioneered over 50 years ago when transplants were first introduced experimentally to treat leukemia. However, as with most donor tissue the understanding of immune rejection of foreign non-self cells was and still is of major concern for the successful treatment of disease using allogeneic (donor) tissue. This is even the case when immuno-histocompatibility is done via matching of the cells to the host. This complication has stymied the field of cellular therapeutics due to the severe adverse events that can result from the administration of donor derived cellular treatments. In the case of BM transplantation they routinely cause Graft versus Host Disease (“GvHD”) as a result of the treatment, with approximately 50% of all such patients reporting complications. The percentage of mortality as a result of this last resort treatment intervention even today is staggering with up to 17% of all severe liver/gut GvHD cases resulting in death(<a href="http://www.mesoblast.com/product-candidates/oncology-hematology/acute-graft-versus-host-disease" target="_blank">1</a>).</span></div>
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhGIrlukOuPNa9IWECi34Ad6IMKnBCGuLe42boaesaNF_wcD9dMtcfJt6HCHMp1tK9Y4qkU7Q8BfqS4CibTz0fVwYaQ3IYE0Reronvqne81p-Om6Er9xdR2IlnJ_UpbBCjk2vqFhR9ckvt_/s1600/MSC-tissue-types.jpg" imageanchor="1" style="clear: right; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><span style="color: black;"><img border="0" height="262" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhGIrlukOuPNa9IWECi34Ad6IMKnBCGuLe42boaesaNF_wcD9dMtcfJt6HCHMp1tK9Y4qkU7Q8BfqS4CibTz0fVwYaQ3IYE0Reronvqne81p-Om6Er9xdR2IlnJ_UpbBCjk2vqFhR9ckvt_/s320/MSC-tissue-types.jpg" width="320" /></span></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;"><span style="font-size: xx-small;">NIH.gov</span></td></tr>
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<span style="background-color: transparent; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">As a field the discovery and isolation of Mesenchymal Stromal Cells (“MSCs”), a small subset of BM niches representing less than 0.01% of all HSCs, was a watershed moment. It was a true breakthrough as these cells were found to be able to replicate as multipotent precursors and can be differentiated into fat, bone and cartilage. The isolation and clonal nature of these MSCs opened up a whole new avenue for cellular investigation. Further sources of MSCs were discovered in a range of bodily tissues, including fat, perinatal tissue and dental pulp. The technology for human application of these adult cells gave rise to the stem cell industry we know today. Upwards of 500+ clinical trials using MSCs are registered currently in the US central database clincaltrial.gov for a variety of unmet disease indications (<a href="https://clinicaltrials.gov/ct2/results?term=mesenchymal+cells&no_unk=Y" target="_blank">2</a>). </span></div>
<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">In addition, there is a large growing trend of undocumented cases using MSC products in private medical offices as marketed treatments via autologous (self-to-self) therapies (<a href="http://journals.lww.com/neurotodayonline/Fulltext/2015/10220/Unregulated_Stem_Cell_Clinics_Proliferate_in_the.1.aspx" target="_blank">3</a>). These unlicensed medical practitioners using MSCs products are the subject of considerable debate as to where the line should be drawn between required regulatory oversight and freedom of medical use in private clinics for autologous treatments. The US FDA is currently reviewing draft guidelines (<a href="http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/ucm427692.htm" target="_blank">4</a>,<a href="http://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/UCM469751.pdf" target="_blank">5</a>,<a href="http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/ucm427795.htm" target="_blank">6</a>,<a href="http://www.fda.gov/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/Tissue/ucm419911.htm" target="_blank">7</a>) for treatment products using MSCs. They are preparing to define what constitutes </span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">more than minimal manipulation and cell use parameters. This is with a view to determining clinical trial requirements for MSC biologics, in keeping with current drug development procedures already in place.</span><br />
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<i><span style="font-family: "calibri"; line-height: 1.38; white-space: pre-wrap;"><b>Safe and Effective?</b></span><span style="font-family: "calibri"; line-height: 1.38; white-space: pre-wrap;"><b> </b> </span></i><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;"> </span></div>
<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">The prospect of MSC utility for therapeutics has been due in large part to the evident immunological privileged nature of MSCs and their potential for universal application without immunosuppressive drugs – unlike HSCs themselves. Although MSCs have an antigen profile they lack major class antigens which makes them relatively immune-privileged to the host system thereby allowing for donor derived cell treatments without treatment rejection in low dose regimes. </span></div>
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-size: xx-small;">The Scientist - Keith Kasnot</span></td></tr>
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<span style="background-color: transparent; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">The properties of MSC have been appropriately described as “ambulatory” and “paramedic” – i.e. they’re built to respond to injury in the body and assist in its repair. How they detect, migrate and signal, in addition to what biological manner they act, and what way in different circumstances, is a source of considerable study. It seems clear though now that their “method of action” (“MOA”) is modulatory in nature via complex regulatory mechanisms (<a href="http://www.cell.com/cell-stem-cell/abstract/S1934-5909(13)00406-2" target="_blank">8</a>). One such mechanistic attribute is via the excretion of bioactive factors (vesicles, exosomes et al) and work to facilitate cell to cell communication networks (<a href="http://onlinelibrary.wiley.com/doi/10.1002/stem.2298/abstract;jsessionid=B6CC452E95134F98E8633FAE7FDE3080.f04t01" target="_blank">9</a>).</span></div>
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<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">Much has been written about the potential of tissue derived MSCs as a treatment option for a host of acute, immune and degenerative conditions. However, the field is still developing and protocols are being tested and adjusted to maximize possible outcomes. I’ve added an overview video below on the challenges and issues faced by MSCs product developers’ to-date by a leading expert in the field Dr. Jacques Galipeau of Emory University. The presentation highlights a number of findings on research and data in this sector and is well worth watching</span></div>
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<iframe allowfullscreen="" class="YOUTUBE-iframe-video" data-thumbnail-src="https://i.ytimg.com/vi/uLWjb6irjO0/0.jpg" frameborder="0" height="266" src="https://www.youtube.com/embed/uLWjb6irjO0?feature=player_embedded" width="320"></iframe></div>
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<span style="font-size: xx-small;">Dr. Jacques Galipeau of Emory University</span></div>
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<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 20.24px; text-align: justify; white-space: pre-wrap;"><br /></span></div>
<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">As mentioned, and referred to in the video, numerous clinical studies are underway on the use of MSCs and case reports have been published on both the potential benefits and in certain cases a lack of statistical benefit in patients receiving these cells from a variety of tissue sources.</span></div>
<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">With regard to the clinical trial results there is clear validation of MSCs safety profile, which is fundamental to their successful translation. Potential treatment efficacy of MSCs is suggestive to-date of positive activity on various outcome measures in a number of reported studies. These positive results are counter-balanced with questions on method of action (“MOA”) and some failed studies. This somewhat mixed picture generally points to issues relating to the development of medicinal products and cellular biologics should be viewed as no different. </span></div>
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<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">A few of the better known company examples of MSC sector developments in the sector are briefly summarized below with links to the company for further details on the data.</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjNBFLPzdppx00HqafnT6xfVUsbJoZUSrOptwTk8RGIViDaCbCLGkZrPqwi8d3l8ScTZPO9YTzzBmU5GlLxKdX4Qs0p_4Z4Q1o_nNU-L18IkKaSp_6xl1P9_iGdXR9OURv7JHmOfS3GQjV1/s1600/Tigenix_logo_colour_rgb.jpg" imageanchor="1" style="clear: right; float: right; font-size: 14.6667px; margin-bottom: 1em; margin-left: 1em;"><span style="color: black;"><img border="0" height="56" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjNBFLPzdppx00HqafnT6xfVUsbJoZUSrOptwTk8RGIViDaCbCLGkZrPqwi8d3l8ScTZPO9YTzzBmU5GlLxKdX4Qs0p_4Z4Q1o_nNU-L18IkKaSp_6xl1P9_iGdXR9OURv7JHmOfS3GQjV1/s200/Tigenix_logo_colour_rgb.jpg" width="200" /></span></a><span style="background-color: transparent; font-family: "calibri"; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><a href="http://www.tigenix.com/" target="_blank"><span style="font-size: xx-small;">TiGenix</span></a><span style="font-size: 14.6667px;"> (adipose/fat) – has moved on from the 1st EU approved and marketed autologous (“auto”) MSC cell therapy called ChrondroCelect for cartilage repair to an allogeneic (“allo”) product strategy with solid Phase III results in hand for Cx601 in Crohn’s Disease. This will mark their first allo indication nearing approval with other adipose stem cell products in the pipeline. </span></span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgd2MDr4_9ZWCj7sbmFutUl1QhzX-9bL_iiQz5ELM8LTsYy6xcA306bSp-kzpYknptJxLa8GMFcX7aongV72W_8mwCaVM-VjA-RNKo5nfRq2ZzTjGnrPMICULWeGOK_5hbPxAaEA2h06PgV/s1600/Mesoblast.jpg" imageanchor="1" style="clear: right; float: right; font-size: 14.6667px; margin-bottom: 1em; margin-left: 1em;"><span style="color: black;"><img border="0" height="39" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgd2MDr4_9ZWCj7sbmFutUl1QhzX-9bL_iiQz5ELM8LTsYy6xcA306bSp-kzpYknptJxLa8GMFcX7aongV72W_8mwCaVM-VjA-RNKo5nfRq2ZzTjGnrPMICULWeGOK_5hbPxAaEA2h06PgV/s200/Mesoblast.jpg" width="200" /></span></a><span style="background-color: transparent; font-family: "calibri"; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><a href="http://www.mesoblast.com/" target="_blank"><span style="font-size: xx-small;">Mesoblast</span></a><span style="font-size: 14.6667px;"> (BM) – bought the first approved western auto cell therapy Prochymal for GvHD from Osiris which had mixed results and was never released. They are developing a full in-house line-up of allo product candidates with good support data and are partnered with a Teva Pharma. Notable pipeline news include marketing approval of TemCell in Japan for GvHD with local partner JCR Pharma (repackaged Osiris product) and solid data in late stage trials (MSC-100-IV for GvHD also, MPC-150-IM for heart and MPC-06-ID for back pain, amongst others).</span></span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiTBRZJGX7W3dgobqE8qtUBiFB00_X0G7RE1uy05myswAL1VKLVyMtJ_guh2EXYw8epZTkjm3QMBFoMwx-mtzmd754TcMco4JgfPoIfUmQjBTaZOY-lqP2YIdCW35bExWEpOYgqJbK6jX-Y/s1600/Atherysis.gif" imageanchor="1" style="clear: right; float: right; font-size: 14.6667px; margin-bottom: 1em; margin-left: 1em;"><span style="color: black;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiTBRZJGX7W3dgobqE8qtUBiFB00_X0G7RE1uy05myswAL1VKLVyMtJ_guh2EXYw8epZTkjm3QMBFoMwx-mtzmd754TcMco4JgfPoIfUmQjBTaZOY-lqP2YIdCW35bExWEpOYgqJbK6jX-Y/s1600/Atherysis.gif" /></span></a><span style="background-color: transparent; font-family: "calibri"; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><a href="http://www.athersys.com/" target="_blank"><span style="font-size: xx-small;">Athersys</span></a><span style="font-size: 14.6667px;"> (BM) – lost Pfizer as a program partner for MultiStem after releasing mediocre data in ulcerative colitis. A second Phase II read-out, this time in stroke, also failed to meet endpoints. However, newly released interim data in its ongoing stroke study is now suggestive of positive results from the homing-in strategy on potential earlier treatment window benefit. Also of note are the additional clinical programs in development for cardiovascular and inflammatory/immune indications. In addition there’s a solid validation deal with Healios of Japan for MultiStem in that market and use of the product for Healios’ ongoing development programs. </span></span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgg07donCq0HtBBiQ4m3864k8X4Gz8AAREs3cH6Laimpnc-gIzzO6YDIxW7rW0jTgoXTuThzsIfHxb_wUGdgJSio990PYJ_2HrSSOrSogYyvRL0VVKWb5MTA4m_7_zT_xEMGLoMh5xwFO6j/s1600/pluristem.jpg" imageanchor="1" style="clear: right; float: right; font-size: 14.6667px; margin-bottom: 1em; margin-left: 1em;"><span style="color: black;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgg07donCq0HtBBiQ4m3864k8X4Gz8AAREs3cH6Laimpnc-gIzzO6YDIxW7rW0jTgoXTuThzsIfHxb_wUGdgJSio990PYJ_2HrSSOrSogYyvRL0VVKWb5MTA4m_7_zT_xEMGLoMh5xwFO6j/s1600/pluristem.jpg" /></span></a><span style="background-color: transparent; font-family: "calibri"; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><a href="http://www.pluristem.com/" target="_blank"><span style="font-size: xx-small;">Pluristem</span></a><span style="font-size: 14.6667px;"> (placenta) – “PLX” product line for vascular, muscular and immune indications in early </span></span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.2; white-space: pre-wrap;">stage clinical trials (PI & PII) with solid data in muscle and critical limb ischemia. Promising preclinical results for bone marrow repair with government sponsorship for rapid route to market in acute radiation syndrome.</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgcJpQDHKF5DJ1j5xdA512MxMm8GuLoBGtsB4mu9WqdlG3tmiKCNP8WNI2O0hzM84I_oTQFUAfqXuwPUydrVaau2_ZIZgKtpVNQQYoNro7jkCudLABMx4Cd-XxgiO8XJEk6Vc2kL9qHVMhG/s1600/vericel.png" imageanchor="1" style="clear: right; float: right; font-size: 14.6667px; line-height: 17.6px; margin-bottom: 1em; margin-left: 1em;"><span style="color: black;"><img border="0" height="35" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgcJpQDHKF5DJ1j5xdA512MxMm8GuLoBGtsB4mu9WqdlG3tmiKCNP8WNI2O0hzM84I_oTQFUAfqXuwPUydrVaau2_ZIZgKtpVNQQYoNro7jkCudLABMx4Cd-XxgiO8XJEk6Vc2kL9qHVMhG/s200/vericel.png" width="200" /></span></a><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgcJpQDHKF5DJ1j5xdA512MxMm8GuLoBGtsB4mu9WqdlG3tmiKCNP8WNI2O0hzM84I_oTQFUAfqXuwPUydrVaau2_ZIZgKtpVNQQYoNro7jkCudLABMx4Cd-XxgiO8XJEk6Vc2kL9qHVMhG/s1600/vericel.png" imageanchor="1" style="clear: right; float: right; font-size: 14.6667px; margin-bottom: 1em; margin-left: 1em;"><span style="color: black;"><br /></span></a><span style="background-color: transparent; font-family: "calibri"; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><a href="http://vcel.com/" target="_blank"><span style="font-size: xx-small;">Vericel</span></a><span style="font-size: 14.6667px;"> (BM for heart program) – previously known as Aastrom </span></span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.2; white-space: pre-wrap;">with a long history of development</span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.2; white-space: pre-wrap;"> of auto MSCs for heart and CLI indications with poor accumulated data continues to develop the heart product in clinical studies with recent positive data after previous endpoint </span><span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.2; white-space: pre-wrap;">failure, indicative of statistical benefit. In 2014 they secured additional auto cell therapy products from Sanofi (Carticel & MACI – cartilage and Epicel – skin) which had previously received certain market authorizations and are generating revenue with patient benefit. </span></div>
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<span style="font-family: "calibri";"><span style="font-size: 14.6667px; line-height: 17.6px; white-space: pre-wrap;"><br /></span></span></div>
<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">Indicative data sets for comparative analysis and ratio breakout are yet to be tabulated with regard to which conditions and methodologies the cells work well for and in which cases they don’t help all that much or at all. However, one must be cautious when assessing the efficacy value of cellular products as they are biologics and there are many issues relating to their degree of effectiveness, such as: their source; derivation method; inherent donor variability; passage potency; culture conditions & scale-up manufacturing; cold chain methodology; target indication; patient population; disease states and application methods, amongst others. As a result not all cellular products will perform well in human studies. These issues play a significant role in whether they achieve benefit in tests on patients, and to what extent in relation to standard of care. Although the jury is still out there is a general agreement based on empirical data that these cells are on the whole safe, when developed and used appropriately. Where they have been shown to have positive outcome and biological activity there is acknowledged room for improvement with regard to enhancing efficiency, potency and cell mechanistic action, which is encouraging. </span></div>
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<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">One aspect of the development of industrial scale cellular therapies speaks to the need for increased replicative capacity, lower passaged products and standardization via use of optimization technologies and shifting to pluripotent cell sources instead of donor derived batch processing of multipotent cells. </span></div>
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgqFltkYNTjnTuXIkQ24merjA_-a7QySQDIQJ7bUlPTtglI-KTK5N348T2z2AsUegd6txCBV32QlRiiQVaZu46eet-qenGKh807OTZrxci0RqqtKKz1X1_rX59fNdMZpVUpzbeEYwttO1rQ/s1600/Octane+Bioreactors2.jpg" imageanchor="1" style="clear: left; line-height: 1.38; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><span style="color: black;"><img border="0" height="150" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgqFltkYNTjnTuXIkQ24merjA_-a7QySQDIQJ7bUlPTtglI-KTK5N348T2z2AsUegd6txCBV32QlRiiQVaZu46eet-qenGKh807OTZrxci0RqqtKKz1X1_rX59fNdMZpVUpzbeEYwttO1rQ/s320/Octane+Bioreactors2.jpg" width="320" /></span></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;"><span style="font-size: xx-small;"><a href="http://octaneco.com/" target="_blank">Octane</a> Bioreactors</span></td></tr>
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<span style="vertical-align: baseline;"><span style="font-family: "calibri";"><span style="font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">As a result of this progressive development culture method adjustments gleaned from the early pioneering work of MSC development are giving rise to efficiencies of process and improved </span></span></span><span style="font-family: "calibri"; font-size: 14.6667px; white-space: pre-wrap;">manufacturing protocols for next generation methods in both multipotent and <a href="http://msemporda.blogspot.com.es/p/cynata.html" target="_blank">pluripotent products</a>. The above mentioned early leaders in MSC product offerings are beginning to line up their treatments for entry to the market, while the sector looks to prepare and trial the more advanced cell factories of the future.</span><br />
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiD_XJ-TTYkjmbCPuaEowaWXAWTXdyZDWKAr6-Iul8L5xy-hOoyPlQKRS7OuRi397RqT8ZldjGHiB8JAMmP-gps7eXktdxApM8MdO7TlPC7SOwUlsQ-hK6Up5GikSXV8lsaVsboA2sAMuTi/s1600/Wheelock-Nolta-Arnett-Tempkin.jpg" imageanchor="1" style="clear: right; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><span style="color: black;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiD_XJ-TTYkjmbCPuaEowaWXAWTXdyZDWKAr6-Iul8L5xy-hOoyPlQKRS7OuRi397RqT8ZldjGHiB8JAMmP-gps7eXktdxApM8MdO7TlPC7SOwUlsQ-hK6Up5GikSXV8lsaVsboA2sAMuTi/s200/Wheelock-Nolta-Arnett-Tempkin.jpg" width="200" /></span></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;"><span id="docs-internal-guid-44619ff8-7fe2-3406-f84e-00263883d1a6"><span style="font-family: "calibri"; font-size: 10.6667px; vertical-align: baseline; white-space: pre-wrap;">UC Davis MSC <a href="http://www.ucdmc.ucdavis.edu/publish/news/newsroom/10872#.VujRsCQlV1o.twitter" target="_blank">Investigators</a></span></span></td></tr>
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<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">T</span><span style="font-family: "calibri"; font-size: 14.6667px; white-space: pre-wrap;">his momentum is also being driven by the rise of synthetic constructs using MSCs - the personalized tailoring of targeted medicines for improved performance. MSCs possess inherent homing and immunomodulatory properties and therefore are ideal for use in combination with gene and nano technologies. In addition, the extraction of the inherent cell properties of MSCs for standalone biologic products adds to the overall picture and excitement in the field.</span><br />
<span style="font-family: "calibri"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;"><br /></span></div>
<div dir="ltr" style="line-height: 1.3800000000000001; margin-bottom: 10pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">MSC products are representative of the wider cell therapeutic field and are the standard bearers in the effort to bridge the shifting paradigms of new treatment modalities for patients in need. </span></div>
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<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">Cheers</span><br />
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><br /></span>
<span style="background-color: transparent; color: black; font-family: "calibri"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">Ref: Sector Update on Asian Market for MSCs > "<a href="http://www.bioinformant.com/cell-therapy-in-asia/">Cell Therapy in Asia Erupts with Partnerships and Joint Ventures</a>"</span></div>
@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-43997556406209509492016-02-05T12:51:00.000-08:002016-02-06T01:02:05.098-08:00Progress and the Circle of Scientific Medical Innovation<div class="separator" style="clear: both; text-align: center;">
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh5AplexiT3mypmRTzZKOrhKBOURDZFrpQVhXA3QdGoPiOJ-1-szFix5kMy3ZJv4KCoL32KF0jfCu_TSSDVrXcJ1ieSGnjAr8wOWGt1xTm-CkbDvUKz2NCUXbiSMw12IFuHNmSpcmuR7YLS/s1600/Circle-of-Life2.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="161" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh5AplexiT3mypmRTzZKOrhKBOURDZFrpQVhXA3QdGoPiOJ-1-szFix5kMy3ZJv4KCoL32KF0jfCu_TSSDVrXcJ1ieSGnjAr8wOWGt1xTm-CkbDvUKz2NCUXbiSMw12IFuHNmSpcmuR7YLS/s200/Circle-of-Life2.jpg" width="200" /></a></div>
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Designing an adaptable system without the known variables is often an exercise in caution, unless of course you have been down a similar road before and can rely on established templates for creating repurposed guidelines. In some cases however old parameters simply don't fit any longer and the opportunity to engage in the development of more flexible rules with the benefit of hindsight becomes a net positive in the never ending cycle of innovation driven progress.</div>
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A series of high profile developments along this path have taken place of late on the topic of genetics and the advent of gene editing technologies that have the potential to alter the fate of human disease and the burden it represents to society. These policy reviews and the resulting position statements have been brought on by concerns that human gene editing presents a challenge to the perceived boundaries by which scientific discovery and possible therapeutic interventions are applied. </div>
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However, as we have seen knowledge trumps and is critical to all human endeavors, given information is paramount to decision making. Accumulating scientific data in the unknown cause and effect realm of biological systems provides the fundamental opportunity to address the task of solving real world problems. This is the tenant by which translational science has always operated and without which we wouldn't have made profound human advances to our condition. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEj7BqYh-e04pgyAJ3hvl8WhBjJZi_v0J34KCzbucG9YVPDagJNps0hBaqBR8zOZ6qiQtaN2g0-Lek7oApr-bwEMIxWBLtcAmC3iZNLB1ahCMvNs9VEpWugIHvxyuAO7-rurZdwOdvwrHBdo/s1600/UK+embryo+authority.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: justify;"><img border="0" height="98" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEj7BqYh-e04pgyAJ3hvl8WhBjJZi_v0J34KCzbucG9YVPDagJNps0hBaqBR8zOZ6qiQtaN2g0-Lek7oApr-bwEMIxWBLtcAmC3iZNLB1ahCMvNs9VEpWugIHvxyuAO7-rurZdwOdvwrHBdo/s200/UK+embryo+authority.jpg" width="200" /></a>Existing legislation in Western countries has provided a basis for clarification on the scope of scientific and translational activities, as reviewed <a href="http://msemporda.blogspot.com.es/2015/05/germline-science-embryo-use-law-scope.html" target="_blank">here</a>. Europe expressly prohibits at present germ line editing for reproduction. The UK has always had a pro-knowledge framework for discovery using early stage fertilized pre-embryos up to 14 days. The US presently restricts Federal Funding on embryo creation for research and when destructive practices are employed in the lab. </div>
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What is apparent now is the scientific community's consensus on the inherent value of lab study of early embryonic state development using genetic tools to advance knowledge through research while adhering to the principals of caution in progressing any attempts to implement the alteration of the germ line for reproductive purposes. This was the Middle Way path.</div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEge4l8d05e2Lsd2yMDuvWMO921-9Sg88GuTgMgSyuuelfxC_JKGGmV4DuIq4rp8ZeSxUky9jqdv5FAgTzkhyphenhyphenzfLnNoRq_64FF8EL2G8puOYiiBS9r3UyIhEjGQo5724mCxVaQTWTTWrb9An/s1600/NASEM.png" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="108" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEge4l8d05e2Lsd2yMDuvWMO921-9Sg88GuTgMgSyuuelfxC_JKGGmV4DuIq4rp8ZeSxUky9jqdv5FAgTzkhyphenhyphenzfLnNoRq_64FF8EL2G8puOYiiBS9r3UyIhEjGQo5724mCxVaQTWTTWrb9An/s320/NASEM.png" width="320" /></a></div>
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The US National Academies summation of their International Summit on Gene Editing <a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=12032015a" target="_blank">affirmed the above</a> in early December last year and called for an ongoing forum to further the dialogue on the topic as the science develops with a view to establishing new recommended guidelines if and when appropriate. The fluid nature of the science requires such and was therefore prudent to set this investigative precedent for all respective regulatory bodies to consider, including the US. </div>
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Previously the UK reaffirmed its position as a leading member of the international scientific community by being the first country to consider Mitochondrial DNA replacement therapy for families with genetic disorders wishing to have a baby free of the diseases associated with such inherited problems. Most recently the UK has agreed to allow genetic research on fertilized pre-embryos for infertility studies without intent to implant for reproductive purposes and always adhering to the prior 14 day limit on embryo development. A modification to the UK law was required for the Mitochondrial DNA therapy to proceed to review stage, while the research on early pre-embryos was already allowable under existing legislative framework which required prior approval and strict oversight.</div>
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Following on from the UK's position on Mitochondrial DNA therapy the US National Academies earlier this week <a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=21871" target="_blank">announced its recommendation</a> on this genetic intervention procedure for germ-line modification application. The result being <a href="http://www.nature.com/news/us-panel-greenlights-creation-of-male-three-person-embryos-1.19290" target="_blank">an additional affirmation</a> of the scientific potential to alleviate disease through continued research and potential use of the technology in the US. Notable was the necessary recommended investigational support for early pre-embryo studies. Current congressional <a href="http://www.nature.com/news/us-congress-moves-to-block-human-embryo-editing-1.17858" target="_blank">restrictions</a> inhibit actual Federal support for such studies on viable early stage pre-embryos. This may change in the fiscal year 2017 appropriations Bill as the restriction is not permanent. Support for non-viable pre-embryo research was expressly noted in the US National Academies recommendation paper. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhrDZ4sxKWJoe0gCeVRskmtn4GQ_sqa8IYJg1Ny7XokGBOzTHfmO0fy6IodOCufNZypMUA3wBpY4rGJ_L523i2U6gfP5hrPM8YjoYl-K8zE0QGOYtXQWqKae_GY8a3ElhWYYhWK_HmRuJaZ/s1600/ISSCR.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="63" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhrDZ4sxKWJoe0gCeVRskmtn4GQ_sqa8IYJg1Ny7XokGBOzTHfmO0fy6IodOCufNZypMUA3wBpY4rGJ_L523i2U6gfP5hrPM8YjoYl-K8zE0QGOYtXQWqKae_GY8a3ElhWYYhWK_HmRuJaZ/s200/ISSCR.jpg" width="200" /></a></div>
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgJD52GSA8sijlt6EQ6VbBgFjf8IYQ9PUViwnNtzkvqwhUckuhWy18uoBIOVNah8H2oXLRJsYPqYw7qbicKO7Ey3vyoHRqo4nESl0W1RLVCPPqNzrcrVpRAPYDgllnjOc2qTygbtix9-Ac2/s1600/CIRM.png" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="81" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgJD52GSA8sijlt6EQ6VbBgFjf8IYQ9PUViwnNtzkvqwhUckuhWy18uoBIOVNah8H2oXLRJsYPqYw7qbicKO7Ey3vyoHRqo4nESl0W1RLVCPPqNzrcrVpRAPYDgllnjOc2qTygbtix9-Ac2/s200/CIRM.png" width="200" /></a>Both the <a href="http://californiastemcellreport.blogspot.com.es/2016/02/california-crispr-conference.html" target="_blank">ISSCR</a> and <a href="http://californiastemcellreport.blogspot.com.es/2016/02/california-stem-cell-agency-not.html" target="_blank">CIRM</a> have previously stated that they support research on early stage pre-embryos for scientific purposes and have reiterated that position also this week while calling for renewed study of the societal implication of gene therapy and germ line editing.<br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhzTrAqnnw3yaAPNRm-LfzMnDbJgZc80wsgFehtiWe1Z7YcbFYMYXFr2meeO7qvXcrK0dDDtFKJexL0CmQHb2BVpHMx1t7KTpUHRpnZIeTOqmQP3UV3JOC8rUS3b72HEaQKrWVEUEpNL794/s1600/EPO.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhzTrAqnnw3yaAPNRm-LfzMnDbJgZc80wsgFehtiWe1Z7YcbFYMYXFr2meeO7qvXcrK0dDDtFKJexL0CmQHb2BVpHMx1t7KTpUHRpnZIeTOqmQP3UV3JOC8rUS3b72HEaQKrWVEUEpNL794/s1600/EPO.jpg" /></a>Commercially the recent change in the position of the European Patent Office on the acceptable use of non-viable pre-embryos methods via germ-line modification brings the alignment closer together and bodes well for the application of various stalled avenues of translational science for the benefit of patients in need. </div>
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Coming around full circle, there would be little progress without the support for all forms of scientific innovation.</div>
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Advocacy for Cures.<br />
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Cheers<br />
<br />
Refs:<br />
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1. International Summit on Human Gene Editing Washington DC - "<a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=12032015a" target="_blank">On Human Gene Editing: International Summit Statement</a>" (Dec. 3rd 2016)<br />
2. UK "<a href="http://www.bbc.com/news/health-35459054" target="_blank">Scientists get 'gene editing' go-ahead</a>" (BBC Feb. 1st 2016)<br />
3. The National Academies "<a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=21871" target="_blank">Clinical Investigations of Mitochondrial Replacement Techniques Are ‘Ethically Permissible’ If Significant Conditions Are Met, Says New Report</a>" (Feb. 3rd 2016)<br />
4. California Stem Cell Report items by David Jensen re: CIRM (<a href="http://californiastemcellreport.blogspot.com.es/2016/02/californias-stem-cell-agency-to-tackle.html" target="_blank">1</a>,<a href="http://californiastemcellreport.blogspot.com.es/2016/02/california-stem-cell-agency-not.html" target="_blank">2</a>) & ISSCR (<a href="http://californiastemcellreport.blogspot.com.es/2016/02/california-crispr-conference.html" target="_blank">3</a>) (Feb. 5th 2016)<br />
5. Related blog posts: <a href="http://msemporda.blogspot.com.es/2015/03/the-rise-of-germline-science-human-dna.html" target="_blank">1</a>, <a href="http://msemporda.blogspot.com.es/2015/05/germline-science-embryo-use-law-scope.html" target="_blank">2</a>, <a href="http://msemporda.blogspot.com.es/2015/05/germline-science-embryo-use-law-scope.html" target="_blank">3</a>, <a href="http://msemporda.blogspot.com.es/2015/07/congressional-debate-heats-up-on-germ.html" target="_blank">4</a>@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-5546814890625600942015-11-11T04:55:00.001-08:002016-01-20T08:51:47.449-08:00Science Validation & The Rising Sun<div class="separator" style="clear: both; text-align: center;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg_d0r4_cZ3YafduXMZOZk9mdfoGrHFL29hkE9Ls8fHbAN8WoVCvixhNRreRquYpBCjqz3vENN3jwPJjtxtRkpxAUDeCbasqjId6PmFCfh0fL5bs_BcYafPKWXCRc4uPeo9atNeixULdgoz/s1600/download2.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="132" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg_d0r4_cZ3YafduXMZOZk9mdfoGrHFL29hkE9Ls8fHbAN8WoVCvixhNRreRquYpBCjqz3vENN3jwPJjtxtRkpxAUDeCbasqjId6PmFCfh0fL5bs_BcYafPKWXCRc4uPeo9atNeixULdgoz/s200/download2.jpg" width="200" /></a></div>
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<span style="color: black; font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">Strategy, survival, direction, competition and growth opportunities are but a few fundamental elements in the maze of everyday life all professionals must navigate - no more so than those on the leading edge of managing scientific innovation. </span></div>
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<b id="docs-internal-guid-31550641-f682-4680-aae7-7c937be82e86" style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">Being at the forefront of change often elicits backdraft currents and polarization of entrenched positions. This is natural and can be seen as a positive reinforcement of the high threshold one must strive to in order to achieve acceptance. A sort of quality assay if you will - one which is analogous to peer review in a business setting.</span></div>
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<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">A lot has been written on the <a href="http://newsroom.astellas.us/2015-11-09-Astellas-to-Acquire-Ocata-Therapeutics#assets_136:21909" target="_blank">news</a> of the <a href="http://www.astellas.com/en/corporate/news/pdf/151110_2_Eg.pdf" target="_blank">Ocata/Astellas deal</a> regarding the value calculation and little on the actual merits of <a href="http://investorstemcell.com/forum/msemporda/34015.htm" target="_blank">Validation</a> for Ocata’s science, the step forward in its development plan and the broader implications for the stem cell sector.</span></div>
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<b style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">This writer has covered Ocata for a long time and can attest to the finer details of the saga that was its <a href="http://www.xconomy.com/boston/2015/11/10/with-379m-deal-for-ocata-astellas-buys-stem-cells-for-eye-diseases/" target="_blank">Survival and Vindication</a>. A road which has seen its fair share of episodic highlights - perhaps too many. </span></div>
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<b style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">One could speak at length and talk of the dedicated character of those involved with the science and the constant pressure within & on the company to prove itself in a demanding uncompromising field. One piece termed the company a “<a href="http://thepenngazette.com/the-lightning-rod/" target="_blank">lightning rod</a>” - the story has all the hallmarks. </span></div>
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<b style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">The <a href="http://www.ipscell.com/2015/11/ocata-bought-by-astellas-of-japan-initial-perspectives/" target="_blank">end of an era</a>? Yes, perhaps it is in many ways. I prefer to see it as the close of the 2nd act in a 3 act structure, where the rain is falling and mingling with the tears. </span></div>
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<b style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">Lost independence isn’t easy to come to grips with. However, moving on from being a small volatile publicly traded company, with all the influences that entails, to being housed within a protective and nurturing parental structure is a very positive outcome - for the programs and patients in need. </span><br />
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;"><br /></span>
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">It signifies so much with regard to the science and efforts to help define the standards of a new treatment methodology in medicine. </span><br />
<span style="font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;"><br /></span>
<span style="color: black; font-family: "arial"; font-size: 14.6667px; line-height: 1.38; white-space: pre-wrap;">The deal is a solid affirmation from established pharma that the stem cell therapeutic sector is worth banking on. This comes on the heels of other momentum building developments in the space and perhaps is indicative of a growth driven consolidation phase. </span></div>
<div style="text-align: justify;">
<b style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">There are many worthwhile questions surrounding the announcement and events leading up to the decision to sell that remain, even after recent company disclosures, and one would look to those involved to address them for the record. Lock stock comes to mind?</span></div>
<div style="text-align: justify;">
<b style="font-weight: normal;"><br /></b></div>
<div dir="ltr" style="line-height: 1.38; margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
<span style="background-color: transparent; color: black; font-family: "arial"; font-size: 14.666666666666666px; font-style: normal; font-variant: normal; font-weight: 400; text-decoration: none; vertical-align: baseline; white-space: pre-wrap;">There is a saying which is often spoken in Catalonia - “the sun always rises” and aims to reinforce and embrace the positive.</span></div>
<span style="font-family: "arial"; font-size: 14.6667px; vertical-align: baseline; white-space: pre-wrap;"></span><br />
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<span style="color: black; font-family: "arial"; font-size: 14.6667px; vertical-align: baseline; white-space: pre-wrap;"><span style="font-size: 14.6667px;">Cheers</span></span></div>
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</span>@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-32185887107744724382015-10-01T04:21:00.000-07:002015-11-02T04:26:06.817-08:00Naїve Human Pluripotency & The Broad Shoulders of Science <div class="MsoNormal" style="text-align: justify;">
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-family: inherit; font-size: xx-small;">Stevenage, UK BioScience Campus</span></td></tr>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;">Scientific debate in the pursuit of knowledge
by way of accumulated evidential data is fundamental, just as socio-economic competition
is needed to spur innovation, product development & growth in commercial
business. Distinct and largely operating on their own, these two worlds have now collided and become integrated in a synthetic process that is driving 21st century evolution. </span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;">As a pillar of progress and community
success, medical science is a central focus of tomorrow’s design. One
in which the health and well-being of society can be calculated and factored
into the spreadsheets of sustainability. The footnotes in such macros are
bolded as requirements to achieve, yet are a challenge to deliver. </span></span></div>
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-family: inherit; font-size: xx-small;">Salk iPS_Ruiz-StemCell</span></td></tr>
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<span style="font-family: inherit; font-size: x-small;">Given this backdrop the nature of a pluripotent
cell, with its ability to generate all tissue types, has been a hot topic of debate
and investigation for many years. Its potential is often cited but the field remains a few steps away with many questions still to be answered. </span><br />
<span style="font-family: inherit; font-size: x-small;"><br /></span>
<span style="font-size: x-small;">Source, Stability and Scale – the trinity of our destiny caught in a matrix of possibilities where clarity of method is needed. </span><br />
<span style="font-size: x-small;"><br /></span>
<span style="font-size: x-small;">S</span><span style="font-family: inherit; font-size: x-small;">cience knows no bounds when it comes
to unresolved issues of definition and process, so the discussion continues. However, with advents in genomic analysis the cell systems of our inner being are becoming clearer and these new insights are helping to provide the answers. </span></div>
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<span style="font-family: inherit; font-size: x-small;">The human "na</span><span style="font-size: x-small;">їve" cell state </span><span style="font-size: x-small;">in the earliest stages of human embryogenesis</span><span style="font-size: x-small;"> </span><span style="font-size: x-small;">is one such focus. The identification and establishment of cell lines along the pluripotent continuum has been a foundational endeavor of the community. Ever since the mouse modelling proved the existence of these powerful engines of growth have the leading labs sought to isolate and engineer the human equivalents. This ongoing work has inspired the field to challenge each other to discover and answer the unresolved questions that will</span><span style="font-size: x-small;"> unlock the full potential of pluripotency. </span><br />
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-family: inherit; font-size: xx-small;">Whitehead Institute MIT</span></td></tr>
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<span style="font-size: x-small;">In its so called </span><span style="font-size: x-small;">"na</span><span style="font-size: x-small;">їve"</span><span style="font-size: x-small;"> state the pre-programming of the early cell development machinery hasn't kicked off yet and committed to its natural tissue <span style="font-family: inherit;">generating pathways, hence the terminology. The use of cells at this early moment </span></span><span style="font-family: inherit; font-size: x-small;">in the cycle could</span><span style="font-size: x-small;"><span style="font-family: inherit;"> alleviate some of the drawbacks of the standard later stage "primed" version, </span>allowing for more efficient </span><span style="font-size: x-small;">homologous recombination</span><span style="font-size: x-small;"> in a therapeutic setting using reprogramming technologies. The </span><span style="font-size: x-small;">na</span><span style="font-size: x-small;">їve state</span><span style="font-size: x-small;"> also has a greater </span><span style="font-size: x-small;">proliferation </span><span style="font-size: x-small;">capability and can differentiate more effectively into all desired tissue types. In addition, these cells are able to form inter species chimeras for research and tissue engineering, a highly valuable addition to the toolbox. </span><span style="font-size: x-small;"> </span><br />
<br />
<span style="font-family: inherit; font-size: x-small;">Over the years I have looked for data on early stage embryonic states, specifically any variations in the genetic profiles of pre-compaction blastomeres and ICM hESCs. The </span><span style="font-family: inherit; font-size: x-small;"><a href="http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0013615" target="_blank">Galan, Á. et al (2010)</a></span><span style="font-family: inherit;"><span style="font-family: inherit; font-size: x-small;"> </span><span style="font-size: x-small;">Valencia paper was one such document I found. Of note here in the more recent research done on early human development was that the variation in profiling was correlated to naїve at a specific stage of human embryogenesis at around the 8 cell stage (referred to in Q&A). This moment evidently coincides to the withdrawal of maternal influence yet prior to the blastocyst wave of fate expression. </span></span><span style="font-size: x-small;"> </span><br />
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-family: inherit; font-size: xx-small;">Benjamin Dodsworth</span></td></tr>
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<span style="font-family: inherit; font-size: x-small;"><span lang="EN-GB">I touched on the pluripotent topic during my
<a href="http://msemporda.blogspot.com.es/p/the-pluripotency-trilogy.html" target="_blank">interviews</a> in </span><st1:country-region><st1:place><span lang="EN-GB">Sweden</span></st1:place></st1:country-region><span lang="EN-GB"> during this year’s annual <a href="http://msemporda.blogspot.com.es/p/isscr-2015.html" target="_blank">ISSCR 2015</a> conference and followed up by reading a then just published paper entitled</span><span lang="EN-GB"> “<a href="http://onlinelibrary.wiley.com/doi/10.1002/stem.2085/full" target="_blank">The Current State of Naїve HumanPluripotency</a></span></span><b style="font-family: Arial; font-size: 14.6667px; white-space: pre-wrap;">¹.</b><span style="font-family: inherit; font-size: x-small;"><span lang="EN-GB">” </span></span><span lang="EN-GB" style="font-size: x-small;"><a href="https://uk.linkedin.com/in/btdodsworth" target="_blank">Benjamin Dodsworth</a> of Oxford </span><span lang="EN-GB" style="font-size: x-small;">co-authored the work </span><span style="font-family: inherit; font-size: x-small;"><span lang="EN-GB">with his colleagues Rowan Flynn and <a href="http://www.wsjlab.com/meet-the-lab/sally-cowley/" target="_blank">Sally Cowley</a> </span></span><span style="font-size: x-small;">(team leader and head of the <a href="http://www.wsjlab.com/" target="_blank">James Martin Stem Cell Facility</a>, affiliated to the <a href="http://www.stemcells.ox.ac.uk/home" target="_blank">Oxford Stem Cell Institute</a>, at the <a href="http://www.path.ox.ac.uk/" target="_blank">Sir William Dunn School of Pathology, University of Oxford</a>).</span></div>
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<tr><td class="tr-caption" style="text-align: center;"><span style="font-family: inherit; font-size: xx-small;">Sally Cowley Ph.D</span></td></tr>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;">The passage in the paper's abstract about the naïve state
not being an “artifact” caught my attention and intrigued me given the differing opinions on the
subject, the extent to which mouse modelling is representative of human developmental biology and the evolving
genetic data analysis of early stage embryonic cell states.</span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"></span></span><br />
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;">I connected with <a href="https://uk.linkedin.com/in/btdodsworth" target="_blank">Ben</a> in a Twitter exchange and he was open to doing a Q&A on the topic, which we
started prior to some subsequent developments in the area (<a href="http://www.nature.com/nsmb/journal/v22/n9/full/nsmb.3066.html#affil-auth" target="_blank">iPS </a></span></span><span style="font-size: x-small;"><a href="http://www.nature.com/nsmb/journal/v22/n9/full/nsmb.3066.html#affil-auth" target="_blank">“2C” totipotent </a></span><span style="font-family: inherit; font-size: x-small;"><a href="http://www.nature.com/nsmb/journal/v22/n9/full/nsmb.3066.html#affil-auth" target="_blank">reprogramming</a></span><b style="font-family: Arial; font-size: 14.6667px; white-space: pre-wrap;">²</b><span style="font-family: "calibri"; font-size: 11pt; line-height: 107%;"> </span><span style="font-family: inherit; font-size: x-small;">and </span><span style="font-family: inherit;"><span style="font-size: x-small;">the</span> </span><a href="http://www.nature.com/ncomms/2015/150903/ncomms9207/full/ncomms9207.html#f1" style="font-family: inherit;" target="_blank"><span style="font-size: x-small;">Karolinska paper</span></a><b style="font-family: Arial; font-size: x-small; white-space: pre-wrap;">³</b><span style="font-family: "calibri"; font-size: xx-small; line-height: 107%;"> </span><span style="font-family: inherit; font-size: x-small;">on early human
development). Comments on the 2C paper are included in the interview below in [brackets]. </span></div>
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<span style="font-family: inherit; font-size: x-small;">Thank you Ben for your feedback & good luck with your research.</span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;">Cheers</span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit;"><u><b>Q&A:</b></u></span></span></div>
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<b><span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;">M - With regard to the human Naive state
generally</span></span><span style="font-family: inherit;"><span style="font-size: x-small;"> and attempts made to create hNaïve cell lines, are we really mainly
discussing iPS reprogramming techniques to revert to an earlier point of
embryogenesis or would you envision a new methodology for ICM hESC cell lines
with them being converted backwards post extraction also? If so do you envision
any technical issues associated with than or in their maintenance?</span></span></b><br />
<span lang="EN-GB" style="font-family: "microsoft sans serif"; font-size: 12.0pt; line-height: 107%; mso-ansi-language: EN-GB; mso-bidi-language: AR-SA; mso-fareast-font-family: "Times New Roman"; mso-fareast-language: EN-US;"></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Very good point. There are clear parallels
to iPS reprogramming techniques. We are currently looking at a method to
convert already established human pluripotent stem cell (hPSC) lines to the
naïve state. However, if the naïve state is indeed as useful as we anticipate
and becomes our new standard, I would expect the emergence of protocols to
generate naïve induced pluripotent stem cells directly from primary cells (such
as fibroblasts) which skip the primed state. If this holds true, I do expect
technical issues. Many protocols for handling hPSCs have been optimised for
cells in the primed state. These will not be ideal for naïve cells. Maintenance
of naïve human cells might also be challenging and current standard operating
procedures will have to be adapted.</span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - You mention hESC differentiation pathways
that are unreachable - which are those?</b></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Endodermal and germline lineages are
difficult to access with our current primed hPSCs. This means that although
possible, it is inefficient. The Hanna lab have actually used naïve cells to
generate primordial germ cells (PGCs) very efficiently. In comparison, primed
cells do not efficiently differentiate into PGCs.</span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;"><br /></span></span>
<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">Just as important as accessing these
differentiation pathways is the maturity of the cells we then produce. Maturity
is the extent to which their functions resemble the in vivo cell type. Naïve
hPSCs might increase the level of achievable maturity (for example of hepatocytes).</span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">But what I find a lot more interesting is
that we have excellent protocols for the differentiation into cells (for
example dopaminergic neurons) which work robustly with some hPSC lines but not
with others. This heterogeneity could be removed with a protocol which uses
cells that are developmentally at the same starting point and without
epigenetic bias. The naïve state could deliver on both of these aspects. </span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - Has there been any focus on comparative
analysis done using hESCs derived from various cell stages of the early human
embryonic Blastomere cell stages 2, 4, 8, 16?</b></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - To my knowledge this has not been performed
using hESCs derived from different developmental time points. However, a very
useful direct comparison of current naïve and primed hES lines to early human
embryonic blastomere cell stages has been performed using single cell
transcriptomics by Huang, Maruyama, and Fan</span></span><b style="font-family: Arial; white-space: pre-wrap;"><span style="color: #444444; font-size: x-small;">⁴</span></b><span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;"> (go directly to Figure 2B). They
used datasets from Vassena et al.</span></span><b style="font-family: Arial; white-space: pre-wrap;"><span style="color: #444444; font-size: x-small;">⁵</span></b><span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">, 2011, Xie et al.</span></span><b style="font-family: Arial; white-space: pre-wrap;"><span style="color: #444444; font-size: x-small;">⁶</span></b><span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">, 2010 and Yan et al.</span></span><b style="font-family: Arial; white-space: pre-wrap;"><span style="color: #444444; font-size: x-small;">⁷</span></b><span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">, 2013
and compared gene expression to various naïve cells.</span></span><span style="font-family: "arial"; white-space: pre-wrap;"><b><span style="font-size: 14.6667px;"> </span></b></span></div>
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<span style="font-family: inherit; font-size: x-small;"><br /></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - Why is the Naive state also referred to
as Ground State? Is there any technical reason?</b> </span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Ground state and naïve state both describe
the earliest accessible and unbiased cellular state. These terms are
interchangeable.</span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - Do you believe the reprogramming concept
being studied will ultimately be pursued to the point where reversion produces
a Totipotent state in order to fully map the process?</b></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Possibly, but there are many technical
hurdles to overcome and ethical issues to consider.</span></span><br />
<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;"><br /></span></span>
<span style="font-size: x-small;"><b>[M - Do you have a as follow-up comment on this point with regard to the recent Inserm Totipotent development?</b></span><br />
<span lang="EN-GB"><span style="color: #444444; font-size: x-small;"></span></span><br />
<span style="color: #444444; font-size: x-small;">B - The 2C paper is indeed a very interesting piece of work which I have been following closely. However, I would like to see more evidence for totipotency, in particular higher efficiency differentiation down difficult lineages such as PGCs. There is not enough evidence to show that these cells are indeed totipotent. For our lab, totipotent cells are unnecessary and we won’t be using these.]</span><br />
<span style="font-family: inherit; font-size: x-small;"><br /></span>
<span style="font-family: inherit; font-size: x-small;"><b>M - Do existing techniques adequately result
in Pluripotent cells able to be scaled and applied effectively to therapeutic
programs?</b></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Current techniques allow the production of
induced pluripotent cells to be scaled up. However, before iPS cells can be
used therapeutically, the field needs to overcome some fundamental issues. Two
main challenges revolve around the host eliciting an immune response to hES or
iPS cells even when sourced from the same individual and on the other hand,
pluripotent cells have been changed to allow proliferation. This raises concern
that these cells could be more susceptible to becoming cancerous. There is a
lot of preclinical work to be done.</span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span style="font-family: inherit; font-size: x-small;"><b><span lang="EN-GB">M -</span> In your conclusion you point to the
protocols yielding different results which has yet to be interpreted
conclusively, as well as the transient nature of the actual biological moment
in-vivo which it may occur. In ad<a href="https://draft.blogger.com/null" name="_GoBack"></a>dition you point to the
possibility of a scale of different states along the defined continuum. In that
respect would you say any in-vitro activity to reproduce these embryo-genesis
states are by defacto man made events and the best we can expect ultimately is
a "like" status?</b></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
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<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Absolutely. Any cell grown into the lab is
unlikely to be exactly the same as the in vivo counterpart. As long as we keep
this in mind and factor it into our data interpretation, this is not a problem.
</span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - The data you cite regarding the primate
transcript HERVH indicates that mouse systems are distinct to that of primates
in this specific area (at least that monkey species). This would indicate that
aspects of the human embryo-genesis system are biologically different to that
of mouse, in certain ways. Does that perhaps also apply to cell prodigy
behavior in your opinion?</b></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - The paper discussing HERVH is an excellent
piece of work which shows compellingly that pluripotency networks are indeed
different between human and mouse. And you are right, we can also see these
differences in the cellular behaviour. Mouse and human ES cells cannot be
cultured in vitro in the same way. The networks which allow capture of naive
pluripotency in mouse are not identical to the human system. </span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - The utility advantages you mention of
Naive versus Primed indicate manufacturing bias towards use of Naive in the
future. Can you outline the utility issues specifically for naive cell use and
do you view this for specific clinical purposes or for certain discovery
processes.</b></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - Although some labs are currently working on
clinical applications, we are focusing on using hPSCs for modelling only. The
human naive state promises a lot of benefits – if it is indeed similar to the
naive state in mouse. Extrapolating from the mouse, homogeneity would be
expected to be improved in naive cell populations. This means that cells are
held not in a spectrum of states but all at exactly the same developmental time
point. Differentiation protocols could be a lot more effective when applied to
a uniform starting point. Other benefits include higher cell yields due to
faster doubling times and easier handling. </span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><b>M - The statement that "TGFβ might not
be essential in the human system" caught my attention. Can you elaborate
on that in light of published data.</b></span></span></div>
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<span lang="EN-GB"><span style="font-family: inherit; font-size: x-small;"><br /></span></span></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-GB"><span style="color: #444444; font-family: inherit; font-size: x-small;">B - In the past, TGFβ signalling was required
to maintain hPSCs in culture. However, the requirement of TGFβ signalling is a
trait associated with the primed state. In addition, the inhibition of TGFβ
signalling increases efficiency of mouse iPSC reprogramming. This is why it
would be interesting if we can culture hPSCs without TGFβ.</span></span></div>
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<span style="font-family: inherit; font-size: xx-small;">##</span></div>
<br />
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: x-small;">[Follow-up Q relating to the Karolinska analysis paper</span><b style="font-family: Arial; font-size: x-small; white-space: pre-wrap;">³</b><span style="font-family: inherit; font-size: x-small;"> on early human development was left unanswered prior to publishing]</span></div>
<br /></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;"><u><b>Q&A Refs:</b></u></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;">1. Dodsworth, B. et al. (2015). The Current State of Naïve Human Pluripotency</span><span style="font-family: inherit; font-size: xx-small;">.</span><span style="font-size: xx-small;"> <span style="line-height: 19.5px;"><a href="http://onlinelibrary.wiley.com/doi/10.1002/stem.2085/full" target="_blank">Stem Cells. doi: 10.1002/stem.2085</a></span></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;"><br /></span>
<span style="font-family: inherit; font-size: xx-small;">2. Ishiuchi, T. et al (2015). Early embryonic-like cells are induced by downregulating replication-dependent chromatin assembly. <a href="http://www.nature.com/nsmb/journal/v22/n9/full/nsmb.3066.html#affil-auth" target="_blank">Nature Structural & Molecular Biology 22, 662–671 (2015) doi:10.1038/nsmb.3066</a></span><br />
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;">3. Töhönen, V. et al. Novel PRD-like homeodomain transcription factors and retrotransposon elements in early human development. <a href="http://www.nature.com/ncomms/2015/150903/ncomms9207/full/ncomms9207.html#f1" target="_blank">Nat. Commun. 6:8207 doi: 10.1038/ncomms9207 (2015).</a></span><br />
<span style="font-family: inherit; font-size: xx-small;"><br /></span>
<span style="font-family: inherit; font-size: xx-small;"><span lang="EN-GB" style="line-height: 19.046px;">4. Huang, K. et al. (2014). The </span><st1:placename><st1:place><span lang="EN-GB" style="line-height: 19.046px;">Na</span></st1:place></st1:placename>ï<st1:placename><st1:place><span lang="EN-GB" style="line-height: 19.046px;">ve</span></st1:place><span lang="EN-GB" style="line-height: 19.046px;"> </span><st1:placetype><span lang="EN-GB" style="line-height: 19.046px;">State</span></st1:placetype></st1:placename><span lang="EN-GB" style="line-height: 19.046px;"> of Human Pluripotent Stem Cells: A Synthesis of Stem Cell and Preimplantation Embryo Transcriptome Analyses. </span><a href="http://www.sciencedirect.com/science/article/pii/S1934590914004056" target="_blank"><span style="line-height: 19.046px;">Cell Stem Cell</span><span style="line-height: 19.046px;"> </span><span style="line-height: 19.046px;">15</span><span style="line-height: 19.046px;">(4): 410-415.</span></a></span><br />
<span lang="EN-GB" style="font-family: inherit; font-size: xx-small; line-height: 19.046px;"><br /></span>
<span style="font-family: inherit; font-size: xx-small;"><span style="line-height: 19.046px;">5. </span><span style="line-height: 19.046px;">Vassena, R. et al. (2011). </span><span style="line-height: 19.046px;">Waves of early transcriptional activation and pluripotency program initiation during human preimplantation development </span><span style="line-height: 19.046px;"><a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4074286/" target="_blank">Development 138, 3699–3709</a></span></span><br />
<span lang="EN-GB" style="font-family: inherit; font-size: xx-small; line-height: 19.046px;"><br /></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;"><span lang="EN-GB" style="line-height: 19.046px;">6. </span><span style="line-height: 19.046px;">Xie, D. et al. (2010). </span><span style="line-height: 19.046px;">Rewirable gene regulatory networks in the preimplantation embryonic development of three mammalian species" </span><span style="line-height: 19.046px;"><a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2877577/" target="_blank">Genome Res. 20, 804–815.</a></span></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small;"><span style="line-height: 19.046px;">7. </span><span style="line-height: 19.046px;">Yan, L. et al. (2013). </span><span style="line-height: 107%;">Single-cell RNA-Seq profiling of human pre-implantation embryos and
embryonic stem cells. </span><span lang="EN-GB" style="line-height: 21.4px;"><a href="http://www.nature.com/nsmb/journal/v20/n9/full/nsmb.2660.html" target="_blank">Nat. Struct. Mol. Biol. 20, 1131–1139.</a></span></span><br />
<span style="font-family: inherit; font-size: xx-small;"><br /></span>
<span style="font-family: inherit; font-size: xx-small;"><u><b>Selected Other Refs (in no particular order):</b></u></span></div>
<div class="MsoNormal">
<span style="font-family: inherit; font-size: xx-small; text-align: justify;"><br /></span>
<span style="font-family: inherit; font-size: xx-small; text-align: justify;">Takahashi/Yamanaka review of the iPS reprogramming pluripotency</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span>
<span style="font-size: xx-small;">Takahashi,K., et al. A developmental framework for induced pluripotency. <a href="http://dev.biologists.org/content/142/19/3274" target="_blank">Development 2015 142: 3274-3285; doi: 10.1242/dev.114249</a></span><span style="font-family: inherit; font-size: xx-small;"><a href="http://dev.biologists.org/content/142/19/3274" target="_blank"> </a></span><br />
______<br />
<span style="font-family: inherit; font-size: xx-small;">Salk paper on region specific PSCs (2015):</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Wu, J. et al. An alternative pluripotent state confers interspecies chimaeric competency. <a href="http://www.nature.com/nature/journal/v521/n7552/full/nature14413.html" target="_blank">Nature 521, 316–321 (21 May 2015) doi:10.1038/nature14413</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">_______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Genomic analysis using single cell RNA (2013)</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Xue, Z. et al. Genetic programs in human and mouse early embryos revealed by single-cell RNA sequencing. <a href="http://www.nature.com/nature/journal/v500/n7464/full/nature12364.html#affil-auth" target="_blank">Nature 500, 593–597 (29 August 2013) doi:10.1038/nature12364</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">_______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Naive cells in hESC culture using a HERVH promoter & gene analysis of ICM & early embryo cells</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Wang, J. et al. (2014). Primate-specific endogenous retrovirus-driven transcription defines naive-like stem cells. <a href="http://www.nature.com/nature/journal/v516/n7531/full/nature13804.html#affil-auth" target="_blank">Nature 516, 405–409, doi:10.1038/nature13804</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">_______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">1st Naive Paper MIT (w/ Hanna now in Israel, Weizmann)</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Hanna, J. et al. (2010). Human embryonic stem cells with biological and epigenetic characteristics similar to those of mouse ESCs. <a href="http://www.pnas.org/content/107/20/9222.full" target="_blank">Proc Natl Acad Sci U S A. 2010 May 18; 107(20): 9222–9227.</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">_______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">A.Smith Cambridge downstream transcription factor Tfcp2l1 in Naive conversion</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Martello, G. et al (2013). Identification of the missing pluripotency mediator downstream of leukaemia inhibitory factor. <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3791366/" target="_blank">EMBO J. 2013 Oct 2; 32(19): 2561–2574. doi: 10.1038/emboj.2013.177</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Singapore use of 3iL creates a closer native epiblast state of pluripotency "Naive" (rewiring of regulatory circuitry)</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Chan, Y-S. et al. (2013). Induction of a Human Pluripotent State with Distinct Regulatory Circuitry that Resembles Preimplantation Epiblast. <a href="http://www.sciencedirect.com/science/article/pii/S1934590913005067" target="_blank">Cell Stem Cell. 2013 Dec 5; Vol 13, Issue 6. doi:10.1016/j.stem.2013.11.015</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Hanna Weizmann Institute use of 2iL & in-vitro derivation of mouse like naive cells capable of forming inter-species mouse–human chimeric embryos</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Gafni, O. et al (2013). Derivation of novel human ground state naive pluripotent stem cells. <a href="http://www.nature.com/nature/journal/v504/n7479/full/nature12745.html" target="_blank">Nature. 2013 Dec 12;504(7479):282-6. doi: 10.1038/nature12745.</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Seattle Washington alternative derivation method to Naive state</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Ware, C. et al. (2014). Derivation of naïve human embryonic stem cells. <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3970494/" target="_blank">Proc Natl Acad Sci U S A. 2014 Mar 25; 111(12): 4484–4489. doi:10.1073/pnas.1319738111</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Whitehead MIT Talen mediated reporter system for naive derivation medium 5iL (R. Jaenisch)</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Theunissen, T. et al. (2014). Systematic Identification of Culture Conditions for Induction and Maintenance of Naive Human Pluripotency. <a href="http://www.cell.com/cell-stem-cell/fulltext/S1934-5909(14)00298-7" target="_blank">Cell Stem Cell doi: 10.1016/j.stem.2014.07.002</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">______</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">A. Smith Cambridge team uses simple transient expression of two transcription factors to rewire back to Naive</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Takashima, Y. et al (2014). Resetting Transcription Factor Control Circuitry toward Ground-State Pluripotency in Human. <a href="http://www.cell.com/cell/abstract/S0092-8674(14)01099-X" target="_blank">Cell, Vol.158, Issue 6, 2014 Sept 11. DOI: 10.1016/j.cell.2014.08.029</a></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">______</span></div>
<div class="MsoNormal">
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Valencia early embryo gene analysis</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Galan, Á. et al (2010). Functional Genomics of 5- to 8-Cell Stage Human Embryos by Blastomere Single-Cell cDNA Analysis. <a href="http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0013615" target="_blank">PLOS | One 2010, Oct 26. DOI: 10.1371/journal.pone.0013615</a></span></div>
<div style="text-align: justify;">
<span style="text-align: start;"><span style="font-family: inherit; font-size: xx-small;">______</span></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Developmental biology focus on human tissue</span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;"><br /></span></div>
<div style="text-align: justify;">
<span style="font-family: inherit; font-size: xx-small;">Gerrelli, D. et al. (2015). Enabling research with human embryonic and fetal tissue resources. <a href="http://dev.biologists.org/content/142/18/3073" target="_blank">Development 2015, Sept 15. doi: 10.1242/dev.122820</a></span><br />
_____<br />
<span style="font-family: inherit; font-size: xx-small;">Harvard led w/ Daley/Jaenisch/Rossant - Comments by Hanna</span><br />
<span style="font-family: inherit; font-size: xx-small;"><br /></span>
<span style="font-family: inherit; font-size: xx-small;">De Los Angeles, A. et al (2015). Hallmarks of pluripotency. <a href="http://www.nature.com/nature/journal/v525/n7570/full/nature15515.html#affil-auth" target="_blank">Nature 525, 469–478 (24 September 2015) doi:10.1038/nature15515</a></span></div>
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-15649593255530238342015-08-25T05:14:00.000-07:002015-08-26T02:53:47.347-07:00Sally Temple - Cells, Leadership & Audacious Innovation<div class="separator" style="clear: both; text-align: center;">
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<span style="font-family: Arial;"><span style="font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;">The eye is often referred to as the window into the soul. Whether that is true or not depends I suppose on who’s doing the viewing, as the subjective interpretation invariably dictates the meaning. This however in science can be balanced via rigorous protocols of evidence based assessment in a clinical setting and peer review. The subjective becomes objective and the perspective become clearer.
In the stem cell field some of the earliest approaches to regenerative medicine have been in the CNS, with a number of high profile clinical stage multi and pluripotent trials. Those in the clinic with ongoing trials are reporting promising indications of disease stability and restorative potential. More data is required but the overall momentum moving forward portends to a variety of treatment methodologies from a number of cell sources.
An active area of CNS clinical research has been for the retina, where there are unmet medical conditions in need of new effective solutions for low vision & blinding diseases. Early attempts to restore retinal function via the transplantation of donated adult and fetal retinal tissue and cells were deemed inefficient and lacked solid efficacy data. Those experiments however have paved the way for the current focus on using more developed & novel multipotent cells, as well as from pluripotent sources.
One such retinal program is being led by Dr. Sally Temple of the <a href="http://neuralsci.org/" target="_blank">Neural Stem Cell Institute</a> based on a unique population of adult retinal stem cells. I sat down with Sally at ISSCR 2015 and it's fitting she rounds out the Interview segments from Sweden as she is <a href="http://www.isscr.org/home/about-us/isscr-leadership/officers-and-board-of-directors" target="_blank">ISSCR's President Elect</a> now and is looking to the future, as we all are, with high expectations and great promise to meet those tangible opportunities head on.</span></span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg9OwWJu2MpSYWE9ZGaKz31Uuf9wPZSLlykMmWVcKzp3vu9rovRHLZhXmmNu49F4b6ywzpO8k-ld-MvlCi-88bXAXBKdTnLZMDuBMYP7reh3ayoi2AkIgLCGT6O1kCJ1eDmwBIBsixjE7XN/s1600/Stern_NSCI.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg9OwWJu2MpSYWE9ZGaKz31Uuf9wPZSLlykMmWVcKzp3vu9rovRHLZhXmmNu49F4b6ywzpO8k-ld-MvlCi-88bXAXBKdTnLZMDuBMYP7reh3ayoi2AkIgLCGT6O1kCJ1eDmwBIBsixjE7XN/s200/Stern_NSCI.jpg" width="200" /></a></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgX2kM6IynbcntZy6HcmoKbr1Vlnh91vb1mK4H-s9VEo1gPsqzGim5njbsTiP9WbTvE4nW7ClHQh3rk1N8W0ROM5-VL5DGlxKrwiIwd61fW26-nLHNkMKu1FicFK8wvl9JVxQ8KUbnrMVZl/s1600/NSCI+logo.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="71" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgX2kM6IynbcntZy6HcmoKbr1Vlnh91vb1mK4H-s9VEo1gPsqzGim5njbsTiP9WbTvE4nW7ClHQh3rk1N8W0ROM5-VL5DGlxKrwiIwd61fW26-nLHNkMKu1FicFK8wvl9JVxQ8KUbnrMVZl/s200/NSCI+logo.jpg" width="200" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">The Neural Stem Cell Institute </span><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">(NSCI) is home to some of the most interesting work in stem cell technology today. Its origin as a research hub for neural cell investigation lies with Sally's history and her pursuit and discovery of the first CNS stem cells in the mouse. As with a number of other leading scientists she started with uncovering complex neural biological systems and the mechanistic pathways of cell constructs of the CNS, which included the eye. She is the recipient of the <a href="https://www.macfound.org/fellows/813/" target="_blank">MacArthur "Genius" Award</a> and a highly respected leader in the field. </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">NSCI is funded by the </span><a href="http://stemcell.ny.gov/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">NY State NYSTEM</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> along with <a href="https://youtu.be/yG074T4YT-U" target="_blank">donations</a> as a non-profit and is based in upstate NY - near Albany, in a town called Rensselaer. It holds a </span><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2013330302A1&KC=A1%20%20&FT=D&ND=3&date=20131212&DB=EPODOC&locale=en_EP" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">foundational patent estate</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> to an adult stem cell discovery that now forms the lead translational focus of the institute - an adult retinal stem cell in the RPE layer which can be sourced from donor tissue and expanded to therapeutic doses. This same cell can also form a variety of other cell types via a biological trans-differentiation pathway called the </span><a href="https://en.wikipedia.org/wiki/Epithelial%E2%80%93mesenchymal_transition" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">EMT</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> into bone, fat and cartilage. The team has published a number of high profile papers (eg </span><a href="http://www.sciencedirect.com/science/article/pii/S1934590911005790" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">1</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">,</span><a href="http://www.ncbi.nlm.nih.gov/pubmed/23097100?dopt=Abstract" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">2</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">,</span><a href="http://www.cell.com/stem-cell-reports/abstract/S2213-6711(13)00130-6#Introduction" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">3</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">) on the science which underpin the clinical translation work. Their projects have many prestigious collaborators, including the </span><a href="http://www.kellogg.umich.edu/news/ar14/new-stem-cell-discoveries-point-to-treatment/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Kellogg Eye Center</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> and </span><a href="http://www.mountsinai.org/profiles/timothy-blenkinsop" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Mount Sinai</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">, amongst others.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"><br /></span><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">The eye is uniquely interconnected as a sensory organ, yet accessible, which has made it a natural target for NSCI to lead off with. The program is earmarked for a clinical trial in the not too distant future. RPE cell transplants are a hot cell therapy area. A number of groups are in clinical trials using various different sources and application methods using RPEs - notably </span><a href="http://msemporda.blogspot.com.es/p/ocata-eye.html" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Ocata</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">, </span><a href="http://msemporda.blogspot.com.es/p/rik-ip.html" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Riken</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">, </span><a href="http://msemporda.blogspot.com.es/p/univ-coll-london.html" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Coffey/Pfizer</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> & </span><a href="http://msemporda.blogspot.com.es/p/blog-page.html" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">BioTime/CellCure</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">. Some groups are also in the clinic using different retinal cells, while others still are in various pre-clinical stages of development. All this attention and focus on the eye is for a good reason - it's accessible and in-vivo activity can be observed in detail. However, most importantly the momentum is building as the data reported to-date is showing safety & potential efficacy. </span><br />
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">Sally's </span><a href="http://neuralsci.org/about-us/our-team" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">team</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> at NSCI includes her co-founder & partner </span><a href="http://www.capitalregionretina.com/about_us/about_us.htm" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Jeffrey Stern</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">, a retinal surgeon, and a notable listing of well respected scientists and researchers, including: </span><a href="http://neuralsci.org/about-us/our-team/chris-fasano-research-director-neural-stem-cell-institute" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Chris Fasano</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">, a leading member of the investigator team, who is also known for producing the official ISSCR </span><a href="http://stemcellpodcast.com/about" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Stem Cell Podcast</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> with </span><a href="https://www.linkedin.com/pub/yosif-ganat-ph-d/5/a91/b08" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Yosif Ganat</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">.</span><br />
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">The work at the institute is not solely eye cell centric, as you will see when exploring the various </span><a href="http://neuralsci.org/our-research" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">sub-sections of the research</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> going on there. The basic theme throughout is indeed neural and CNS in general. </span></div>
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<span style="font-family: inherit; font-size: x-small;">Stem Cell Podcast w/ Sally Temple</span><br />
<iframe frameborder="0" height="30" scrolling="no" src="http://stemcellpodcast.com/?powerpress_embed=403-podcast&powerpress_player=mediaelement-audio" width="320"></iframe> <span style="font-family: inherit; font-size: x-small;"><br /></span><span style="font-family: Arial; font-size: 14.6666666666667px; line-height: 1.38; text-align: justify; white-space: pre-wrap;"><br /></span><br />
<span style="font-family: Arial; font-size: 14.6666666666667px; line-height: 1.38; text-align: justify; white-space: pre-wrap;">The adult stem cell discovery that Sally, Jeff and collaborators at their NSCI uncovered has resonated throughout the community. It's simplicity is captivating and it's implications far reaching. The very nature of regeneration and the body's own capacity to heal itself is powerful stuff. That is what we all wish for, methods by which we can assist our own abilities in all manners throughout our lives - why not also with our own health. </span><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgsTwB_CWjUOq9bCVOP80WLCAR0aJR8_QoHXh6n-HGXDSQl80yQdNiC848z5U9VjzWN6NnctRG1mhvlmpjpi4yVRnWmec65rGyH9JkIyhgRq9j-aFHk7Fqd-_2CHzPym_l0g0XgVVhtaqbL/s1600/salamander.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="176" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgsTwB_CWjUOq9bCVOP80WLCAR0aJR8_QoHXh6n-HGXDSQl80yQdNiC848z5U9VjzWN6NnctRG1mhvlmpjpi4yVRnWmec65rGyH9JkIyhgRq9j-aFHk7Fqd-_2CHzPym_l0g0XgVVhtaqbL/s320/salamander.jpg" width="320" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">Yet, there is still a basic question to be resolved - if there are these cell populations in our organs & tissue, just waiting for those cues, can we indeed awaken our "Inner Salamander?" </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">I hope you find the interview transcript below informative. I have great admiration for innovators and no more so that those that fight for patient solutions in a not-for-profit foundation. </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"><br /></span><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">Good luck Sally, Jeff and all the team in my home State!</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">Cheers</span></div>
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<span style="color: black; font-family: Arial; vertical-align: baseline; white-space: pre-wrap;"><b><u>Interview</u></b></span><span style="color: black; font-family: Arial; vertical-align: baseline; white-space: pre-wrap;"> </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Can you explain your </span><a href="http://neuralsci.org/our-research/macular-degeneration-program" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">discovery of an adult retinal stem cell</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> and use of that for research and therapeutics.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"><br /></span><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - The idea for discovery research and looking for retinal cells that might have regenerative potential I have to attribute to my husband </span><a href="http://www.capitalregionretina.com/about_us/about_us.htm" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Jeff Stern</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">. He started in basic research and decided he wanted to work with people and went to medical school. He ended up coming back into the field of ophthalmology but with that research mindset. We of course talked over the years about neural stem cells and the discovery of tissues that you’d think don’t have regenerative potential but actually do.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Stimulated to have that potential?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - That’s the point. I firmly believe that we have that ability and if we can simulate it we can.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Somewhat like the salamander effect?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Exactly. So Jeff put in recently for the </span><a href="https://nei.nih.gov/audacious" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Audacious Goals competition of the National Eye Institute</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">. All the applications were anonymous so no one knew who submitted what and it was open to everyone, worldwide. They picked 10 winners and one of those was Jeff’s project. He was picked for “Reawakening your Inner Salamander” to take advantage of that.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - I like it. I used a salamander image a little while ago - it’s a poignant reference.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST . It is and of course the salamander RPE can regenerate and make the entire neural retina. So if you remove the photoreceptors and you remove the neural retina entirely in salamanders the RPE cells will change, proliferate and then make new retina cells.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Was this an area of study for you and your husband?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - We were aware of that because when Jeff worked in vision doing physiology, the physics of electrical physiology, he worked with salamanders and so he was very familiar with the regenerative literature and we thought let’s look in the human eye for a stem cell and if so could it be activated. We did experiments to establish these stem cells in the human system. We grew them in clones so we could watch an individual cell and see how many progeny it could make.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - From what source?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - We took it from human cadaver tissue and we removed the retina and took the RPE, which you could obtain very cleanly. We removed the anterior portion of the eye, which people have said may contain proliferative cells in perhaps a ciliary margin.</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgVfW8oc0xKZOtZE4uO9ue_O90cEG498I_b5XBQfTWK-Jmt_vJlKVjH4VAExsjcIkJlblw0ZiHrBwqxWrnGQIOhJYoTWmRIlmFH-GpHWJDibgAHz4QC7k4nHdqreVjETdS6EH5xxB81N1KY/s1600/tropepe.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgVfW8oc0xKZOtZE4uO9ue_O90cEG498I_b5XBQfTWK-Jmt_vJlKVjH4VAExsjcIkJlblw0ZiHrBwqxWrnGQIOhJYoTWmRIlmFH-GpHWJDibgAHz4QC7k4nHdqreVjETdS6EH5xxB81N1KY/s200/tropepe.jpg" width="169" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes, </span><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;"><a href="http://sites.utoronto.ca/neurobiology/retinal-sc.htm" style="text-decoration: none;" target="_blank">Derek van der Kooy</a></span><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> and <a href="http://csb.utoronto.ca/faculty/vincent-tropepe/" target="_blank">Vincent Tropepe</a>. In some animals there’s a ciliary margin but it’s not as clear where the ciliary margin is in humans but just in case we removed the anterior portion. We wanted to look within the RPE and we wanted to make sure we knew what cell type we were looking at. We cloned them and made movies of them. We took them from the eye and demonstrated that only a sub-population, less that 10% and in some preparations only 3% of the RPE cells will divide extensively.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - In-vivo people have found it very hard to see any proliferative cells but there are circumstances</span><span style="font-family: Arial; font-size: 14.6666666666667px; line-height: 1.38; white-space: pre-wrap;"> in which the RPE is thought to proliferate. Unfortunately under certain pathological circumstances you will see the RPE layer migrate through the retina and out into the vitreous and proliferate through creating these awful contractile membranes which will pull the retina off. That type of epiretinal membrane formation is quite common.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Almost like a mutated cell process.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - It’s like some of the cells are undergoing some of the transformative processes of the </span><a href="https://en.wikipedia.org/wiki/Epithelial%E2%80%93mesenchymal_transition" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">EMT</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> state. So we knew there were circumstances under which some of the RPE can proliferate in-vivo. Perhaps sometimes this can be beneficial. Maybe they could proliferate a little bit and help the retina recover from damage.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - They already do a lot of work.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes, the RPE are amazing. They’re such a humble little cell but if they die the retina dies. That’s how important they are. They are important for the blood retina barrier, fluid balance, cytokine protection, phagocytosis and more. So we found a sub-population of the cells will self-renew extensively making hundreds and thousands of cell progeny from one cell.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - To recap there are adult retina cells that can proliferate as evidenced by the EMT phenomena and that if stimulated can be a source of retinal tissue</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Exactly, so the idea is there’s a sub-population that can be activated to proliferate. If those are the cells that contribute to those abnormal masses we don’t know for sure but what we do know is that the cells can proliferate. We can take a single cell and make numerous progeny. We can split those prodigy up so now we have clones of those originals that you can then put in different media.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - More so than what was achieved with fetal cells?</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjZ8xvjaGMSpg-MsvRpw0gexHvj-ZZABtzHhrK65gtmdXiSsLIeZeH485MqsCrDBKV1FG1biZLX9vrkM31jnm73VOzNl4VpV4LI5g6L0ldePLAIxwVg2-PdNZ6TuI9XhgOezT9UQm9t3zd9/s1600/RPESC+transdif.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="205" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjZ8xvjaGMSpg-MsvRpw0gexHvj-ZZABtzHhrK65gtmdXiSsLIeZeH485MqsCrDBKV1FG1biZLX9vrkM31jnm73VOzNl4VpV4LI5g6L0ldePLAIxwVg2-PdNZ6TuI9XhgOezT9UQm9t3zd9/s320/RPESC+transdif.jpg" width="320" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - I don’t know if they cloned a single fetal cell. These are adult cells that we cloned out. From a single cell we get a clone and put it in different media conditions and we have shown that the same cell that can give RPE can also produce fat, cartilage and bone.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes so RPE can make MSCs and can undergo EMT. We think that could be a good model for the epiretinal formation. We don’t know if it’s the originating cell in-vivo but it can do that. It was a surprise because you wouldn’t have thought of a CNS cell giving rise to MSC progeny.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - I’ve spoken with </span><a href="http://www.uic.es/en/teacher/al-atari-abou-asi-maher" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Dr. Maher in Barcelona</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> who is pioneering a lot of work on wisdom teeth and he says the same thing in reverse. They can make neural crest cells and other non-MSC cell types, plus the MSC lineages . Does this have to do with the CNS connection also?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - The CNS is the brain, the retina and the spinal cord. The neural crest of course is from the dorsal part of the neural tube and its migratory. The cranial neural crest does have progenitors that give rise to bone and cartilage etc. A mixture of cells, as well as neural cells. We know that our cells are CNS cells at the beginning and are probably not going through the neural crest stage. They don’t seem to make sensory neurons and sympathetic neurons etc. We don’t think of changing RPE into neural crest. For some reason the RPE has retained the potential to make MSCs for whatever reason. I can’t explain but that is the case.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Have you done genetics on that?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes we’re in the process of doing that now and studying this progression into MSCs because we want to understand that so we can prevent, but that happens pathologically. At the same time we know we can take those cells and make beautifully stable RPEs for 2 years in culture.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - and make a lot of them</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh1aIohkxnJ6S61Isdfg6rtfKWogR_3Mak-JLrCRjP27It5mbLjViAqK9dBU0hY5orYM3re6JG2nFHaFByS1CYYdMMVUeaDYsbjMiWtGaFyVu7ePiH4kTO7SkLYRBYVf29SrpkmG6qANndw/s1600/Paper+RPESC+to+MSCs.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh1aIohkxnJ6S61Isdfg6rtfKWogR_3Mak-JLrCRjP27It5mbLjViAqK9dBU0hY5orYM3re6JG2nFHaFByS1CYYdMMVUeaDYsbjMiWtGaFyVu7ePiH4kTO7SkLYRBYVf29SrpkmG6qANndw/s200/Paper+RPESC+to+MSCs.jpg" width="181" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes a lot of them. From one donor we can make 5 x 10⁸ cells which is a lot. Let’s say a patient age-related macular degeneration may require 50,000 or 100,000, because you’re only covering that tiny macular region, we hoping one donor’s cells will be able to treat hundreds of patients. We have made plans for all the moving parts. You have to get manufacturing and regulatory to approve so we’re not doing it ourselves. We’re using a facility and transferring the technology so we know they are making the highest quality cells.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes it is. An academic GMP facility at the </span><a href="https://www.rochester.edu/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">University of Rochester</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> and they’ve been wonderful. We do a lot of back and forth to make sure the cells are correct.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - This is sort of a NY project?</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgVQNyo7pvw4uaBFij_sVAl1Qr586JHTEEybe73RewkBnlNUFHOWGn6uVgISD6ryh3PGAbZEVaq_TKQ4kCzafBeEUj2PaTowrTxUyc9O8uQwm8DvZ40FjAlZ4ic5o-RP8NAciaSD-yb2sL4/s1600/NYSTEM.jpg" imageanchor="1" style="clear: left; float: left; font-size: 14.6666669845581px; line-height: 20.2399997711182px; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" height="182" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgVQNyo7pvw4uaBFij_sVAl1Qr586JHTEEybe73RewkBnlNUFHOWGn6uVgISD6ryh3PGAbZEVaq_TKQ4kCzafBeEUj2PaTowrTxUyc9O8uQwm8DvZ40FjAlZ4ic5o-RP8NAciaSD-yb2sL4/s200/NYSTEM.jpg" width="200" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - It is mostly. It’s funded through the NY State via the </span><a href="http://nystem.com/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">NYSTEM</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> program. We wouldn’t have been able to do this without them. They have been tremendous. It’s very expensive. We have got to solve this problem of why it costs so much to do this. They gave us $10.8m over 4 years to do all the preparations for the manufacturing and the efficacy to get to an IND. What we’re hoping once we get through that process is that we can then move into clinical trials.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - If you can show there’s a signal. I’m not sure if you’ll need to go through a small trial to get to that stage.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Probably a Phase 1. We’re planning about 18 patients. It’s very interesting to be in an area like this. In the beginning people weren’t talking very much about the RPE and then there was a recognition that this would be a great target tissue because it’s the eye and you can actually watch what’s happening once you put the cells in. There are sensitive visual tests.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Have you added all those specialists to the team now?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes. Jeff of course is a retinal surgeon.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - We think it’s important to be hands off with the safety study, so it will be done independently. That way we have some comfort. We’ll know the cells are safe and there will be no conflict. </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Once you publish you can receive credit. </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - The efficacy data to-date is really exciting.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Using the RCS rats?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes, the RCS rat. You know people have said with that model anything works. This is not true.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - You can look at the past examples</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - We haven’t published yet but what we have shown is that the cells have to be at a particular stage of development.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - That’s what I’ve been talking about before and with the community here.</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiAa9MGyT7Pc9BgbqHXNyahqK_3stbOz8OseBdauE-JU6BHVTml_gpxe2nITuHe_O7inNnMW-tAFRO74Z8yFpxNicC_IAlH_hViJQUs0L5YIKb6pjUGfAOzF05tlr7oyK9W2Vo_qHbfeE34/s1600/ali.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="141" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiAa9MGyT7Pc9BgbqHXNyahqK_3stbOz8OseBdauE-JU6BHVTml_gpxe2nITuHe_O7inNnMW-tAFRO74Z8yFpxNicC_IAlH_hViJQUs0L5YIKb6pjUGfAOzF05tlr7oyK9W2Vo_qHbfeE34/s200/ali.jpg" width="200" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - </span><a href="http://www.ucl.ac.uk/ioo/genetics/gene-and-cell-therapy/research-team/staff-iris-profiles/robin-ali" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Robin Ali</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Yes that’s right Robin Ali’s work and clinically with </span><a href="https://www.ocata.com/clinical/ongoing-clinical-trials" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Dr. Lanza’s trials</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> having used cells differentiated to a certain point.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - There’s a sweet spot in the developmental profile - the earliest proliferating cells and the latest mature cells don’t work as well as cells in the middle of the process.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Yes. It’s important to get there as efficiently as possible, extract, freeze & thaw?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes. That sweet spot was a surprise so we’re lining up all these elements to use the cells. I do feel good now having different groups using different sources. Some using iPS, some using ES some putting them on a scaffold and some injecting a suspension - like we plan to do.</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgdi5LLm2wP5eT3Zohfn9PWKbsrLAkhXv4O3dpybOH9-egmCtCLl2YDeK4T9cUN_mHUjpDTfHs1d87DfF_0q1l-BeIljC2OhyphenhyphenV3D6T10QRmF5bG6IDX6GgIvOyPR7ZVr8gfkqfyqQgp92U4/s1600/Masayo+Takahashi+-+Asahi+Shimbun+file+photo.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgdi5LLm2wP5eT3Zohfn9PWKbsrLAkhXv4O3dpybOH9-egmCtCLl2YDeK4T9cUN_mHUjpDTfHs1d87DfF_0q1l-BeIljC2OhyphenhyphenV3D6T10QRmF5bG6IDX6GgIvOyPR7ZVr8gfkqfyqQgp92U4/s200/Masayo+Takahashi+-+Asahi+Shimbun+file+photo.jpg" width="147" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - I </span><a href="http://msemporda.blogspot.com.es/p/masayo-takahashi-hope-yes-patients-first.html" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">spoke with Masayo Takahashi</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> the other day, she’s wonderful, and she was explaining how excited her team was about the progressional steps they’re taking from monolayer to suspension. There’s an acknowledgement that there’s a need for suspension in certain cases.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Oh good. I’m glad she’s doing that.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - That was very important to hear as I felt there was the advanced stage but there are of course other stages. Robin Ali felt that photoreceptors need to come into play more and importantly so as to restore function & vision. That’s certainly true depending on the disease, state of the eye and point in time. The patient acceptance of surgery along that progression is vital to understand because if you’re looking at 20/40 or 20/80 you're going to have a different opinion than if you were 20/200+ so there is an issue there imo.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - So Jeff, if he was here, he would say to you “I’m a retinal surgeon and if there was a non-surgical solution I would prefer it.” That’s why we’re excited by the cell we’ve identified as it’s in our eyes. The RPE is so neat, it’s actually laid down in the embryo so when we look at the eye it’s the black center. Those cells were done when you were in utero and really don’t proliferate very much. So we think that’s one of the reasons we’ve been able to activate them from even a 99 year old. They haven’t been used up. There’s no hayflick limit as they haven’t been dividing and dividing and exhausted. They have preserved their potential to divide. We take them out and put them in culture. These cells from 99 year olds that have not divided for a century will start to divide in 36 hours.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Source therefore is not that big an issue for you. Is it the standardization in the manufacturing area that will be a challenge?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Not really, cadaver eyes are readily available because they are already collected for corneal transplants</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - The donor consent forms are already there.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes. People are so wonderful in their generosity because these are light & vision saving possibilities. So the cornea is already taken, we take the part that is generally thrown away and utilize that. They’re available and they’re in us. So if they’re there and we could activate them safely for an endogenous repair that would be the goal.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Have you seen the BMP4 inhibition study from Derek van der Kooy’s team? He was trying to do something similar. Evidentially there was some form of stop in his cells also and they’re working to find some chemical formula to regulate inhibition but you have to be very specific otherwise they show off target effects.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - The RPE cells in-vivo don’t divide very much, if at all, the question is whether this is because of inhibition or the lack of activators. So what we found is we can take growth factors that stimulate the growth in-vitro and put those in the eye of animals and they do activate the cells. We think lack of activators is probably one of the reasons and we can add these. At the same time it’s possible that if you add in something that Derek is describing you get even more activation but I think you have to be very careful. You don’t want too much activation because there would be a concern there you could get a growth.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - </span><a href="http://jcyte.com/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">jCyte</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> are doing some interesting work. They’re looking at delivering the factors by way of cells intravitreally.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes we’re looking to actually isolate the factors</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - CIRM has funded their program. Henry Klassen is moving it and they’ve got an </span><a href="http://news.uci.edu/press-releases/fda-greenlights-uci-clinical-trial-of-treatment-for-blinding-disease/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">approved IND</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> for RP [since this interview clinical trial has started - see <a href="http://jcyte.com/2015/08/cirm-press-release-august-2015/" target="_blank">here</a>].</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes he’s putting cells in to protect</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - He experimented on different formats and settled on the cells as factor delivery vehicles as his approach first</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Good idea</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - There are other possibilities for action via MSCs intravitreally or systemically plus via some of the recent work on neuroprotection using photoreceptor progenitor factors. Is that something similar in how it may work?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Perhaps. I know that Jensen are putting umbilical cord cells under the retina </span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - They haven’t been too revealing in terms of data</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Right, that’s something I would say is so helpful if we’re working in the same area to share as much as possible because we learn from each other. I don’t know what they’re doing and it’s a highly sensitive area of the body, so safety is a key issue. So to have to inject subretinally once may be ok, but to have to do it repeatedly is a concern. Certainly if you could do an intravitreal injection of an activating factor that has great appeal.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Is that where you’d like to get to</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes. Our animal studies in that area are progressing. We think we have a pipeline developing</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - So the first would be the transplant then during that phase you would develop the concept further?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes the endogenous stimulation that’s exactly what we’re doing</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - That’s a plan that will be successful imo - the degree of success is yet to be seen of course but it’s worth every effort.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Of course</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - I noticed you were working with the Israelis on an element of the protocols using </span><a href="https://en.wikipedia.org/wiki/Nicotinamide" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">NIC</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> expansion. Are you utilizing some of that methodology?</span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEi37E9LL-KTqOGjTiZy2JWkkzwy9uhnpkVKG2k4B8qg4Iwpe8uQ1xXKZGn1EeuvkeXl-YpNL7iX_8CMFXWQdesb04rRE4sqxFJkTyhpKKv-Dfye42HN-l2bfOkPiZ9N5p1Y0ifYwyE3-V9A/s1600/Reubinoff-150x150.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEi37E9LL-KTqOGjTiZy2JWkkzwy9uhnpkVKG2k4B8qg4Iwpe8uQ1xXKZGn1EeuvkeXl-YpNL7iX_8CMFXWQdesb04rRE4sqxFJkTyhpKKv-Dfye42HN-l2bfOkPiZ9N5p1Y0ifYwyE3-V9A/s1600/Reubinoff-150x150.jpg" /></a></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiL1WgRJZ4CigIX-l141G6_pko83Op9CTIPdUh-5wBvbqbY5xPdBxsV1S4fxiiSCNhv8_CEW8saAnHMNSoQgJJXBI1nAUwDI_gJBfbFy1KMyx3_QNgt86g7o5n3R-VwJHzbFzEBWaxxU65h/s1600/PROFESSOR-EYAL-BANIN_v2-150x150.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiL1WgRJZ4CigIX-l141G6_pko83Op9CTIPdUh-5wBvbqbY5xPdBxsV1S4fxiiSCNhv8_CEW8saAnHMNSoQgJJXBI1nAUwDI_gJBfbFy1KMyx3_QNgt86g7o5n3R-VwJHzbFzEBWaxxU65h/s1600/PROFESSOR-EYAL-BANIN_v2-150x150.jpg" /></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - We also grow iPS cells and I think you’re referring to </span><a href="http://www.cellcureneurosciences.com/company/executive-management/" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Eyal Banin and Benjamin Rubinoff</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;"> and the use of NIC. We find it is beneficial yes.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - It helps with proliferation or how?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Not quite sure what it does to the cells but it helps the differentiation of the cells and they look robust.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Do they over mature as a result?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - I don’t think so</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Ok there’s a sweet spot issue that’s important</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Yes so that is the case for our adult cells, which I mentioned we’re preparing a paper on.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - When’s that due out?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Oh yes, I think Richard Davis, who’s working with us on that, would say “ah, the figures are almost all done, writing it up”</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - In the post!</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - I know, it’s in the post! I’ll will let you know when it’s ready.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Thx, would love to read it. I think we’ve covered so much, just a few more notes here. The Allo source, is there a need for immunosuppressants - systemic or local dose? How will that work?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - I have to say when doing a clinical trial you have less leeway than you’d imagine on these different details. We’ve been encouraged to do a very strong immunosuppression early on and part of our clinical team includes physicians that specialize in immune issues in the eye. A uveitis specialist. We recruited a very prominent scientist at Mount Sinai, his name is </span><a href="http://www.mountsinai.org/profiles/douglas-jabs" style="text-decoration: none;" target="_blank"><span style="color: #1155cc; font-family: Arial; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Douglas Jabs</span></a><span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">. So we’re aware of that. Because the RPE is the blood retina barrier if it is diseased you could get it broken down. So we’re starting with Allo and the tissue is prevalent and available enough that we could HLA match to help reduce the immune issue. Then, given the cells are already in the eye, if the activation product doesn’t work we could probably do an extraction & expansion for an autologous transplant of cells.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Would you do an iPS or a retina stem cell sample from the eye?</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - We’d probably take it directly from the eye</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - Because a skin biopsy or blood sample is easier</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - It is. We also know we can take these cells from the subretinal fluid. They’re there and it’s probably just going into the subretinal space and sucking some out. The reason we know this is that in certain circumstances they have to take out fluid such as in a retinal detachment and normally that is thrown away. We have a protocol to grow cells from that. It’s small scale and it doesn’t work every time but we’re not actively trying to harvest the cells right now, but in theory it could be done.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">M - I’m very happy for you and your team - it’s great work. At the end of the day fighting hard for the future solutions is really all about the next generation and what we can do for our own loved ones who are older. If we can stop this evil circle it will be worth it and try to do it in a way that makes it economically viable. Thank you so much Sally.</span></div>
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<span style="color: black; font-family: Arial; font-size: 14.6666666666667px; vertical-align: baseline; white-space: pre-wrap;">ST - Thank you.</span></div>
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<span style="font-family: Arial;"><span style="font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;">Refs: </span></span></div>
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<ul>
<li><a href="http://www.sciencedirect.com/science/article/pii/S1934590911005790" style="font-family: Arial; font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;" target="_blank"><span style="color: black;">Adult Human RPE Can Be Activated into a Multipotent Stem Cell that Produces Mesenchymal Derivatives</span></a></li>
<li><a href="http://www.sciencedirect.com/science/article/pii/S1934590911005959" style="font-family: Arial; font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;" target="_blank"><span style="color: black;">Untapping the Potential of Human Retinal Pigmented Epithelial Cells</span></a></li>
<li><a href="http://www.ncbi.nlm.nih.gov/pubmed/23097100?dopt=Abstract" style="font-family: Arial; font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;" target="_blank"><span style="color: black;">The culture and maintenance of functional retinal pigment epithelial monolayers from adult human eye</span></a></li>
<li><a href="http://www.cell.com/stem-cell-reports/abstract/S2213-6711(13)00130-6#Introduction" target="_blank"><span style="color: black;"><span style="font-family: Arial;"><span style="font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;">Human RPE Stem Cells Grown into Polarized RPE Monolayers on a Polyester Matrix Are </span></span><span style="font-family: Arial; font-size: 14.6666669845581px; line-height: 20.2399997711182px; white-space: pre-wrap;">Maintained after Grafting into Rabbit Subretinal Space</span></span></a></li>
<li><span style="font-family: Arial; font-size: 14.6666666666667px; line-height: 1.38; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;"><a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=8481313.PN.&OS=PN/8481313&RS=PN/8481313" style="font-size: 14.6666666666667px; line-height: 1.38; text-decoration: none;" target="_blank">RPESCs Patent - US Grant July 2013</a></span></li>
<li><span style="font-family: Arial; font-size: 14.6666666666667px; line-height: 1.38; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;"><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2013330302A1&KC=A1%20%20&FT=D&ND=3&date=20131212&DB=EPODOC&locale=en_EP" style="font-size: 14.6666666666667px; line-height: 1.38; text-decoration: none;" target="_blank">RPESC Patent Family - EspaceNet</a></span></li>
<li><a href="http://worldwide.espacenet.com/searchResults?submitted=true&locale=en_EP&DB=EPODOC&ST=advanced&TI%20%20=&AB=&PN=&AP=&PR=&PD=&PA=&IN=sally+temple&CPC=&IC=&Submit=Search" style="font-family: Arial; font-size: 14.6666666666667px; text-decoration: none;" target="_blank"><span style="color: black; font-size: 14.6666666666667px; text-decoration: underline; vertical-align: baseline; white-space: pre-wrap;">Related Program IP</span></a></li>
</ul>
<br />@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-89480339021263935652015-07-31T02:01:00.000-07:002015-08-25T05:19:27.805-07:00ISSCR 2015 Annual Meeting Coverage<div class="separator" style="clear: both; text-align: center;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiFe0iKJVwPXpmdI5wDbtX_OuaxBoEEVfNaQEkwuxyfqG2T4vgX2PIWieDIns-Ne6N17n2iNpalNNqHjNx2tfpoaf4MVYqvmKfqYGr9KO719kp-2ZbmL9GMhh5nJ6hK9JL1Vd-gDeCzqWMY/s1600/ISSCR-2015-Banner.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" height="191" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiFe0iKJVwPXpmdI5wDbtX_OuaxBoEEVfNaQEkwuxyfqG2T4vgX2PIWieDIns-Ne6N17n2iNpalNNqHjNx2tfpoaf4MVYqvmKfqYGr9KO719kp-2ZbmL9GMhh5nJ6hK9JL1Vd-gDeCzqWMY/s400/ISSCR-2015-Banner.jpg" width="400" /></a></div>
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<span style="text-align: justify;">I recently attended the annual meeting of the </span><a href="http://www.isscr.org/" style="text-align: justify;" target="_blank">International Society for Stem Cell Research</a> and<span style="text-align: justify;"> have been writing up the coverage in dedicated articles which you will find the links to below and also to the right.</span></div>
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<span style="text-align: justify;">The interview with Dr. Sally Temple entitled <b style="font-style: italic;">"</b></span><i><b>Cells, Leadership & Audacious Innovation"</b></i> can be found <a href="http://msemporda.blogspot.com.es/p/there-is-saying-that-you-save-best-for.html" target="_blank">here</a>.</div>
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I wish to thank the ISSCR for the opportunity and to Michelle Quivey, Snr Comms Mgr, for her professional handling of the media scheduling.</div>
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Cheers<br />
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<a href="http://msemporda.blogspot.com.es/p/isscr-2015-introduction.html" target="_blank">ISSCR 2015 Introduction</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgclfl069RYIw06xtmlKtO4ZEbOA8jKSsqJWxVbJZyV8PhiUYQu2H3Wd3ubq03PpbfOId05q-zl0am63DCjrEplUIfADgz5nQdxkEqVHHu4UYhA3LyMA6udAFPm5-RC-4QTwqgdOnLgJWM-/s1600/ISSCR-2015-Intro.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="197" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgclfl069RYIw06xtmlKtO4ZEbOA8jKSsqJWxVbJZyV8PhiUYQu2H3Wd3ubq03PpbfOId05q-zl0am63DCjrEplUIfADgz5nQdxkEqVHHu4UYhA3LyMA6udAFPm5-RC-4QTwqgdOnLgJWM-/s200/ISSCR-2015-Intro.jpg" width="200" /></a></div>
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<a href="http://msemporda.blogspot.com.es/p/stem-cells-aging-brain-public-symposium.html" target="_blank">Stem Cells & The Aging Brain - Karolinska Symposium</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjVVblTI4TQ17DagTu9uGK0xBa7ak6W8L8UF1R_EjRuABCkbQTD6gH7ubqFa_ZCRfvIdPVHm_2-O-GORgGbqM-UtYTG7_mnxZgrQbK2z8QV9fRvXhUUfJbO-o9cYkXoy12NADI7hMBEk4-y/s1600/ISSCR-2015-brain.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em; text-align: center;"><img border="0" height="198" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjVVblTI4TQ17DagTu9uGK0xBa7ak6W8L8UF1R_EjRuABCkbQTD6gH7ubqFa_ZCRfvIdPVHm_2-O-GORgGbqM-UtYTG7_mnxZgrQbK2z8QV9fRvXhUUfJbO-o9cYkXoy12NADI7hMBEk4-y/s200/ISSCR-2015-brain.jpg" width="200" /></a><br />
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<a href="http://msemporda.blogspot.com.es/p/biotech-spin-outs-discovery-company.html" target="_blank">Biotech Spin-Outs: Discovery > Company</a><br />
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<a href="http://msemporda.blogspot.com.es/p/media-hype-esi-bio-cirms-chairman.html" target="_blank">Media Hype | ESi Bio | CIRM's Chairman</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjUsFsKgV9Ay9Rl6Q25v2TKBYStNkT10JopBNPv5c6BgPZGcWY8YvHETKTp-RlxsNpK4JyWNhY651ngRK-q6RlQ-8UdSALdcijaipfIXzJ-LpAX3FZB3zkcneGXcSawCyfs8Gr9jDrJo_gJ/s1600/ISSCR-2015-media-hype.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em; text-align: center;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjUsFsKgV9Ay9Rl6Q25v2TKBYStNkT10JopBNPv5c6BgPZGcWY8YvHETKTp-RlxsNpK4JyWNhY651ngRK-q6RlQ-8UdSALdcijaipfIXzJ-LpAX3FZB3zkcneGXcSawCyfs8Gr9jDrJo_gJ/s200/ISSCR-2015-media-hype.jpg" width="194" /></a><br />
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<a href="http://msemporda.blogspot.com.es/p/masayo-takahashi-hope-yes-patients-first.html" target="_blank">Masayo Takahashi - "Hope - Yes" & "Patients First"</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhkSA60T1YU7hDUdgWQkPp_PhaAlzKHMvWCD3j5fSijUjJgzMjvnFjjvHsvG7YKj2Cuic4TbJCjg1JHMgJJvsjwVNkLb82whwReb6qy6g0hbPVryplLmRHY8GlMDPT1nNqQ-Y3LV69TVYV7/s1600/ISSCR-2015-Masayo.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" height="196" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhkSA60T1YU7hDUdgWQkPp_PhaAlzKHMvWCD3j5fSijUjJgzMjvnFjjvHsvG7YKj2Cuic4TbJCjg1JHMgJJvsjwVNkLb82whwReb6qy6g0hbPVryplLmRHY8GlMDPT1nNqQ-Y3LV69TVYV7/s200/ISSCR-2015-Masayo.jpg" width="200" /></a><br />
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<a href="http://msemporda.blogspot.com.es/p/gdf11-adult-mscs-ms-nyscf-mitont.html" target="_blank">GDF11 | Adult MSCs > MS | NYSCF Mito/NT</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjTq8Z-lWBiXcAQ6U0Um9mgOS2PDPKTWqqH8JpA1lc8FvMiDh9GeVIhhcgEwd6CV4NZf1DFFopLqKoM2LAuh-L-vgrKX3IM2f_XDlYzjOOSkniTezgvncj7P-7NpyTz-6KSujpHItNmBFcG/s1600/ISSCR-2015-GDF11.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em; text-align: center;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjTq8Z-lWBiXcAQ6U0Um9mgOS2PDPKTWqqH8JpA1lc8FvMiDh9GeVIhhcgEwd6CV4NZf1DFFopLqKoM2LAuh-L-vgrKX3IM2f_XDlYzjOOSkniTezgvncj7P-7NpyTz-6KSujpHItNmBFcG/s200/ISSCR-2015-GDF11.jpg" width="193" /></a><br />
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<a href="http://msemporda.blogspot.com.es/p/the-pluripotency-trilogy.html" target="_blank">The Pluripotency Trilogy</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjE7DrqBmsD3EKsmXgnwVva4Bo9CKU35FfB_t9ewHf4TYAKdSxw75R_2aIMaGRi9lg24qqPdhX3zaP63bh6G_zDSr3bhRiHnM2FuqOeCepfcMujy8kjKBcQkPcURXMwOYqv7lciwbvejeXo/s1600/ISSCR-2015-Pluripotency.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjE7DrqBmsD3EKsmXgnwVva4Bo9CKU35FfB_t9ewHf4TYAKdSxw75R_2aIMaGRi9lg24qqPdhX3zaP63bh6G_zDSr3bhRiHnM2FuqOeCepfcMujy8kjKBcQkPcURXMwOYqv7lciwbvejeXo/s200/ISSCR-2015-Pluripotency.jpg" width="194" /></a><br />
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<a href="http://msemporda.blogspot.com.es/p/jeanne-loring-of-scripps-research.html" target="_blank">Jeanne Loring - Parkinson's, Mice, DNA, hPSCs & Rhinos</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiG7by_pz4pH_ujnWK4HNV8wGk67BSfh2cZJiXquyWCgb0igGUTMKOMAirU2xy7LPPi6tKEmG4PrBxworRBsBUuDRENMQeMYBi1A1IfG7oQ8f5UyWlOmy7S3g7CqreJvhTTCvQFzde0_vt6/s1600/ISSCR-2015-Loring.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em; text-align: center;"><img border="0" height="194" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiG7by_pz4pH_ujnWK4HNV8wGk67BSfh2cZJiXquyWCgb0igGUTMKOMAirU2xy7LPPi6tKEmG4PrBxworRBsBUuDRENMQeMYBi1A1IfG7oQ8f5UyWlOmy7S3g7CqreJvhTTCvQFzde0_vt6/s200/ISSCR-2015-Loring.jpg" width="200" /></a><br />
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<a href="http://msemporda.blogspot.com.es/p/utility-by-design-bio-engineered.html" target="_blank">Utility by Design - Bio-Engineered MedTech Devices</a><br />
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgvUcO_n-P3lho36PI3qMAvTEWq2O3KO6vQ40oR0cy7XWk3M4oDIDagg3s9QYWCHu8RyHPOV-gR1a5yP6MtLwCjy9L8ssI0_F2zEWvbR-akQ8iPmAVl8frmV0-Uoweod6oP0DBIm0IQMfaA/s1600/Utility-by-Design.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" height="198" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgvUcO_n-P3lho36PI3qMAvTEWq2O3KO6vQ40oR0cy7XWk3M4oDIDagg3s9QYWCHu8RyHPOV-gR1a5yP6MtLwCjy9L8ssI0_F2zEWvbR-akQ8iPmAVl8frmV0-Uoweod6oP0DBIm0IQMfaA/s200/Utility-by-Design.jpg" width="200" /></a><br />
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-62875517178962183192015-07-01T05:13:00.000-07:002015-07-24T03:30:10.839-07:00Congressional Debate Heats Up on Germ Line Editing<div class="separator" style="clear: both; text-align: center;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhstfXLKBnLtHfxqx1D_9p1KkbfirIOpOb8ytfFaXL97MaOAVjJ1ICMj8AlSnBtxfcJUTBeM77w4mPFkkPml0PqN0pjjSF_PGuj-iNUCeANmqvxMNGZBkFjzXbjuEhf6JU0yC0lkMU0CxiS/s1600/us+reps.jpg" imageanchor="1" style="clear: left; display: inline !important; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhstfXLKBnLtHfxqx1D_9p1KkbfirIOpOb8ytfFaXL97MaOAVjJ1ICMj8AlSnBtxfcJUTBeM77w4mPFkkPml0PqN0pjjSF_PGuj-iNUCeANmqvxMNGZBkFjzXbjuEhf6JU0yC0lkMU0CxiS/s1600/us+reps.jpg" /></a></div>
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Recently a <a href="http://www.nature.com/news/us-congress-moves-to-block-human-embryo-editing-1.17858" target="_blank">story by Sara Reardon</a> for Nature broke that the US House of Representatives had introduced as part of the 2016 spending package a Bill that would seek to add restrictions on top of already existing regulatory provisions and guidelines in respect to human genetic editing of embryos, sperm or eggs (germ line cells).</div>
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The idea would be to restrict access to federal funds to evaluate or permit proposed research applications in this area. This would in effect stall any positive progress in the US in respect to basic biology questions in regard to potential solutions for inherited genetic disorders. Mitochondrial technologies that are being considered would for example be effected by this ban.</div>
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In addition, the language in the House Appropriations Committee's Bill would look to establish an “an independent panel of experts, including those from faith-based institutions with expertise on bioethics and faith-based medical associations” to review recommendations from federal advisory institutions, such as the US Institute of Medicine (IOM), with regard to such technologies and their use.</div>
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Non-viable embryos would be notably allowable under these new proposed rules for research purposes.</div>
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The US National Academy of Sciences (NAS) who oversees the IOM are due to conduct a review of the human gene editing area later this fall, as noted in the video below and detailed in the NAS/NAM press release <a href="http://www8.nationalacademies.org/onpinews/newsitem.aspx?RecordID=05182015" target="_blank">here</a>.</div>
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As a few have pointed out the involvement of "faith-based institutions" doesn't seem to warrant concern as the topic is for the entire community to discuss and participation from all members is recommended. Consensus can only be legitimate if all voices have had equal representation in the discussions.</div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEj8X5Lwy5PicMtKSa47A_bZcycli05ZhGMzm1rWj_eg9gZPH1hwTBQNLpFnQeqSKOZYBoY3IMrp5-Hmexc9eZx49OnNFSosiqoW0XkxMDKzwTgixG4-cV3y__51TmeD8sNGWp18WUf_kXGC/s1600/we+the+people.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: justify;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEj8X5Lwy5PicMtKSa47A_bZcycli05ZhGMzm1rWj_eg9gZPH1hwTBQNLpFnQeqSKOZYBoY3IMrp5-Hmexc9eZx49OnNFSosiqoW0XkxMDKzwTgixG4-cV3y__51TmeD8sNGWp18WUf_kXGC/s1600/we+the+people.jpg" /></a><br />
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However, this I believe is more to the point. Where does it state that democracy first establishes law then allows inclusive debate? This initiative seems on the surface to be more of the same rather than a genuine effort to collaborate on an effective and universally applicable set of guidelines for legislative consideration in all international jurisdictions. </div>
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Cheers</div>
@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-16926410543576363692015-06-20T03:47:00.001-07:002015-06-20T03:49:07.580-07:00The Science and Ethics of Genetically Engineered Human DNA - Congressional Hearing<div class="separator" style="clear: both; text-align: left;">
<span style="font-family: Arial, Helvetica, sans-serif; font-size: x-small;">Backstory and current state-of-play review in a formal setting w/ Q&A - </span><span style="font-family: Arial, Helvetica, sans-serif; font-size: x-small;">Via Paul Knoepfler's Blog post: "</span><span style="font-family: Arial, Helvetica, sans-serif; font-size: x-small;"><i><a href="http://www.ipscell.com/2015/06/doudnacongress/" target="_blank">Doudna & Others Testify Before Congress on CRISPR, Human Germline Editing</a></i>"</span></div>
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<br />@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-8472093205711782632015-05-11T03:49:00.003-07:002015-06-06T05:45:06.452-07:00Germline Science & Embryo Use - The Law & Scope for Applied Research <div dir="ltr" style="margin-bottom: 0pt; margin-top: 0pt; text-align: justify;">
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<tr><td style="text-align: center;"><span style="font-family: Arial, Helvetica, sans-serif;"><img border="0" height="211" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiOw0sKovPRckoUVoN8FoJG5Nf_RUSZwObdOuPgoKEKk9EpsRFrGKkl2DqQslzjrDOVAasS9nf5RkobUv6bzTm681IY_7TXLC9V6htpop3ww_gEAS_GoVaMfwm4OQWXGus_CxR1c5qwYMk1/s320/statment_gene_editing-tech.jpg" style="margin-left: auto; margin-right: auto;" width="320" /></span></td></tr>
<tr><td class="tr-caption" style="text-align: center;"><span style="font-family: Arial, Helvetica, sans-serif; font-size: xx-small;"><a href="http://www.nih.gov/about/director/04292015_statement_gene_editing_technologies.htm" target="_blank">NIH statement on editing human embryo DNA</a></span></td></tr>
</tbody></table>
<div style="clear: left; float: left; line-height: 1.38; margin-bottom: 1em; margin-right: 1em; text-align: left;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span id="goog_953950736"></span><span id="goog_953950737"></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span id="goog_2046138721"></span><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The </span><a href="http://www.nih.gov/about/director/04292015_statement_gene_editing_technologies.htm" target="_blank"><span lang="EN-GB" style="color: #0b5394; font-family: Arial; mso-ansi-language: EN-GB;">recent reiteration by the Director</span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"> of
the NIH, Dr. Francis Collins, that the long held legal position of the US
Federal Government is to </span><a href="http://stemcells.nih.gov/policy/pages/2009guidelines.aspx" target="_blank"><span lang="EN-GB" style="color: #0b5394; font-family: Arial; mso-ansi-language: EN-GB;">not fund destructive embryo research</span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">,
brings the US debate on germ line editing front & center in practical
terms.</span></span><br />
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"><br /></span></span>
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"><br /></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgFL5o8CU4vfDnAVzqXfCltHfS0AfWdTjmBP3Wz9eMZhY00c7G2A6zsD190iL0oYliN-uRRrmAKyT7biYUzIG4XUbK9DQq13Wjbye6_WlyNMZ4q-YXgWFtIw8KePHq1tcadSDeheO5Mq-uo/s1600/epo.png" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><span id="goog_294558775"></span><img border="0" height="100" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgFL5o8CU4vfDnAVzqXfCltHfS0AfWdTjmBP3Wz9eMZhY00c7G2A6zsD190iL0oYliN-uRRrmAKyT7biYUzIG4XUbK9DQq13Wjbye6_WlyNMZ4q-YXgWFtIw8KePHq1tcadSDeheO5Mq-uo/s200/epo.png" width="200" /><span id="goog_294558776"></span></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;">"Use" of human embryos, for their own
benefit, is written into the established </span><a href="http://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:31998L0044&from=EN" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;" target="_blank"><span lang="EN-GB" style="color: #0b5394; font-family: Arial; mso-ansi-language: EN-GB;">Directive 98/44/EC of the European Parliament and of the Council of 6 July 1998 Recital(42)</span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"> <span style="font-family: Arial, Helvetica, sans-serif;">on the legal protection of biotechnological
inventions in European states et al and is a foundational document addressing
this area. The interpretation of this document has led to the European Patent
Office guidelines and appeal rulings. </span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">However, apart
from the embryo "use" issue, the </span><a href="http://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:31998L0044&from=EN" target="_blank"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB; text-decoration: none; text-underline: none;"><span style="color: #0b5394;">Directive (Chapter 6.2.b)</span></span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">
specifically states that "processes for modifying the germ line genetic
identity of human beings" are prohibited from Patentability. </span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div style="margin: 0cm 0cm 0.0001pt;">
<span style="clear: left; float: left; line-height: 1.38; margin-bottom: 1em; margin-right: 1em;"><span style="font-family: Arial, Helvetica, sans-serif;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh_7ITpBRHw3x14vk-EwezQ_hKS-00XENC5DzpSoeW7-Yc8OTAZp74npw6T4ZPJCejhFJ_RYSXtzKCOxDFw_EbVWytrzbZU32PfkCU9F2S0PiJvTzEJh-1T2Xp-Tfz8hqrD5M5StLJAAEid/s1600/european-convention-on-huma.png" /></span></span><span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Also, the </span><a href="http://conventions.coe.int/Treaty/en/Treaties/Html/164.htm" target="_blank"><span style="color: #0b5394;">"Convention for the Protection of Human Rights and Dignity of the Human Being with regard to the Application of Biology and Medicine: Convention on Human Rights and Biomedicine Oviedo,4.IV.1997"</span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"> states "An intervention seeking to modify the
human genome may only be undertaken for preventive, diagnostic or therapeutic
purposes and only if its aim is not to introduce any modification in the genome
of any descendants." This forms part of the overa</span></span><span style="font-family: Arial, Helvetica, sans-serif;">ll</span><span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"> </span><a href="http://www.echr.coe.int/Documents/Convention_ENG.pdf" target="_blank"><span lang="EN-GB" style="color: #1155cc; font-family: Arial; mso-ansi-language: EN-GB;">E<span style="color: #0b5394;">uropean Convention on Human Rights</span></span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">, in
all its parts "The Convention."</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhI7c9XVf5pIiaNHVJzz9mlJ9hWNIpBu_eIxWmVsY_SfeBgLxCN7abLtGyifVOcA32tibmYd-JEWlzGwC8sLeMOKv5MVgG1Ypi1hQsCGqVehejNQCKp_YXqzlg1QDNSOsFAsHd_wN-kPQ1_/s1600/EP-logo-.jpg" imageanchor="1" style="clear: right; float: right; line-height: normal; margin-bottom: 1em; margin-left: 1em; text-align: center;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" height="130" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhI7c9XVf5pIiaNHVJzz9mlJ9hWNIpBu_eIxWmVsY_SfeBgLxCN7abLtGyifVOcA32tibmYd-JEWlzGwC8sLeMOKv5MVgG1Ypi1hQsCGqVehejNQCKp_YXqzlg1QDNSOsFAsHd_wN-kPQ1_/s200/EP-logo-.jpg" width="200" /></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">So in Europe
the issue of Human Rights & Innovation Patentability are</span><span style="clear: right; line-height: normal; margin-bottom: 1em; margin-left: 1em; text-align: center;"></span><span style="line-height: 1.38;"> determined by
guideline standards applied mainly throughout the membership via The
Convention & the BioTech Directive, while Individual National Laws are
deferred to, fundamentally by design, in determining the applicable
interpretations & standards governing the specific ethical/moral &
"ordre public" of that society in biomedical research.</span></span><br />
<br />
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjtlj-gIF4PsklZ3V4DGEv1rGfI84owslh64wa9gjMcXAjaXpcVRtVRZV0tgTfSeqW5I8_fC0YBSD-tXzQ53OI794bV62CIWY-l0mEPmM7wXaBjmTW7cexNhEkFV0O-EDDZ9XaTcoTWg-QI/s1600/nih.png" imageanchor="1" style="clear: left; display: inline !important; float: left; line-height: 1.38; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="125" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjtlj-gIF4PsklZ3V4DGEv1rGfI84owslh64wa9gjMcXAjaXpcVRtVRZV0tgTfSeqW5I8_fC0YBSD-tXzQ53OI794bV62CIWY-l0mEPmM7wXaBjmTW7cexNhEkFV0O-EDDZ9XaTcoTWg-QI/s200/nih.png" width="200" /></a><span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;">In the US, it's
also the actual "use" of embryos for research, including the
derivation itself of ESCs, that is the Federal funding restriction. This is a
result of, exclusively at the time of drafting, the destructive method employed
to derive hESC lines. In addition research funding into destructive embryo
studies in areas such as nuclear transfer & genetic modifications of the
germ line were also restricted, as a result. The reiteration of this established position
recently by the NIH reminds all of the reality of the current funding law
governing destructive practices on "human embryos."</span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">However, historically
there has been somewhat of a mixed approach in practice applied to federally
funded embryonic research in the US. The NIH has authorized funding for decades
using donated IVF supernumerary embryonic stem cell lines for research. On the
one hand the law states that no funding is allowed that destroys embryos (e.g.
blastocyst ICM extraction of stem cells, or in this most recent case genetic
editing on embryos that would result in their destruction). However, on the
other hand, this law doesn't apply when work is done on Federal registry
approved embryonic stem cell lines (see </span><a href="http://stemcells.nih.gov/policy/pages/2009guidelines.aspx" target="_blank"><span style="color: #0b5394;">NIH guidelines</span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">). This
reality is a middle way compromise to support the nascent field of advanced
research into developmental biology and has resulted in significant progress in
the understanding and therapeutic potential of pluripotent cell technologies.<o:p></o:p></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Of note, more
recent non-destructive methods, nor research using non-viable embryos, have yet to be written in. For example, non-embryo-destructive sourced Blastomere ESCs,
nor non-viable SCNT-ESCs or Parthenogenetics-hpESCs, are notably excluded from federal
support. So the actual working model isn't that current nor flexible to the
evolving technology, which is reason to review the legislature as a result of
the sector’s broadening scope.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The use of
natural eggs in SCNT-ESCs/hpESCs was perhaps the concern and avowed aspect of
these methods to the Federal Government - but has there been a recent review on
this position given IVF has become a standard option in fertility treatment?
Also the sector is moving fast and emerging reprogramming techniques look to
create synthetic eggs, what then? Is this not a reasonable question to be
asking now, given the discussion? </span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">This line can and may very well be taken further with
technology to create synthetic sperm. Will the combination of synthetic eggs
and sperm be the next ethical issue? I believe there needs to be a concerted
effort to get ahead of the science & write updated Laws that apply new
guidelines with scientifically prudent standards, while remaining open and
flexible to potential benefit & future possibilities.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiKi1yaPSBdgYQnTebgUriOkxc7BPqJXyMlkYAHRuJNVxmuxqcKS9WCTA_YtboA_wInspKaphbkvdZC72cTSHjFnKkiDpqtc8FIY35J-Y5MwBK3LC6ppzPwUfbbj9cli73toFRXMN76b_M9/s1600/curia-logo.png" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEiKi1yaPSBdgYQnTebgUriOkxc7BPqJXyMlkYAHRuJNVxmuxqcKS9WCTA_YtboA_wInspKaphbkvdZC72cTSHjFnKkiDpqtc8FIY35J-Y5MwBK3LC6ppzPwUfbbj9cli73toFRXMN76b_M9/s200/curia-logo.png" width="192" /></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Further, the <a href="http://curia.europa.eu/juris/document/document.jsf?text=&docid=162117&pageIndex=0&doclang=EN&mode=lst&dir=&occ=first&part=1&cid=445883" target="_blank"><span style="color: #0b5394;">European Court of Justice has ruled recently that non-viability</span></a> is a determining
characteristic of the definition of a "human embryo," therefore
non-viable zygotes & arrested/mutated pre-embryos that cannot develop do
not fall within the restrictions of the Biotechnology Directive, as they have
been ruled not to be considered by definition an "human embryo."
However controversial this position may seem to some it is an accurate reflection
and interpretation of the foundational biotechnology law in Europe, while
deferring to national member states the issue of ethical/moral &
"ordre public."</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjBzX0KgaLvt9yI6ssWRDKdbD2W7qpo6vce9eTRp94kU63-38CuMfDtGn7sd_38KJNYsXe5a-pjj279DqLWhqR14Hxtd6AmBa2uK3Qh_aRPaVRExcSLJwJseXgwJf5MZTUXZGA9iwIJnXfa/s1600/European_Court_of_Human_Rights_logo.svg.png" imageanchor="1" style="clear: right; float: right; line-height: normal; margin-bottom: 1em; margin-left: 1em; text-align: center;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" height="120" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjBzX0KgaLvt9yI6ssWRDKdbD2W7qpo6vce9eTRp94kU63-38CuMfDtGn7sd_38KJNYsXe5a-pjj279DqLWhqR14Hxtd6AmBa2uK3Qh_aRPaVRExcSLJwJseXgwJf5MZTUXZGA9iwIJnXfa/s320/European_Court_of_Human_Rights_logo.svg.png" width="320" /></span></a><span style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"></span><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Fundamentally the underlying principles of the
protection of life in the </span><a href="http://www.echr.coe.int/Documents/Convention_ENG.pdf" target="_blank"><span style="color: #0b5394;">Chapter 1 Article 2 of The Convention</span></a> <span lang="EN-GB" style="line-height: 1.38;">doesn't state explicitly that germ line cells, nor for that matter an embryo or
fetus, are to be given specific reserved consideration. This has been tested at
the </span><a href="http://echr.coe.int/Pages/home.aspx?p=home" style="line-height: 1.38;" target="_blank"><span lang="EN-GB" style="color: #0b5394; font-family: Arial; mso-ansi-language: EN-GB;">European Court of Human Rights</span></a><span lang="EN-GB" style="line-height: 1.38;">.
Should national laws allow in-vitro research on embryos </span><a href="http://conventions.coe.int/Treaty/en/Treaties/Html/164.htm" style="line-height: 1.38;" target="_blank"><span style="color: #0b5394;">The Convention states in Chapter 5 Article 18</span></a><span lang="EN-GB" style="line-height: 1.38;"> that they should "ensure adequate protection of
the embryo" and that "the creation of human embryos for research
purposes is prohibited." This is the general positioning at the European
Human Rights level, as a result of the union of culturally diverse member
states. As previously indicated, individual countries <a href="http://www.eurostemcell.org/stem-cell-map?type=regulations" target="_blank">apply local Laws to their societal ethical positions</a>, which they all do in regard to embryos/ESCs, genetics,
IVF & fetal development, considering The Convention. Generally the
principle of human rights & dignity extends to all human beings and for
that the definition of a "human being" is central to The Convention's
interpretation. The </span><a href="http://www.un.org/en/documents/udhr/" style="line-height: 1.38;" target="_blank"><span lang="EN-GB" style="color: #0b5394; font-family: Arial; mso-ansi-language: EN-GB;">UN's Universal Declaration of Human Rights</span></a><span lang="EN-GB" style="line-height: 1.38;"> is similarly worded and looks to respect individual
human rights, while leaving the question of developing life to individual
societies.</span></span><br />
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="line-height: 1.38;"><br /></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh5nMeBqHkTc1xpKlN4xRGB2MXlKDhvBr_Jidz2pfjHa1FeF7lDHC2QSOnM0pcWF3vlod5bjMLHkuPOTgVQYFyu4HqgW0Zq5CChFszpMh2QnyfuSZpTGLWQIeDRUTsB_gi5pmPxJbYdHk0G/s1600/2000px-UN_emblem_blue.svg.png" imageanchor="1" style="clear: left; float: left; line-height: normal; margin-bottom: 1em; margin-right: 1em; text-align: center;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" height="169" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEh5nMeBqHkTc1xpKlN4xRGB2MXlKDhvBr_Jidz2pfjHa1FeF7lDHC2QSOnM0pcWF3vlod5bjMLHkuPOTgVQYFyu4HqgW0Zq5CChFszpMh2QnyfuSZpTGLWQIeDRUTsB_gi5pmPxJbYdHk0G/s200/2000px-UN_emblem_blue.svg.png" width="200" /></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">With regard to cloning there was a specific </span><a href="http://conventions.coe.int/Treaty/en/Treaties/Html/168.htm" style="line-height: 1.38;" target="_blank"><span lang="EN-GB" style="color: #0b5394; font-family: Arial; mso-ansi-language: EN-GB;">Protocol added to The Convention</span></a><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"> in
1998, and enacted by other governing bodies internationally, as a result of the
discussions surrounding animal cloning at the time. Specifically, The
Convention states that "any intervention seeking to create a human being
genetically identical to another human being, whether living or dead, is
prohibited."</span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhQMJyxMcR9n1Ue0AfNSQVZI0ZrPkoH4nbiwCDIUvCMZ13APrp6IODl1Bi3lZoqQeIwLFeda3Lwz-OJRfTrJKzF9e46WiW7bBzWqVGGLZHvsqe-u8MtZKsstIKvEkouTvdNu7AogZKn4wqP/s1600/StemmaPapaFrancesco_18-03-2.png" imageanchor="1" style="clear: right; float: right; line-height: normal; margin-bottom: 1em; margin-left: 1em; text-align: center;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhQMJyxMcR9n1Ue0AfNSQVZI0ZrPkoH4nbiwCDIUvCMZ13APrp6IODl1Bi3lZoqQeIwLFeda3Lwz-OJRfTrJKzF9e46WiW7bBzWqVGGLZHvsqe-u8MtZKsstIKvEkouTvdNu7AogZKn4wqP/s1600/StemmaPapaFrancesco_18-03-2.png" /></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The objections
to assisted reproduction by the Vatican or Christian Right may very well be </span><span style="line-height: 1.38;">subjectively valid, from their reasoned perspective, but that view, however
correct or positioned to be morally sound, doesn't acknowledge nor properly
address the very real practical issues inherent in today's advanced fertility,
cell & genetic sciences. Many of the issues previously debated are being
clinically applied with results. New ethical challenges and redefinitions are
required by all as the science evolves, with appropriate regulatory &
societal frameworks adapted, as necessary.</span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The fact that new technological advancements are being
designed to address medical needs of those that suffer from, or may fall victim
to, potentially treatable biological conditions warrants considered thought as
to how best to unify behind the effort to achieve a host of goals in the
process. Through public education and the application of successful next
generation technology the substance and impact science can have on solving the
very issues that divides opinion is possible.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The ethics of today will give way to the ethics of
tomorrow, and so on - it's nature's way. Man plays his part in this cycle and
uses what is available with intellect and inventiveness. Change is a process of
adjustment and one could say that is the will of nature's law. The only
unnatural aspect would be if man himself becomes defined as synthetic, which
is, from this writer's perspective not the goal. </span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><span style="font-family: Arial, Helvetica, sans-serif;"><img border="0" height="184" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg77mQoXhnNRjB5sneNKarwoWcKThaI60LwC_7g2Z3iB0pLBPw0BYxo27R_NGBOx840Rvq4zcXBMqU-1tDunqQWfafmqldcQmrA93JE9lMxoFYdO4bCBNpY9ELur-pH01vwzy12v7jGBJwR/s320/CRISP-Genome-Editing-Geekswipe-Res-1.jpg" width="320" /></span></span><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Germ line editing in clinical practice is indeed
unnecessary at present until proven otherwise. However, basic research using
gene editing technology of germ line cells is necessary, based on clearly
defined updated ethical frameworks - with governmental support, if possible. The <a href="http://www.isscr.org/home/connect" target="_blank"><span style="color: #0b5394;">recent ISSCR Connect discussion</span></a> on the issue was well presented and reasoned. More open dialogue
is required and opinion sought from all stakeholders. There are too many
questions yet unanswered to not search for the clues by all means so one day we
may apply that knowledge to human frailty & suffering in developing or
developed humans. That goal would be best served by furthering basic research
efforts using genetics back to our original cells. iPS technology wouldn't have
been invented had it not been for human embryonic research, which wouldn't have
been possible without animal cloning studies…. the shoulders' metaphor applies.</span></span><br />
<span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;"><br /></span>
<span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;">From my perspective if gene editing research using
germ line cells and pre-embryos is to be limited entirely to private
companies then that would curtail potential scientific progress in research using
non-viable donations or technology methods which cannot develop into a human by
design.</span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="margin: 0cm 0cm 11pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjIM52pr3sOpajP5JBVrkbZBSsSgHZcsuvuqHoIDvPWRQhaDiU83mhY2IviUOLbLKYVUnZEeBnwBxObZGoU5MdwG40HdvHwLKd_n3ow07sr9jbUZsvVvxDdlOKwYBWRHRYXSZjliWqzyhoc/s1600/Seal_of_the_United_States_C.png" imageanchor="1" style="clear: right; float: right; line-height: normal; margin-bottom: 1em; margin-left: 1em; text-align: center;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjIM52pr3sOpajP5JBVrkbZBSsSgHZcsuvuqHoIDvPWRQhaDiU83mhY2IviUOLbLKYVUnZEeBnwBxObZGoU5MdwG40HdvHwLKd_n3ow07sr9jbUZsvVvxDdlOKwYBWRHRYXSZjliWqzyhoc/s1600/Seal_of_the_United_States_C.png" /></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Congress has
the opportunity to get ahead of these issues and address squarely this area of </span><span lang="EN-GB" style="line-height: 1.38;">leading biotechnology innovation in new legislation. This was shown to be
important during the protracted court case against the Federal Govt.’s funding
of scientific research using approved hESC lines. <a href="http://www.nature.com/news/high-court-ensures-continued-us-funding-of-human-embryonic-stem-cell-research-1.12171" target="_blank"><span style="color: #0b5394;">The high court ruled in favor of the Govt.</span></a> but there was considerable
discussion in legal circles of the need to update the law. The underlying legal
basis being the <a href="http://en.wikipedia.org/wiki/Dickey-Wicker_Amendment" style="color: #0b5394;" target="_blank"><span style="color: #0b5394;">Dickey Wicker amendment</span></a>, which was written in 1995, and is considered by many to
be too </span>ambiguous <span lang="EN-GB"><span style="line-height: 1.38;">and not a suitable
legislative document for the sector moving forward. The need for a
comprehensive bill is generally acknowledged. The use of Federal funds in
developmental biology research should allow for opportunities to explore all non-destructive
areas of the science to advance medical knowledge so that it does not impede
the progress of scientific discovery for the benefit of all. Patients'
interests must be considered paramount and consensus sought on majority based
positioning. Public education can be an effective tool in defining such
efforts. </span></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">For example, currently there is an area of ambiguity
with the written NIH hESC text on embryo donations, as a cell can be harvested
from a pre-embryo and used for that embryo's own benefit, if not for all. </span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Ethical considerations are required to be taken into
account, but not at the expense of an agreed roadmap to progress. If after
broad inclusive deliberations legislative regulations & sector guidelines
are updated, then that achieves the goal. However, I would add a caveat, it’s
important to include into any new laws the non-viable/non-destructive aspects
succinctly, as well as a considered inclusion of a benefit review for technologies
applied to viable potential human embryos in-vitro & in-vivo.</span></span><br />
<span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;"><br /></span>
<span style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38;">However, "use" of embryos isn't the full picture.
There remain issues of scope with respect to reprogramming technology, assisted
reproduction techniques et al which should be clearly stated as part of new
regulations & guidelines in the area.</span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Clarification is needed as to the somatic
reprogramming limits that are acceptable and where there should be restrictions,
if any, applied. Synthetic forms of human germ line cells and the creation of
pure or part-synthetic embryos for cellular harvest cannot be overlooked and
needs similarly considered language. Issues such as same sex couples wishing to
use technology to assist having "natural" babies using reprogrammed
cells back to the germline, artificial wombs and attempts at eugenics et al
should be broadly covered in the legislative language. </span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">If there were clear legislature on the issues, after
dialogue with all stakeholders, this would assist in eliminating the negative
spin on today's advancing scientific discoveries that looks squarely to cure
disease. Science would benefit from that clarity. The future possibilities
would remain, however, guidelines would be established. </span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 11pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Consider if you
will that if the science advances and we are able to achieve that long string
of .999s on safety, what will be the benefit/risk scenario if implementation
occurs some time in the future? Such science can be debated at that point and
submitted for consideration, as long as there's a benefit window.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Generally in the future there may be a manner in which
genetic technology proves its human potential for the application of germ line
intervention. Leaving that door closed while holding a preexisting key isn't
such an unethical position IMO - flexibility in today's fast paced scientific
world is important.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><span style="font-family: Arial, Helvetica, sans-serif;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjvZXos4kG7BhyDhUuerfA8-k8_OCjBxOJY3T-yKApKtigFchdPFgMDrEqg6xOs5FCoy7t0OhUUMb6HYJZrNuxbtxeYOn8psb4OH0xglhhzokZ1C-Xqi0wLG-RgyEnN5idw0jV4ewSQaoFo/s1600/brass-keysm.jpg" /></span></span><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The challenge in establishing regulations in this area
will not be easy, but it isn't insurmountable. A flexible legal &
regulatory basis for steps forward in the research is what is required IMO.
Checkpoints along the way so that the whole map is not null if one road is
opened up upon the presentation of correct documentation. A straightforward
mechanism should be agreed for the review process that encompasses the
appropriate nominated bodies. Congressional oversight may be appropriate but
the nature of such a flexible system would be best served to have it's own
adaptation authority once the law is written.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjtD7aapXL5Suyt103UoLjg96AU7mC67VX83MCiWbJ8DWXb97NjXGgTymdhsMZFgTF0xuk14T7zT9JU0MFc_woDCdSpK9UVBT-6JqFYhkEizimAkf9iNsPfI12QY8RMHcYUe5w3w5OIbm9v/s1600/fda.png" imageanchor="1" style="clear: right; float: right; line-height: normal; margin-bottom: 1em; margin-left: 1em; text-align: center;"><span style="color: black; font-family: Arial, Helvetica, sans-serif;"><img border="0" height="93" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjtD7aapXL5Suyt103UoLjg96AU7mC67VX83MCiWbJ8DWXb97NjXGgTymdhsMZFgTF0xuk14T7zT9JU0MFc_woDCdSpK9UVBT-6JqFYhkEizimAkf9iNsPfI12QY8RMHcYUe5w3w5OIbm9v/s200/fda.png" width="200" /></span></a><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">I have stated previously that the reduction in IVF
supernumerary embryo creation should be a </span><span lang="EN-GB" style="line-height: 1.38;">stated goal with new specific
governance stipulations & authority guidelines over the fertility sector.
This I believe is central to a consensus </span><span lang="EN-US" style="line-height: 1.38;">building</span><span lang="EN-US" style="line-height: 1.38;"> </span><span lang="EN-GB" style="line-height: 1.38;">working
model.</span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">The point is we as a society cannot any longer avoid
the reality of the present and promise of the future by applying yesterday's
fixed reasoning to bear. Without informed, concise & regularly updated
language of the day the necessary support and freedom to research innovative
solutions to pressing medical and biological issues will be unnecessarily
limited.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><span style="font-family: Arial, Helvetica, sans-serif;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjZMLQhyphenhyphen5a0SH1E4psgtl8QODChEylPsxhu1ROaQAhRr4m7g6JJ2AaKhofrcEh6pzSqrgJoPMyEFQH9MiKCPBFslgeIEPtJdAHuPGkHGtRAlMTCdyUKrvj4jJZzbYGdi5AesFn8TvktDuPy/s1600/as17-134-20387sm.jpg" /></span></span><span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">Today the US is the leader in ethical biomedical
technology but without Bold Action, Decisive New Legislature and Increased
Government Support, across the board, the promise of tomorrow's technology with
not meet the expectations of the people nor address the full potential for
American solutions for the Common Good.</span><span lang="EN-GB"><o:p></o:p></span></span></div>
<div class="MsoNormal" style="line-height: 1.38;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br /></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span lang="EN-GB" style="color: #444444; font-family: Arial, Helvetica, sans-serif; mso-ansi-language: EN-GB;">Cheers</span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><br />
<span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;"><br /></span></span></div>
<div style="line-height: 1.38; margin: 0cm 0cm 0.0001pt;">
<span style="font-family: Arial, Helvetica, sans-serif;"><span lang="EN-GB" style="color: #444444; font-family: Arial; mso-ansi-language: EN-GB;">References</span>:</span></div>
<div style="margin: 0cm 0cm 0.0001pt 36pt; text-indent: -18pt; vertical-align: baseline;">
<div style="text-align: left;">
<div style="line-height: 1.38;">
<br /></div>
<ul style="line-height: 1.38;">
<li><a href="http://www.nih.gov/about/director/04292015_statement_gene_editing_technologies.htm" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">Statement on NIH funding of research using gene-editing technologies in human embryos</span></a></li>
<li><a href="http://stemcells.nih.gov/policy/pages/2009guidelines.aspx" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">National Institutes of Health Guidelines on Human Stem Cell Research</span></a></li>
<li><a href="http://www.echr.coe.int/Documents/Convention_ENG.pdf" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">European Convention on Human Rights ("The Convention")</span></a></li>
<li><a href="http://conventions.coe.int/Treaty/en/Treaties/Html/168.htm" style="font-family: Arial, Helvetica, sans-serif; text-align: justify; text-indent: -18pt;" target="_blank"><span style="color: #0b5394;">Prohibition of Cloning Human Being</span></a></li>
<li><a href="http://conventions.coe.int/Treaty/en/Treaties/Html/164.htm" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">Convention on HumanRights and Biomedicine, Oviedo, 4.IV.1997</span></a></li>
<li><a href="http://eur-lex.europa.eu/legal-content/EN/TXT/HTML/?uri=CELEX:31998L0044&from=EN" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">Dir 98/44/EC European Parliament/Council '98: Legal Protection Biotech Inventions</span></a></li>
<li><a href="http://curia.europa.eu/juris/document/document.jsf?text=&docid=162117&pageIndex=0&doclang=EN&mode=lst&dir=&occ=first&part=1&cid=445883" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">European Court of Justice ruling on Parthenotes as not "human embryos"</span></a></li>
<li><a href="http://www.coe.int/t/dg3/healthbioethic/Activities/04_Human_embryo_and_foetus_en/CDBI-CO-GT3(2003)13E.pdf" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">Council of Europe -Steering Committee on Bioethics: In Vitro Embryo Protection</span></a></li>
<li><a href="http://www.un.org/en/documents/udhr/" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span lang="EN-GB" style="color: #0b5394; mso-ansi-language: EN-GB;">United Nations: The Universal Declaration of Human Rights</span></a></li>
<li><a href="http://echr.coe.int/Pages/home.aspx?p=home" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span style="color: #0b5394;"><span lang="EN-GB" style="color: #1155cc; mso-ansi-language: EN-GB;">European</span><span lang="EN-GB"> </span><span lang="EN-GB" style="color: #1155cc; mso-ansi-language: EN-GB;">Court</span><span lang="EN-GB"> </span><span lang="EN-GB" style="color: #1155cc; mso-ansi-language: EN-GB;">of</span> Human <span lang="EN-GB" style="color: #1155cc; mso-ansi-language: EN-GB;">Rights</span></span></a></li>
<li><a href="http://en.wikipedia.org/wiki/Dickey-Wicker_Amendment" style="font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-indent: -18pt;" target="_blank"><span style="color: #0b5394;"><span lang="EN-GB">Dickey</span><span lang="EN-GB"> </span><span lang="EN-GB">Wicker</span><span lang="EN-GB"> </span><span lang="EN-GB">Amendment</span></span></a></li>
<li><a href="http://www.nature.com/news/high-court-ensures-continued-us-funding-of-human-embryonic-stem-cell-research-1.12171" style="font-family: Arial, Helvetica, sans-serif; text-align: justify; text-indent: -18pt;" target="_blank"><span style="color: #0b5394;">US Supreme Court ensures continued US funding of human embryonic-stem-cell research</span></a></li>
<li><span dir="LTR" style="color: #0b5394; font-family: Arial, Helvetica, sans-serif; line-height: 1.38; text-align: justify; text-indent: -18pt;"><a href="http://www.isscr.org/home/connect" target="_blank"><span lang="EN-GB" style="color: #1155cc; mso-ansi-language: EN-GB;">Genetic Modifications in Humans:The Scientific Methods, Medical Applications and Ethical Implications</span></a><a href="http://echr.coe.int/Pages/home.aspx?p=home"><span style="color: #1155cc; mso-ansi-language: EN-GB;"> </span></a></span><span lang="EN-GB" style="font-family: Arial, Helvetica, sans-serif; font-size: x-small; text-align: justify; text-indent: -18pt;">(<i>ISSCR</i>
Connect Video On Demand: Dr. George Daley & Prof. Timothy Caulfield)</span></li>
<li><span lang="EN-GB" style="font-family: Arial, Helvetica, sans-serif; font-size: x-small; text-align: justify; text-indent: -18pt;"><a href="http://www.eurostemcell.org/stem-cell-map?type=regulations" target="_blank">European regulatory map on ES research via Eurostemcell.org</a></span></li>
</ul>
<ul style="line-height: 1.38;">
</ul>
</div>
</div>
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-11533780393558001552015-04-23T10:11:00.003-07:002015-04-23T11:17:40.357-07:00Germline Editing using CRISPR-Cas9 - Totipotent Cell Research<table cellpadding="0" cellspacing="0" class="tr-caption-container" style="float: left; margin-right: 1em; text-align: left;"><tbody>
<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgOAZ_SoFNeV36gtLh5whx5ZDRbxmYpPRRwc5lSBRdQgLfkL7GT8bfzWPycBzFwmyONKaecmMYwW70klnINM0qpcW5i2MYuquBn9o6WG6ooslXMjsfUqFnv2jVgj-P8RaY4DMtDTsdgb_wE/s1600/Gene+Edited+Chinese+Image.jpg" imageanchor="1" style="clear: left; margin-bottom: 1em; margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgOAZ_SoFNeV36gtLh5whx5ZDRbxmYpPRRwc5lSBRdQgLfkL7GT8bfzWPycBzFwmyONKaecmMYwW70klnINM0qpcW5i2MYuquBn9o6WG6ooslXMjsfUqFnv2jVgj-P8RaY4DMtDTsdgb_wE/s1600/Gene+Edited+Chinese+Image.jpg" height="232" width="320" /></a></td></tr>
<tr><td class="tr-caption" style="text-align: center;">Dr.Yorgos Nikas/SPL</td></tr>
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Yesterday the first <a href="http://link.springer.com/article/10.1007%2Fs13238-015-0153-5" target="_blank">published paper</a> was released detailing the preliminary scientific attempts at modifiying the germline of a human fertilized egg - a zygote.<br />
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What was striking here wasn't so much the attempt being officially published but the storm it has caused in the scientific community.<br />
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The science is far from effective and needs a great deal of work before it is even possible to consider using in a clinical setting. That much was shown. There are many steps before any such work should be even contemplated, as <a href="http://www.isscr.org/home/about-us/news-press-releases/2015/2015/04/23/release-on-human-germline-genome-modification" target="_blank">guided by the ISSCR</a> in their call for a clinical moritorium on proceding with any human translational work. I personally believe that position will be respected given the laws in place that govern such human germline science. If there is a precedent the ban on attempts to pursue human cloning has shown to be effective.<br />
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What is curious here in respect to this work, undertaken in China, on germline editing is that it has quickly followed the cautionary notices by the scientific community leadership that the science was being practiced in human germline cells. This was and is a signal that there is much more going on behind the scenes than we know and that the nature of such developments isn't necessarily coordinated or managed in any practical manner. To some that is the issue perhaps...<br />
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The explosion of interest and experimental use of gene editing technology has opened up a proverbial Pandora's box of issues that have yet to be addressed collectively in a meaningful manner. But as these things go it's not always possible to steer scientific advances as the peer review process is built to open up the knowledge gates to replicability and improvement - hence the strength in it's design. Coordinated stakeholder dialogue is urgently needed and initiatives established.<br />
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Man's curiosity and pursuit of knowledge has driven momumental changes in our own lifetimes - that is accelerating and those that wish to somehow control the speed of which it is happening may find themselves catagorized as part of the old guard and not hip to the trending interwoven nature of the instant always on tech culture of today and tomorrow.<br />
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The ever expanding numbers of brains educated to think of new solutions to existing problems with ubiquitious tools made easy will allow discovery and experimentation to flourish. No one should be surprised when this momentum spawns innovation - especially in the new frontier of biological system design.<br />
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My comments on <a href="http://www.ipscell.com/2015/04/gmhumanembryos/#comment-31503" target="_blank">Paul Knoepfler's Blog</a> about the natural reservations of the developments are below:<br />
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<i>"Thx Paul for the viewpoint. </i><br />
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<i>I believe you´re correct to have reservations about the unknown steps on the discovery path to gene line editing. However, I do believe it's important to participate in order to have any chance to mold the outcome. As we have just learned the science is indeed moving ahead, like it or not. I don't believe debating the merits of the possibilities does justice to the technology - at least to me that doesn't seem productive at this stage. </i><br />
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<i>Some may not wish to go down this discovery path but as many have pointed out - it's not something anyone or even a group(s) can put a halt to, it seems. </i><br />
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<i>I would like to see the West actively engaged in an initiative to put a scientific team of institutional investigators together to spearhead a collaberative international effort to lead coordinated research into geneline editing - in all it's forms (there are many of course research methods to explore). This way the open nature of such an initiative would be beneficial to all stakeholders and those on the ground can draw down from this central resource.</i><br />
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<i>Attention grabbing science papers as a methodology to present the public the work being done adhoc doesn't strike me as proactively getting ahead of the issues... </i><br />
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<i>Also, isn't it possible to establish a seperate arm's length oversight department, in an existing organizational structure? This would officially sanction & coordinate the independent local discovery groups to inform and assist the lab work & validate data collection for the entire space. Or are we looking at global independent action, reaction, applied science and IP castle building for human elitism?"</i><br />
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<tr><td class="tr-caption">Belmonte paper ref</td></tr>
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Further on the issue of gene editing, today Juan Carlos Izpisua Belmonte’s group published a <a href="http://www.cell.com/cell/abstract/S0092-8674(15)00371-2" target="_blank">paper in Cell</a> that suggests a possible route to modify mutated mitochondrial for disease elimination using an alternative to the above "CRISPR-Cas9" technology but similar in puprose "TALEN" system.<br />
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The sum of these parts and the overall emerging landscape of profound medical advancements is a net positive IMO, while there remains a need to establish a collective approach to coordinating the possibilities for Common Good.<br />
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Cheers <br />
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-82754526884136612392015-03-23T06:42:00.000-07:002015-04-06T13:08:06.802-07:00The Rise of Germline Science - Human DNA Modification<div style="text-align: justify;">
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgPhphXz2DffK45_heqK50sktymrMfySYKSxUnz4Ptr-WdZYL80E5rh-UT0kkgWqrJfngSiBPzccqXHvCadXx5rvmwROzQZrVElSqCJfa7n988lLK2vqFUPWfMX7Q-acrfAe-2QSe-asF1O/s1600/DNA1120crop.png" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgPhphXz2DffK45_heqK50sktymrMfySYKSxUnz4Ptr-WdZYL80E5rh-UT0kkgWqrJfngSiBPzccqXHvCadXx5rvmwROzQZrVElSqCJfa7n988lLK2vqFUPWfMX7Q-acrfAe-2QSe-asF1O/s1600/DNA1120crop.png" height="159" width="320" /></a><span style="font-size: large;">G</span>enetic modification of human DNA to correct disease causing code has long been a stated goal of medical science. Ever since the discovery of the double helix has man dreamed of understanding the inner workings of its own self, at its very core. The successful mapping of our genome took us closer to that universally acknowledged ambition. Time has not stood still since then nor has science strayed yet from its intended course. The rapid expansion of practical applications for genetics addressed to medical needs is a testament to the pursuit of that knowledge for good. In time there will be more progress and deeper understanding of the impact changes to our code will have - in the meantime research must continue apace and the wings of discovery allowed to extend outwards. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjV4NH3eQleFxKuawx-aHERpu75G1qW1yhZ-BrfrZuEe8_VNRLTnfTWW8gBJ8yu5erlc5ug95JNN7uOnyszLA2QAk8yY79BR6p0tHoPOM9Lxw1zZtmgNPf-x_1r7CCGupH5MT6lN51mCtmo/s1600/ISSCR_logo.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjV4NH3eQleFxKuawx-aHERpu75G1qW1yhZ-BrfrZuEe8_VNRLTnfTWW8gBJ8yu5erlc5ug95JNN7uOnyszLA2QAk8yY79BR6p0tHoPOM9Lxw1zZtmgNPf-x_1r7CCGupH5MT6lN51mCtmo/s1600/ISSCR_logo.jpg" height="100" width="320" /></a><span style="font-size: large;">M</span>y perspective on extending genetic research to germ line DNA editing is in line with the <a href="http://www.isscr.org/home/about-us/news-press-releases/2015/2015/03/19/statement-on-human-germline-genome-modification">recent guidance</a> from the International Society for Stem Cell Research (<a href="http://www.isscr.org/" target="_blank">ISSCR</a>). </div>
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<span style="font-size: large;">R</span>ather than positioning early, and perhaps prematurely, the ongoing conversation is best taken forward with open engagement and presentation of additional scientific data, as it becomes available. Without additional ethical germline research, supported by industry & regulators, the topic of whether there is reason for undue concern cannot be fully addressed scientifically, apart from debating the broader question of should we (which is conditional imo). To reach any natural conclusion will take time and further industry wide clinical application of non-germline editing technologies, acceptance of less invasive genetic modification technologies, such as mitochondrial transfer, and the implementation of other embryo technologies for human benefit. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg-ty2Wka9bOpQzgpy6-xYhyphenhyphenF5Dw9MMl528Q9_tQw5x3DrTv1qR7UvWRUKLRDBscYGvCvSWlhJRKDklduHo0PtVFhCaGMV6INbavdRXgvb5PI7hoAjPdt1n8wHdTCofJKgm04fForNghMkH/s1600/rocks-in-balance.full_.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg-ty2Wka9bOpQzgpy6-xYhyphenhyphenF5Dw9MMl528Q9_tQw5x3DrTv1qR7UvWRUKLRDBscYGvCvSWlhJRKDklduHo0PtVFhCaGMV6INbavdRXgvb5PI7hoAjPdt1n8wHdTCofJKgm04fForNghMkH/s1600/rocks-in-balance.full_.jpg" height="212" width="320" /></a><span style="text-align: justify;"></span></div>
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<span style="font-size: large;">A</span>n observation that underlies the issue is perhaps the historical fear factor effect of the "dual-use dilemma." That is to say the mere fact that there exists a possibility of misuse of scientific discoveries has in the past created significant barriers to progress and inhibited otherwise important developments in the timely advancement of research efforts to eradicate human disease & suffering. Human cloning wasn't on the agenda when Dolly was born, however the realization that SCNT technology had a possible "dual-use" potential has hobbled otherwise beneficial therapeutic sources for ES cells. The entire field of "embryo" science has itself been caught up in an unproductive polarization issue of creation, life & intervention for cell science application. Even today only a very small percentage of the public actually know science is able to establish ES cell lines across all "embryo" technologies in a non-destructive ethical manner for personal & universal cellular treatments. This generally needs to be factored in when discussing the topic as it's unethical in itself not to address the fundamental reasons for many objections to "embryo" science and how it has clouded the perspective of delivering solutions to those in need. Solutions need to be sought, compromise established & educational outreach in the mass media targeted to this very point to end the divisiveness. This germline debate presents an ideal opportunity for such rapprochement.</div>
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<span style="font-size: large;">H</span>ow is early genetic modification of DNA at the germ/embryo stage going to be any different than the hES cell source miscues of the past unless the rhetoric of the applied technological benefit conversations reflect the lessons learned? </div>
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<span style="font-size: large;">G</span>etting ahead of the issue is important, as it is with all emerging technological innovations that involve health. However, ensuring the safe sector development of recombinant DNA isn't the same as the ethical impact of human cloning, creating tens of millions+ supernumerary IVF embryos, engineering synthetic eggs/sperm & embryos, conducting germ line editing for disease or introducing DNA species' enhancements et al. These topics aren't challenges for science to overcome alone, they are fundamentally moral & ethical questions of benefit and use. They require careful scientific study, presentation in an open forum (not pay walled), thoughtful inclusive dialogue, education and public outreach. </div>
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<span style="font-size: large;">P</span>erhaps Leadership should suggest specific beneficial goals and propose a working version of a current dual use limitation charter when addressing the issue in the public framework. This will enable the field and public to express themselves within a clearly defined yet productive manner - i.e. what is potentially acceptable to Leadership and what isn't at this stage. Otherwise I can see the conversation repeating itself along the same lines as the media spin of scientific hyper reporting for readership numbers & incorrect assumptions of SCNT=human cloning, which was & still is an acknowledged no-go area in embryonics. Remember this concern set the tone for almost 20 years now of fallout in the field, not as a result of any specific efforts to progress human cloning but for the open door it presented. This was irrespective of the inherent scientific value in autologous ES cells & the sound pursuit of careful research driven embryo based technologies.</div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgU-qo8CvGFmD0UI40Ob5FaXggqFi4Kky34vArTq23dli34Lbr8eLsEelUbpbWle7ZeGvhBUDF8ffBK46ShkD7a-Kq9AcPhIcYuWAUFYm1XRZ_0t5hyphenhyphenQwxhBYzBkG4XvZxWSxKmp1bBjs3n/s1600/fig_3_thompson.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgU-qo8CvGFmD0UI40Ob5FaXggqFi4Kky34vArTq23dli34Lbr8eLsEelUbpbWle7ZeGvhBUDF8ffBK46ShkD7a-Kq9AcPhIcYuWAUFYm1XRZ_0t5hyphenhyphenQwxhBYzBkG4XvZxWSxKmp1bBjs3n/s1600/fig_3_thompson.jpg" height="158" width="320" /></a><br />
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<span style="font-size: large;">H</span>istory has presented a number of important dual use dilemmas over the course of human history, nuclear power may have been/is the most impactful to human life: energy versus war; restraint versus force; detente versus annihilation; rule of law versus rogue use... One can do the trade offs in perhaps more applicable sciences, for example: synthetic life forms; biological constructs; chemical engineering et al. We live in a never ending cauldron of possibilities where all outcomes increasingly exist as a result of human ingenuity. The rule of law and our moral codes are the only binding principles that the community can foster to fundamentally shape human will. If there is a need for new laws then do so with factual education, public debate and openness centered around a health priority of "for the people" being paramount. However, competitive currents in science run deep today and as such active participation with guidelines would seem the only logical course. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhDehBdZB99xf9dv5ZOOryMhzM436YD927KAJioKPtuD4BhwaNeiVPhi-TT4-OhQ7UxLTPfuEpBAE62xFpcF2EazDwdTdPPrLnWtlIGNROD8kyGhrdl-oxUKM_AyJK4n7by9GyvLmNXcOnx/s1600/aan.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhDehBdZB99xf9dv5ZOOryMhzM436YD927KAJioKPtuD4BhwaNeiVPhi-TT4-OhQ7UxLTPfuEpBAE62xFpcF2EazDwdTdPPrLnWtlIGNROD8kyGhrdl-oxUKM_AyJK4n7by9GyvLmNXcOnx/s1600/aan.jpg" height="102" width="320" /></a></div>
<span style="font-size: large;">I</span>f there were publicly exposed real life cases where germ line modification would alleviate suffering & disease in newborn children then I would recommend that be shared as widely as possible. Certain neurological diseases have been mentioned - if germline science is the only way forward in some of these cases it's important to publicly state that & make it clear why. These and other indications may very well be the first cases where there is a real need to use germline/embryo editing, however somatic cell gene therapy is without doubt the immediate and overwhelming priority. </div>
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<span style="font-size: large;">I</span> sketch below a fictitious scenario whereby a family has no choice, unless assisted by science - would it be ethical and morally correct to deny them their human rights to bear healthy children, should a technology be available to solve their case?</div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEihaIzULIh6lWZZ-Tto_hdfS_Myj7RhPymfbbvygrarM6rYM3Y0bKtiMbstSdWFDZCiO_d2cb6QqRyk2GaGku5I57eweIyT5Sn7VtsbY6ytoc1VxZt57WWoDFCecysa7rFb2NSETqxCNF1_/s1600/illumina-dna-seq-library-prep.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEihaIzULIh6lWZZ-Tto_hdfS_Myj7RhPymfbbvygrarM6rYM3Y0bKtiMbstSdWFDZCiO_d2cb6QqRyk2GaGku5I57eweIyT5Sn7VtsbY6ytoc1VxZt57WWoDFCecysa7rFb2NSETqxCNF1_/s1600/illumina-dna-seq-library-prep.jpg" height="115" width="400" /></a></div>
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<span style="font-size: large;">W</span>hat happens when a known gene mutation is detected in an IVF embryo to a family with a genetic mutation history? Choice & hope drove them to look to protect against the very real & terrible consequence of giving birth to a child that will only suffer and die. If they didn't choose IVF fertility they would have no ability to protect the health of their child. God to them doesn't mean accepting a cruel and unjust outcome that imposes suffering on an innocent if there is an alternative. Don't implant that embryo and select/create more embryos until there is one which doesn't have the mutation? Is that acceptable to those that oppose IVF supernumerary embryo creation? What happens when all the created IVF embryos have the mutations? Don't implant any and send the couple home without solving their problem at considerable cost and emotional stress? Is that ethical? </div>
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<span style="font-size: large;">W</span>hat happens when the Docs find a potentially suitable IVF embryo but advise that there is a risk that the couple faces that the disease could manifest itself anyway without complete replacement of the relevant DNA strands? Implant without a DNA edit and hope for the best? Try to detect any malformation issues during fetal development? Then what? Fetal intervention? If that's not possible? T<span style="text-align: left;">ry to solve any postnatal genetic issues with best efforts? What if it's too late for any real chance to help and the child is effectively DOA or critical beyond hope and cannot be expected to regain near normal function even with the most advanced modern treatment at the cellular level? Don't risk it? </span></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEihaIzULIh6lWZZ-Tto_hdfS_Myj7RhPymfbbvygrarM6rYM3Y0bKtiMbstSdWFDZCiO_d2cb6QqRyk2GaGku5I57eweIyT5Sn7VtsbY6ytoc1VxZt57WWoDFCecysa7rFb2NSETqxCNF1_/s1600/illumina-dna-seq-library-prep.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><br /></a></div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgA8tz_4QCT7mvNDbkxFv8i4f77onqGMW2lQEW4DivHyQzkhOfkrwa3FkgWbrUcrc6QrXAvBlxzshG6vTzTG8fIkik7AtVIS3xWoMbsWC2RyJAAz7icyY7WF2_QE4FbzXrzpAB-ZjYwQHCo/s1600/brain+issue+baby.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em; text-align: center;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgA8tz_4QCT7mvNDbkxFv8i4f77onqGMW2lQEW4DivHyQzkhOfkrwa3FkgWbrUcrc6QrXAvBlxzshG6vTzTG8fIkik7AtVIS3xWoMbsWC2RyJAAz7icyY7WF2_QE4FbzXrzpAB-ZjYwQHCo/s1600/brain+issue+baby.jpg" height="179" width="320" /></a><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgA8tz_4QCT7mvNDbkxFv8i4f77onqGMW2lQEW4DivHyQzkhOfkrwa3FkgWbrUcrc6QrXAvBlxzshG6vTzTG8fIkik7AtVIS3xWoMbsWC2RyJAAz7icyY7WF2_QE4FbzXrzpAB-ZjYwQHCo/s1600/brain+issue+baby.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><br /></a><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgA8tz_4QCT7mvNDbkxFv8i4f77onqGMW2lQEW4DivHyQzkhOfkrwa3FkgWbrUcrc6QrXAvBlxzshG6vTzTG8fIkik7AtVIS3xWoMbsWC2RyJAAz7icyY7WF2_QE4FbzXrzpAB-ZjYwQHCo/s1600/brain+issue+baby.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><br /></a><span style="font-size: large;">T</span>his on the other hand is not fiction. My brother has a seemingly healthy young 7 yr old son but the child's siblings weren't so lucky. Both were taken to full term, delivered and died. My sister-in-law had excellent medical care and after the loss of the first child, within days of birth, was monitored constantly throughout the 2nd & 3rd pregnancies. Her second child was again naturally conceived and made it through the gauntlet. The 3rd child died weeks after being born "healthy" & welcomed home into the family. They have suffered immensely and you can sense their deep sadness but they have joy & love in their hearts with the one that survived. We pray that little chap makes it all the way but if there were a way to screen the germline of his parents they may not have needed to play Russian roulette. In hindsight and with use of today's technology who would in their right mind try to conceive naturally knowing there was a significant risk, especially after having a previous issue? If a fatal brain disorder could go undetected in today's system and manifest itself in 2 out of 3 full term pregnancies then there is a real need to improve the process. Genetics presents the best hope to protect against such outcomes and it must be taken forward for all families. Parents to be should seek basic genetic testing and those at most risk must be offered IVF & advanced screening as standard. Informed decisions can be made once the available options are presented by the medical experts. If at some point in the future the process includes safe interventional genetics to assist life in being born healthily then I favor presenting such a choice to would be parents, if science has made it possible. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhJn4eZIqbI-Rg0zwr8WS7KhPFGjNCMYjWG366CBjq-Ez_Qf3TAX6BZM9dfs_cRvflJyykm6SHd1eRNrIkjZ2ST76mHb50V17ZIYUAYq6Pv6jzkQ10Kj3WvoDyq1dvc_OE9YARnzhyphenhyphenLhEbb/s1600/healthy-infant.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em; text-align: center;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhJn4eZIqbI-Rg0zwr8WS7KhPFGjNCMYjWG366CBjq-Ez_Qf3TAX6BZM9dfs_cRvflJyykm6SHd1eRNrIkjZ2ST76mHb50V17ZIYUAYq6Pv6jzkQ10Kj3WvoDyq1dvc_OE9YARnzhyphenhyphenLhEbb/s1600/healthy-infant.jpg" height="142" width="200" /></a><span style="font-size: large;">T</span>he parents of tomorrow are ultimately responsible for the next generations and therefore should be considered with weight, as with those that have direct experience in the diseases for which this germline technology would apply, if any. I don't believe shock is an emotion the youth of today exhibit when presented with the technology of tomorrow. Rather it would be a shock to all if the very real images of affected children were associated with political inaction to cure, if there was a chance to avoid such pain & suffering.</div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjxuqtDE8aliyfulbdd40MjvBIFDwOJpXEGIabP3DjDL0SgGfmUrxqpJ88BGqWf33p6kGle5yPKU5W_tgevA6mlbdfq6NNfeAcClhOyB_hs0P-ughCoMxXG-y6udGZ5CCWRHrXlMrv_DbFU/s1600/Remains-of-the-Day-_Forgotten-Christopher-Reeve-DVD.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjxuqtDE8aliyfulbdd40MjvBIFDwOJpXEGIabP3DjDL0SgGfmUrxqpJ88BGqWf33p6kGle5yPKU5W_tgevA6mlbdfq6NNfeAcClhOyB_hs0P-ughCoMxXG-y6udGZ5CCWRHrXlMrv_DbFU/s1600/Remains-of-the-Day-_Forgotten-Christopher-Reeve-DVD.jpg" height="145" width="200" /></a></div>
<span style="font-size: large;">H</span>aving said that the entire field of somatic genetics and cellular therapeutics is just emerging and needs to be the focus to establish itself prior to more advanced possibilities taking over the headlines. The media feed off controversy so it's best to find a way to agree than to publicly disagree, for the sector.</div>
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<span style="font-size: large;">O</span>nce the intervention technologies are sound the medical community has an obligation, if not a legal duty, to present the options. In time once the public are familiar with the detection technology their acceptance of the more complex interventional options will become less futuristic and ethically questioned, in those cases where the only guarantee is germ line modification. </div>
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<span style="font-size: large;">G</span>enetics holds enormous promise, as do potent cellular therapeutics. The union of the two, and their by-products, will be a powerful force for good. Early detection, intervention and eventually genetic modifications will be the key to freeing humans from their own frailties, which if managed as a universal community goal will serve rather than enslave generations to come. This future must be controlled and accessible to all.</div>
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<span style="font-size: large;">F</span>or the moment though I can't see any reason whatsoever to accept personalized genetic enhancements nor use germline technology, if proven safe, beyond the absolute required need. Intervention to eliminate any possibility of irreversible disease, when a couple has no opinion, is where science can help bear a healthy child free of fatal developmental mutations. </div>
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjR2i5VCqQDvr7GqTvLcBeOtMa1LRiyRyOChOtGl0XRA87PKjTH3vBTUzMVLVSxzhd-azKqoc-GECZwj0OvpCBX1O3rtnz4gY5xgN0gt08qLvA4gakqJCs4aZzQTkCCuvfyt120xekg2ZhH/s1600/WHO.gif" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjR2i5VCqQDvr7GqTvLcBeOtMa1LRiyRyOChOtGl0XRA87PKjTH3vBTUzMVLVSxzhd-azKqoc-GECZwj0OvpCBX1O3rtnz4gY5xgN0gt08qLvA4gakqJCs4aZzQTkCCuvfyt120xekg2ZhH/s1600/WHO.gif" height="200" width="196" /></a></div>
<span style="font-size: large;">P</span>erhaps in time there will be safe cost effective universal "vaccine" like concepts that edit our DNA in order to eradicate disease, strengthen our physical constructs, enhance our immune systems and extend our cellular longevity. A legacy any generation would envy... That would be a world worth imagining into existence - along with some other wish list items! </div>
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<span style="font-size: large;">A</span>dvocacy for <span style="font-family: inherit;">cures.</span><br />
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<span style="font-size: large;">C</span>heers<br />
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<span style="font-size: large;">R</span>eferences:<br />
<a href="http://www.technologyreview.com/featuredstory/535661/engineering-the-perfect-baby/" target="_blank"><span style="font-family: inherit;">MIT Gene Editing Article</span></a><br />
<a href="http://www.ipscell.com/2015/03/georgechurchinterview/" target="_blank"><span style="font-family: inherit;">PaulK Blog Interview with Dr. George Church</span></a><br />
<a href="http://www.nature.com/news/don-t-edit-the-human-germ-line-1.17111" target="_blank"><span style="font-family: inherit;">ARM Moratorium Request</span></a><br />
<a href="http://www.isscr.org/home/about-us/news-press-releases/2015/2015/03/19/the-isscr-statement-on-human-germline-genome-modification" target="_blank"><span style="font-family: inherit;">ISSCR Guidance Statement</span></a><br />
<a href="http://www.sciencemag.org/content/early/2015/03/18/science.aab1028.full" target="_blank"><span style="font-family: inherit;">Genetic Scientists Policy Recommendations (pay walled)</span></a><br />
<span style="font-family: inherit;"><a href="http://portal.unesco.org/en/ev.php-URL_ID=13177&URL_DO=DO_TOPIC&URL_SECTION=201.html">Universal Declaration on the Human Genome & Human Rights - UNESCO</a><br /><a href="https://www.genome.gov/10001744">NIH Bioethics Resource</a></span><br />
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</h3>@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-42515953874265834772015-01-13T08:37:00.002-08:002015-08-25T02:05:13.880-07:00The Hemangioblast Progeny - Ocata's Blood, Immune & Cancer Programs<div style="text-align: justify;">
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<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg5Lv47Kwvv2OIM_smFpRudR-ba7TX-YWP73Qe-vIX1CTTUA3Vc96Q4fEIDLw9TB-mZ9MLYPmbmyV_jNvP649r9XLT9U3Di0GC-M9ZVHLfH3pfclMot01h13LNHzy0TwNJ5akx3_vt4py5P/s1600/Hemangioblast.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="158" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEg5Lv47Kwvv2OIM_smFpRudR-ba7TX-YWP73Qe-vIX1CTTUA3Vc96Q4fEIDLw9TB-mZ9MLYPmbmyV_jNvP649r9XLT9U3Di0GC-M9ZVHLfH3pfclMot01h13LNHzy0TwNJ5akx3_vt4py5P/s200/Hemangioblast.jpg" width="200" /></a></div>
When we speak of the various programs Ocata has in development, apart from the Eye, they are referred to as "other" and are separated into boxes so the general targets become clearer - Blood, Immune & Cancer.</div>
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However, it important to understand that all these "others," and their various derivative relations, are based off of the Hemangioblast cell state precursor... i.e. <span style="font-size: x-small;">e</span>Immune Modulation using MSCs, <span style="font-size: x-small;">e</span>Cancer Targeting using Dendritic/NK cells & <span style="font-size: x-small;">e</span>Blood Repair using Platelets, all via Ocata's Non-Destructive <a href="http://investorstemcell.com/forum/msemporda/8412.htm" target="_blank">hESC Platform source</a> or from any of the alternative Pluripotent Platform technologies (i.e. <a href="http://investorstemcell.com/forum/msemporda/23533.htm" target="_blank">iPS</a>, <a href="http://msemporda.blogspot.com.es/2014/12/parthenogenesis-derived-pluripotent.html" target="_blank">Parthenogenesis</a>, <a href="http://investorstemcell.com/forum/msemporda/23656.htm" target="_blank">SCNT</a>).</div>
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Tissue engineering (organs) isn't yet in the preclinical phase at Ocata but could of course be a focus of research and product development/IP licensing if the company opts to push forward in that direction down the line with <a href="http://msemporda.blogspot.com.es/p/pluripotent-science.html" target="_blank">Pluripotent cells</a>, most likely with Autologous reprogrammed cells using <a href="http://investorstemcell.com/forum/msemporda/23533.htm" target="_blank">iPS</a>, <a href="http://msemporda.blogspot.com.es/2014/12/parthenogenesis-derived-pluripotent.html" target="_blank">Parthenogenesis</a> or <a href="http://investorstemcell.com/forum/msemporda/23656.htm" target="_blank">SCNT</a> Pluripotent sources.</div>
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For now it's the Allogeneic Hemangioblast offspring and as such all discussions in <a href="http://msemporda.blogspot.com.es/p/ocata-blood.html" target="_blank">Blood</a>, <a href="http://investorstemcell.com/forum/msemporda/31863.htm" target="_blank">MSC/Immune</a>, <a href="http://investorstemcell.com/forum/msemporda/25320.htm" target="_blank">Cancer</a> should be considered in relation to this when reviewing what the opportunity is for product development.</div>
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A program for engineered eDendritic cells targeting a pathogenic or virus strain can be separated from an engineered eDendritic cell deal for a Cancer indication - both derived from a Patented Pluripotent eDendritic cell IP rights chain that goes back three Patent layers to source. However, on top of those source IP rights is the engineered cell product itself (synthetic Product Claim) which will be the Exclusive Product License.</div>
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Is it more beneficial to break-up IP than outright License it wholesale per cell type? Will this occur When and If unique synthetic versions can be developed to optimize & uniquely address target market indications? For example why License the entire Cancer market to one partner when there are multiple potential partners in the space? Granted a potential Partner may address a cross-section of the space but one Partner may only be interested in or dominate in a few disease areas while others may be active in unrelated diseases and so forth... Each can have a tailored product using exclusive IP from cell origin to intermediate cell states to final proprietary formulation...</div>
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This is the strategic design for securing multiple partnerships to Ocata's underlying Platform & Program IP.</div>
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It is the unique or synthetic nature of the final product for distinct targets that will exponentially and dramatically expand the scope of collaborative understanding with Industry players.</div>
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The explosion in awareness and movement in cellular targeting via synthetic biology is the beginning of the paradigm shift in medicine.</div>
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CAR-T has been a research focus for a couple of years ago now - which was a welcome adjunct to an on-again off-again research effort on immunity, genetics & gene editing technologies. The nature of beating disease by way of empowering & harnessing the immune/blood system is fundamental to next generation therapies. <a href="http://msemporda.blogspot.com.es/p/cancer.html" target="_blank">Emily</a> was one such proof many had been hoping for. Not that pure cells themselves or synthetic editing aren't invaluable in their own right to fight disease, but that complex modified biological cell constructs are possible as future effective treatments.</div>
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As with all cell treatments the ability to manufacture an inexhaustible supply of a replicable standard as an off-the-shelf product is what will separate the mass market offering from the targeted niche. The cell progeny differentiated to a terminal state and used in therapeutics worldwide is akin to a small molecule drug produced on an assembly line.</div>
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Autologous treatments against Cancer may be required when specific and unique disease states & expression patterns apply and will therefore require bespoke cellular targeting. However, in the broader market a product with replicable standards, cost & scale will be vital and sought after by way of Allogeneic product methodologies. </div>
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Some Allo programs already exist based on adult cell sources however when the inherent expression benefits of Pluripotent derived cell factories are combined with synthetic multi-target gene edits and/or pathway modifications the final product becomes naturally more effective and valuable, in addition to the replicable conformity & economies of scale benefits. </div>
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Allogeneic product isn't by itself the definition of a complete solution purely because it's from an immortal source & is replicable to a standard but it is when combined with the inherently more potent properties of early cell state gene expression and enhanced synthetic action/targeting... </div>
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Product is the goal and when next gen product is available early prototypes, niche offerings & more costly general alternatives will be mooted.</div>
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Profitability models favor a wider adoption formula for acute, unmet and orphan diseases where there is no effective standard of care. Pricing will vary depending on volumes.<br />
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Repeat treatment requirements are yet to be determined in many cases for cellular therapies and may factor in to longer term chronic care maintenance, more in line with existing treatment options. <br />
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The science is on the cusp of having a solid fighting chance against disease, trauma & degeneration. That is perhaps something I wouldn't have said only a few years ago. However, doubt is gone in my mind and only time is left to answer what will be the optimal mechanism to defeat each target. Knowledge is expanding exponentially and as a result I imagine within a relatively short period of time the majority of our molecular pathways and stepwise complexities of action and reaction within our cellular communities will be known.<br />
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There is a race on that has only one possible ending - the complete and total banking of knowledge of the biological process map of our physical selves.</div>
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Just as The Human Genome Project opened the doors to a world of new discoveries, the uncovered realities of cellular based mechanisms are paving the way for fundamental changes in our societies that will impact the very nature of how we live our lives. Once disease is tamed our planet will turn to sustainability to maximize the efforts of our new longevity. The full cycle may take a generation or two to occur but it is inevitable and beginning now.</div>
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Ocata is one of the many at the forefront of this new dawn, contributing with knowledge & potential. Their unique understanding, developmental expertise, early cell state technology, IP positioning and mission is what sets them apart as a sector leader and underscores what the opportunity represents as a whole.</div>
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Cheers@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-51807317953037284772014-10-17T09:16:00.004-07:002014-12-15T09:34:39.000-08:00Creation, Ensoulment, Birth & The Ethical Use of a Donated Cell<div class="MsoNormal" style="text-align: justify;">
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<tr><td style="text-align: center;"><a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjOCyv3hEZaiOq2f-dZqai2fOwJtDxowim9Sbz5aA02lcnDkc2WURNA8iDqJ3tnCz6YiVlrDErYnKWRoqvi4c2Ky2WmGvI3gmkpFvqKM7ARFf1I3dRrx7mGb2B3lv6eL6-A-hkOSfo6iHXT/s1600/Raphael-Plato-and-Aristotle.jpg" imageanchor="1" style="margin-left: auto; margin-right: auto;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEjOCyv3hEZaiOq2f-dZqai2fOwJtDxowim9Sbz5aA02lcnDkc2WURNA8iDqJ3tnCz6YiVlrDErYnKWRoqvi4c2Ky2WmGvI3gmkpFvqKM7ARFf1I3dRrx7mGb2B3lv6eL6-A-hkOSfo6iHXT/s1600/Raphael-Plato-and-Aristotle.jpg" height="265" width="400" /></a></td></tr>
<tr><td class="tr-caption"><span style="font-family: Arial, Helvetica, sans-serif; font-size: xx-small;">“<i>School of Athens</i>” by Rafael w/ Plato and Aristotle - referred to by</span><br /><a href="http://en.radiovaticana.va/news/2014/11/25/pope_francis_address_to_european_parliament/1112318"><span style="font-family: Arial, Helvetica, sans-serif; font-size: xx-small;">Pope Francis during EU Parliamentary address Nov 25, 2014</span></a><span style="font-family: Arial, Helvetica, sans-serif; font-size: xx-small;"><span style="background-color: white; text-align: justify;"><span style="line-height: 24px;"><br /></span></span></span></td></tr>
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There is a bridge between the opinions that
divide us on this subject. I have written a number of times on the Middle Way,
a Compromise if you will, that will heal the scar and abate the fear.</div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">There is an ethically sound scientific process to
allow for a donated Pluripotent stem cell from Human pre-embryos to be used for
medicine.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">The cellular process of embryogenesis has been
studied at great length. The fundamental origin of Human Life is indeed the Creation
of a pre-embryo – that Sperm and Egg moment and the days thereafter of cell
preparation for a Mother’s acceptance. This fertilization moment and the
immediate cell divisions after are central to the beginnings of Healthy Physical
Life. This understanding is universally acknowledged.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">The success of that biological union in the
combination of DNA from both parental sexes (male & female) provides the
early catalytic events that set in motion the journey to deliver a full term
baby. The process is clearly not perfect by anyone’s measure and doesn’t at all
guarantee stepwise progress nor a healthy outcome. There are a number of
important & vital moments along the way – most importantly to all is the
Mother’s acceptance of the pre-embryo into her uterus via the Implantation process
around Day 8 or 9 post fertilization.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">The period before Implantation is referred to as
the “pre-embryo” phase, as it is clear from the scientific evidence and analysis
that the cellular process of developmental Life isn't sufficiently complete to
begin the formation phase of Human Life unless these early individual cells develop
to the Blastocyst stage, are accepted and successfully embedded in the uterus
wall of the Mother. Unless that happens a woman will pass the pre-embryo cells
out of her system, as she does with the uterus walls during her monthly period
cycle. Some women never accept a pre-embryo into their uterus as a result of many
possible problems, including the genetic instability or malformation of the
combined DNA nucleuses. Some women can only conceive with IVF assistance and pre-embryo
screening help.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">IVF is not the topic here so I will leave that
for others to continue to debate its validity as this blog article is in reference to
the pre-embryo period and the possible non-destructive removal of a single cell
during this post-fertilization phase prior to Implantation.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Fertilization and the pre-embryo period is a
starting cycle without which Human Life couldn't develop, yet it is still merely
a starting signal for the remaining many phases that will confirm and define
what it is to Be Human.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">The scientific distinction is as clear as an
Egg and Sperm. A pre-embryo is the union of DNA strands ready to embark on the
journey to create a Human. A ball of intelligent cells pre-programmed to develop if the
formation is correct, the proper signals received and the internal script processed
successfully. An embryo is the resultant accepted & developing Lifeform once
embedded in the Mother’s uterus. A Fetus is an established growing person with
a beating heart and developing brain within the Mother’s womb. These stages of Life
are clearly delineated by the process itself. Science merely exposed what
Nature has already defined. The success ratio of Nature is nowhere near perfect
in this process, as naturally fertilized ovums are discarded regularly and
assisted conception ratios low.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">What is important to note here is the
difference between a pre-embryo, an embryo and an Human Fetus.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Needless to say all stages are Life and should
be duly respected.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Healthy Human Life can and does develop from a
pre-embryo after one cell is extracted for analysis and medically screened
while the remaining pre-embryo cells continue to divide and develop. The same biological
process is required for all pre-embryos to be accepted into and nurtured by the
Mother’s uterus – including the one that had a cell extracted for genetic
screening. Healthy babies are born every day using various cell extraction
screening techniques, free of genetic issues as a result.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">The confusion here is that during these past
years of discovery cells were used from leftover & donated pre-embryos
that were not needed any longer by couples that had fertility treatment to
conceive. These pre-embryos were donated and had their cells removed in a
process which terminated the pre-embryo’s development. This method of obtaining
cells via donated pre-embryos for science continues. This method of cell
extraction isn't necessary as a cell can be extracted from an earlier stage pre-embryo using a
technique called Preimplantation Genetic Diagnosis (PGD), which is used
daily by fertility clinics world-wide. It can and should be used as the method
of choice to extract a Pluripotent cell from a pre-embryo in order to screen
for genetic issues while also enabling the process of scientific discovery
& cellular science. A Pluripotent cell is not a pre-embryo nor is it
capable of developing to become a pre-embryo or embryo. The use of this PGD
process can & does maintain the integrity of the pre-embryo state and it’s
potential, while allowing for genetic screening & vital treatments for
those that suffer from disease. One does not negate the other.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">While I can appreciate the moral dilemma with
respect to those that feel a pre-embryo is a Human Life, I must differ in that the
science has proven that Human Life isn’t yet defined in these cells. There is
Biological Life of course present and the potential to develop further into a Human
but it isn’t sure whether it may or may not have the integrity to be viable nor
capacity to develop further. It isn't sure either that the Mother’s system will
accept the pre-embryo into her uterus and begin the developmental process of
formation to become what is defined by all as being Human. This process is
dependent on a number of natural system checks and balances. It has been
revealed that a successful pre-embryo must first properly create a DNA construct
and divide correctly to the point of being presentable to the Mother’s uterus.
Incorrectly formed pre-embryos don’t, in almost all cases, pass this initial
test. To say that an incorrectly formed pre-embryo that is rejected by the Mother’s
system is a Human is wrong. This would by extension apply also
to a pre-embryo that seems to have the right cellular makeup but still doesn't
get accepted by the Mother’s uterus for other natural selection reasons. There
are chemical signals within the Mother’s body that function as a viability
filter and She initiates acceptance & provides the signal environment for Human
organ development post Implantation. So unless a pre-embryo embeds in the uterus
we cannot begin to be classified as Human as we haven’t started to emerge from
that pre-embryo ball of cells and take shape. Even then the issue of the many
and correctly passaged development stages of the embryo into a Fetus lies ahead.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Our Churches, Temples & Shrines aren't the
sole purveyors of ethics and moral standing in our community. They certainly
can help us along the path but we must all accept our own responsibility to
understand and draw conclusions on the reality of our progress as a society. If
Religion is anything it is a bellwether for the community and when some
within the community need its guidance it has a duty to inform and guide based on
the best interests of the people themselves, by disseminating knowledge and
wisdom in a practical and spiritual context.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">I say this as it has long been debated whether
pre-embryo cells from donated IVF fertility treatments are sound ethical
starting blocks for medical science. Will this still be the case when the
truth is known that pre-embryos need not be destroyed and their potential
maintained?</span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">It is my opinion that to negate the progress of
medical science in assisting the creation of Life is tantamount to ignoring Man
in their quest for Love & Happiness. Especially if it doesn't harm the
potential of further Life becoming a reality. It is also important to acknowledge that while assisting Life science has opened a door for simultaneous
treatment of the sick. Life and Treatments together in one, as God intended.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Will there be criticism when medical science
proves that Mother Natures’ most important cells – the potential Life forming pre-embryo
cells – are used for the living that suffer and provide needed relief? Or will
the misunderstood concept of the need to destroy prevail while the true facts of early
Life processes, the nature of an independent cell state & its developmental knowledge be withheld from God’s
people?<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Pre-embryo cells can be taken without harm to
the potential of Life and donated to help the living. Tissue willingly provided, as is done regularly today with blood and organs. A child can be born only
when a pre-embryo is accepted by a Mother’s uterus, develops and successfully
grows to term.<o:p></o:p></span><br />
<span lang="EN-US"><br /></span>
<br />
<div class="separator" style="clear: both; text-align: center;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgx_GYE5npNdtzJHxgooQDa9wzSEtwTdWw8kpCqst27d98ktUsLf2w_T6IYYedGmm1wVMEJXiNXu8ry7fpy_ACNIU7LA0OyAFKW_cq-fjXdAXWdeDy3n0BhunaJjRE6gSTrJ5XTJgfpH3tZ/s1600/800px-Human_Fertilization.png" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEgx_GYE5npNdtzJHxgooQDa9wzSEtwTdWw8kpCqst27d98ktUsLf2w_T6IYYedGmm1wVMEJXiNXu8ry7fpy_ACNIU7LA0OyAFKW_cq-fjXdAXWdeDy3n0BhunaJjRE6gSTrJ5XTJgfpH3tZ/s1600/800px-Human_Fertilization.png" height="240" width="320" /></a></div>
<span lang="EN-US"><br /></span>
<br />
<div style="text-align: center;">
<span lang="EN-US" style="font-size: xx-small;">A day 3-4 Morula stage cell extraction does not destroy the potential to create Life. </span></div>
<div style="text-align: center;">
<span lang="EN-US" style="font-size: xx-small;">Later stage cell extractions at the Blastocyst Stage terminates the pre-embryo.</span></div>
<div style="text-align: center;">
<span lang="EN-US"> </span></div>
</div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Ensoulment is by a far the greatest gift to Human
Life – for without it we would be unconscious. Consciousness defines our Personhood,
our Self. It is the defining characteristic that allows the physical to exist
and the non-physical to be reasoned. One could say that this component of Humanity
is Divine. It certainly is one of the central tenets of Religion and for which a
valued guide in our community is sought.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Throughout the History of Christianity there
has been an overriding concept debated within the inner halls of the Institution
itself – Ensoulment and when it occurs.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">More clearly than ever today is the molecular
insight into the developmental phases of the origins of Human Life. This in
itself requires the ethical and moral concepts of the day to be reflected upon.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Terminating a pregnancy – once it is established
in the Mother’s womb – has always been an immoral act in the eyes of the Christian
Church. In certain circumstances the willful termination of a developing Life was
considered unavoidable if the Life of the Mother was at risk. The implications of
this Human Act in the eyes of the Catholic Church was Excommunication as the
punishment, in most cases. However, this was not universally true throughout
history as there was a clear separation between the ethics of the immoral act
of terminating a Life willfully and the punishment itself, if it was performed
early in the developmental cycle post fertilization.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">It seems the “Ensoulment” moment was the
dividing line. A uniformly accepted believe for most of recorded history as
occurring after a period of weeks post fertilization. Therefore the doctrine of
a Human Life’s relationship to God, beyond the potential of a developing Lifeform,
was enshrined in the Church’s teachings in this fundamental Ensoulment moment.
These philosophical discussions within the Catholic Church are a documented
series of changing positions dating back to the beginnings of the Christian
Church.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">When the issue was debated it was framed by the
obvious question of at what point in the development of a Life in the Mother´s womb
is that Life considered Human with a Soul and therefore one with God.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">That Ensoulment moment was long considered 40
days after fertilization at a minimum for a male Fetus and 90 days at the
outside for a female Fetus. The documentation dates back thousands of years to
the Ancient Greek scholars & Aristotle in 350 BC. However, even at that
time there were contradictory opinions that held the Soul entered at the moment
of conception. The teachings of Aristotle of a later Ensoulment moment became widespread
by the time of Augustine in the fifth century as the concept of stage
development of a Lifeform from vegetative to animal to Human became accepted.
Prevailing Christian thought became documented in the 12<sup>th</sup> century by
the preachings of Thomas Aquinas and the Council of Vienne to that of
Aristotle. Yet subsequent Popes differed on the doctrine of excommunication on
the basis of formed versus unformed Fetuses and the topic of Ensoulment as a
consequence. Early Church theologians attempted to bridge this divide with a
Casuistry compromise that allowed for non-ensouled Life to be regarded as
different than that of an ensouled Human Life. Since the eighteenth century the
Church has considered Human Life beginning at conception along with Ensoulment.
Needless to say this debate has raged in the Church since it’s inception as an
Institution.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">What is apparent from a lay perspective is that
the Church has adapted its own views to the popular reality of the day and considered
scientific facts, as presented. The difference between then and now is
fundamental in that today there is molecular science and it has revealed to all
the embryonic process and the value of the cell in curing the living.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">As the reality of the day becomes the norm, there
is and will be assisted births, genetic treatments and cellular science derived
from the earliest stage of Life (naturally conceived, programmed or reprogrammed).
An altered series of biological interventions by science in the process of
serving his fellow man.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">This process can be done without destroying a
naturally fertilized union between an Egg and Sperm. There are many ways to
create that don’t destroy. There is a need to accept and understand what the
difference is between the viability to create Life, the potential of cells and Human
Life itself. Without bridging the divide on the issues a meaningful opportunity
to engage and grow stronger together will be lost.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">There is no question in my mind that there is a
Duty in respecting existing and developing Human Life Implanted, Developing
& Ensouled in the womb through all possible means.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US">Medical assistance by way of Non-Destructive
cell science for the benefit of those in need is similarly a Duty and Humane.<o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<br />
<div align="center" class="MsoNormal" style="text-align: center;">
<span lang="EN-US">###<o:p></o:p></span><br />
<span lang="EN-US"><br /></span>
<br />
<div style="text-align: left;">
See: <a href="http://msemporda.blogspot.com.es/p/advocacy.html" target="_blank">The Blastomere Foundation</a> - related blog article </div>
<span lang="EN-US"><br /></span>
<br />
<div class="MsoNormal" style="text-align: justify;">
<span lang="EN-US" style="font-size: 13.5pt;">References:<o:p></o:p></span><br />
<span lang="EN-US" style="font-size: 13.5pt;"><br /></span>
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://en.radiovaticana.va/news/2014/11/25/pope_francis_address_to_european_parliament/1112318" target="_blank">Pope Francis EU Parliamentary Address Nov 25, 2014</a> </span></div>
<div class="MsoNormal" style="text-align: justify;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.vatican.va/roman_curia/pontifical_academies/acdlife/documents/rc_pa_acdlife_doc_20000824_cellule-staminali_en.html" target="_blank">Vatican hESC Declaration 2000</a> <o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.vatican.va/roman_curia/pontifical_councils/family/documents/rc_pc_family_doc_19831022_family-rights_en.html" target="_blank">Charter of the Rights of the Family</a><o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.sciencedaily.com/releases/2014/02/140213142309.htm" target="_blank">'Black Box' of Embryo Development</a> <o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3118293/" target="_blank">Nonviable Human Embryos</a><o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://online.liebertpub.com/doi/full/10.1089/scd.2013.0364" target="_blank">Totipotency</a> <o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.newscientist.com/article/mg22229670.900-in-the-beginning-the-story-of-the-embryo.html#.VEAAS7AZtuC" target="_blank">In the beginning: The story of the embryo</a> <o:p></o:p></span><br />
<span lang="EN-US" style="font-size: 13.5pt;"><br /></span>
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.cambridge.org/us/academic/subjects/philosophy/ethics/morality-embryo-use" target="_blank">The Morality of Embryo Use</a></span><br />
<span lang="EN-US" style="font-size: 13.5pt;"><br /></span></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.lexology.com/library/detail.aspx?g=00bf462b-c485-4c40-8716-c01cc1281ee9" target="_blank">German Supreme Court on Brüstle</a> <o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.lexology.com/library/detail.aspx?g=9a4015b4-1fd5-416d-9102-daa2393e9555" target="_blank">EU Court of Justice Advocate General on Parthenotes</a> <o:p></o:p></span></div>
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<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://blogs.plos.org/dnascience/2013/10/03/when-does-a-human-life-begins-17-timepoints/" target="_blank">Timepoints in Human Development</a> <o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
<br /></div>
<div class="MsoNormal" style="text-align: left;">
<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.sciencedirect.com/science/article/pii/S193459090700330X" target="_blank">Human Embryonic Stem Cell Lines Generated without Embryo Destruction</a> <o:p></o:p></span></div>
<div class="MsoNormal" style="text-align: left;">
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<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://www.eshre.eu/~/media/emagic%20files/SIGs/Stem%20Cells/Barcelona_Valencia%202010/Simon.pdf" target="_blank">Single Blastomere Non-Destructive 3rd Party Validation(Spain)</a><o:p></o:p></span></div>
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<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://humrep.oxfordjournals.org/content/21/11/2749.full" target="_blank">An ethical analysis of alternative methods to obtain pluripotent stem cells without destroying embryos</a> <o:p></o:p></span></div>
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<span lang="EN-US" style="font-size: 13.5pt;"><a href="http://books.google.es/books?hl=en&lr=&id=eNIkobX6FPoC&oi=fnd&pg=PA84&dq=Blastomere+Non+Destructive+hESCs&ots=D1AJNeQ0gY&sig=osqK-LlpjLDeSAtu2ezHXdcX_Vk#v=onepage&q&f=false" target="_blank">Stem Cells in Reproductive Medicine</a> <o:p></o:p></span></div>
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<span lang="EN-US" style="font-size: 13.5pt;"><a href="https://draft.blogger.com/Outcome%20of%20215%20IVF/ICSI%20Cycles%20with%20PGS:%20http://www.plosone.org/article/info%3Adoi%2F10.1371%2Fjournal.pone.0106433" target="_blank">Biopsy of Human Morula-Stage Embryos</a> <o:p></o:p></span></div>
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@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-5704238749444224772014-09-24T13:45:00.000-07:002015-07-13T12:01:16.968-07:00Blood & The Search For A Universal Drug Delivery System<div class="separator" style="clear: both; text-align: center;">
<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhPn51xK48NKFqjYOH9e_tQYWYYEv_6n-IqHre0Y5sU3cN1knn0G3H2xpfcdsDcuPhYIjbXOFs7WRLZ-Lo3KFQjjPDP9bp-gfBxIxuH7GdxAxVlHkqtgtsTzdr992plR77JLxHRTsExIZaG/s1600/Blood2.jpg" imageanchor="1" style="clear: left; float: left; margin-bottom: 1em; margin-right: 1em;"><img border="0" height="200" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhPn51xK48NKFqjYOH9e_tQYWYYEv_6n-IqHre0Y5sU3cN1knn0G3H2xpfcdsDcuPhYIjbXOFs7WRLZ-Lo3KFQjjPDP9bp-gfBxIxuH7GdxAxVlHkqtgtsTzdr992plR77JLxHRTsExIZaG/s200/Blood2.jpg" width="200" /></a></div>
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">The doctrine that we are all equal under God applies in reality to the very Blood of humanity that runs through our veins. This was the shocking truth that changed an age old way of intellectual & class division. After millennia Science did that not faith. The fluid of life is an interchangeable commodity that drives the body and mind. A genetic brain unique to a person's cells is common enough to be of practical benefit. Donors of all types, colors and creeds can have some siphoned off and provided to those that need a refill. </span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">We accept the DNA of another to live on, with new Blood, without question and without any harm.</span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">With 7.2 Billion humans on the planet you would think that we'd have enough of the Red stuff to go around. Think again. We don't even have enough fresh supplies to meet current demand, let alone future requirements based on the donation system. How can we expect this system to suddenly change to meet the forecasted demand when the population hits 10 Billion within a few decades and then 15 Billion a generation or two later?</span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">Enter science, as usual, to solve the human evolutionary dilemma - Create it.</span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">Not only is it possible to do so now, in inexhaustible volumes to satisfy all, but those cell products can and will be modified by the Scientists & Doctors of this 2nd Blood Revolution to deliver the needed solutions against the parasites, funguses, viruses, bacterias et al. that plague & kill daily in countless numbers.</span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">Engineered weapons of the vascular system that naturally hone in and destroy invading pathogens.</span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;"><br /></span></div>
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<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">The below provides a rough summary of how this is coming together from the perspective of ACTC and it's scientific colleagues.</span></div>
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Cheers</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Phase 1 - Blastomere Derived Renewable Stem Cell Line</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Phase 2a - Clinical Expansion & Banking of Hemangioblast Derived Megakaryocytes</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Phase 2b - Engineered Variants for Drug Delivery Requirements</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Phase 3a - Differentiation of Platelets, Red Blood Cells & Line Derivatives </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Phase 4a - Biocompatible Unit Preparation & Universal Distribution</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Phase 4b - Locally Served Fresh Product via Bioreactor Automated Production</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">________________________________________</span><br />
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<b><span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">REFERENCES:</span></b><br />
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<i style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">Cell</i><span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;"> Research Jan 2011 - "</span><a href="http://www.nature.com/cr/journal/v21/n3/abs/cr20118a.html" style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;" target="_blank">Platelets generated from human embryonic stem cells are functional in vitro and in the microcirculation of living mice</a><span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">" - SCRMI, Univ of Illinois Chicago, Cha Univ, Harvard / BWH & ACTC </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>Blood</i> July 2014 - "<a href="http://www.bloodjournal.org/content/early/2014/07/18/blood-2014-05-574913?sso-checked=true" target="_blank">Platelet bioreactor-on-a-chip</a>" - Harvard / BWH, Univ Colorado & Colorado School of Mines, McGill, ACTC</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>Vector</i> - Boston Children's Hospital Blog March 2014 - "<a href="http://vectorblog.org/2014/03/the-platelet-whisperers/" target="_blank">The Platelet Whisperers</a>" </span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>MedCity News</i> April 2014 - "<a href="http://medcitynews.com/2014/04/biochip-mimcs-body-produces-platelets-address-challenges-platelet-transfusions/%20-%20with%20underlying%20PCT%20Patent%20App%20on%20Tech:%20http://patentscope.wipo.int/search/en/detail.jsf?docId=WO2014107240&recNum=1&maxRec=1&office=&prevFilter=&sortOption=Pub+Date+Desc&queryString=FP%3A%28pct%2Fus2013%2F070910%29&tab=PCT+Biblio" target="_blank">Biochip mimics how the body produces platelets so they could be made in a lab</a>"</span><br />
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<a href="http://i1256.photobucket.com/albums/ii488/msemporda/Mazutis_300x200_zps97ddcfda.jpg" imageanchor="1" style="margin-left: 1em; margin-right: 1em;"><span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><img border="0" src="http://i1256.photobucket.com/albums/ii488/msemporda/Mazutis_300x200_zps97ddcfda.jpg" /></span></a></div>
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>NY Times</i> May 2014 - "<a href="http://www.nytimes.com/2014/05/05/science/young-blood-may-hold-key-to-reversing-aging.html?smid=tw-share&smv1&_r=0" target="_blank">Young Blood May Hold Key to Reversing Aging</a>"</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>UCSF</i> July 2014 - "<a href="http://www.ucsf.edu/news/2014/07/116431/key-aging-immune-system-discovered" target="_blank">Key to Aging Immune System Is Discovered</a>" </span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>Proceedings of the National Academy of Sciences of the USA</i>, June 2014 - Whitehead Institute & MIT - "<a href="http://www.pnas.org/content/111/28/10131.abstract" target="_blank">Engineered red blood cells as carriers for systemic delivery of a wide array of functional probes</a>"</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>The Scientist</i> Sept 2014 - "<a href="http://www.the-scientist.com/?articles.view/articleNo/40979/title/Next-Generation--Blood-Cleansing-Device/" target="_blank">Next Generation: Blood-Cleansing Device</a>" - (Engineered MBL Protein use example in fighting Sepsis - with cell engineering it can be done without dialysis)</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><b><a href="http://ir.advancedcell.com/press-releases/detail/2574/distinguished-scientists-join-advanced-cell-technologys" target="_blank">Advanced Cell Technology's Scientific Advisory Board</a></b></span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><a href="http://web.mit.edu/langerlab/index.html" target="_blank">Dr. Langer's Lab</a> & <a href="http://web.mit.edu/jensenlab/index.html" target="_blank">Dr. Jensen's Lab</a> of MIT</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>The Scientist</i> July 2013 - "<a href="http://www.the-scientist.com/?articles.view/articleNo/36099/title/Narrow-Straits/" target="_blank">Narrow Straits - Transfecting molecules into cells is as easy as one, two, squeeze.</a>" </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>Proceedings of the National Academy of Sciences of the USA</i>, Feb 2013 - "<a href="http://www.neurorexia.com/2013/05/19/blood-magic-old-blood-ages-the-young/%20-%20http://www.pnas.org/content/110/6/2082" target="_blank">A vector-free microfluidic platform for intracellular delivery</a>"</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><i>R&D</i>, July 2013 - "<a href="http://www.rdmag.com/news/2013/07/researchers-put-squeeze-cells-deliver" target="_blank">Researchers put squeeze on cells to deliver</a>"</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Dr. Daley's Lab - "<a href="https://daley.med.harvard.edu/Daley_Lab_Hematopoiesis.htm" target="_blank">Hematopoiesis Research</a>" - HHMI / Children's Hospital Boston / Havard & "<a href="http://cellnet.hms.harvard.edu/" target="_blank">CellNet</a>" - <a href="http://www.cell.com/cell/abstract/S0092-8674(14)00934-9" target="_blank">Children's Hospital Boston / Havard / </a></span><span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;"><a href="http://www.cell.com/cell/abstract/S0092-8674(14)00934-9" target="_blank">Boston Univ</a> & <a href="http://www.ncbi.nlm.nih.gov/pubmed/20888316" target="_blank">iPS mRNA Tech</a></span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">Daley / Children's Hospital Boston / Harvard Patent Families: </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif; font-size: x-small;"><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2012195862A1&KC=A1&FT=D&ND=3&date=20120802&DB=EPODOC&locale=en_EP" target="_blank">Biomechanical Induction of Hematopoiesis</a></span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif; font-size: x-small;"><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2014242046A1&KC=A1&FT=D&ND=3&date=20140828&DB=EPODOC&locale=en_EP" target="_blank">Inhibition and Enhancement of Reprogramming by Chromatin Modifying Enzymes</a></span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif; font-size: x-small;"><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2012101067A1&KC=A1&FT=D&ND=3&date=20120426&DB=EPODOC&locale=en_EP" target="_blank">Methods for Enhancing Hematopoietic Progenitor Cell Engraftment</a> </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif; font-size: x-small;"><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2011151447A1&KC=A1&FT=D&ND=3&date=20110623&DB=EPODOC&locale=en_EP" target="_blank">Method to Produce Induced Pluripotent Stem (iPS) Cells from Non-Embryonic Human Cells</a> </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif; font-size: x-small;"><a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2010278792A1&KC=A1&FT=D&ND=3&date=20101104&DB=EPODOC&locale=en_EP" target="_blank">Method of Enhancing Proliferation and/or Hematopoietic Differentiation of Stem Cells</a></span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">________________________________________</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><b>Advanced Cell Technology's Patent Portfolio for the Blood Program</b>:</span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<u><span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">INEXHAUSTIBLE SOURCE OF RENEWABLE STEM CELLS:</span></u><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><a href="http://www.ncbi.nlm.nih.gov/pubmed/23329636" target="_blank">Blastomere Non-Destructive Human Embryonic Stem Cell Technology</a> </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<u><span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><a href="http://worldwide.espacenet.com/inpadoc?DB=EPODOC&locale=en_EP&FT=D&CC=US&NR=2011183415A1&KC=A1" target="_blank">Blastomere Patent Family</a></span></u><br />
<br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">5 Granted US Patents: <a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=7838727.PN.&OS=PN/7838727&RS=PN/7838727" target="_blank">1</a>, <a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=7893315.PN.&OS=PN/7893315&RS=PN/7893315" target="_blank">2</a>, <a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=8642328.PN.&OS=PN/8642328&RS=PN/8642328" target="_blank">3</a>, <a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=8742200.PN.&OS=PN/8742200&RS=PN/8742200" target="_blank">4</a>, <a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=8796021.PN.&OS=PN/8796021&RS=PN/8796021" target="_blank">5</a></span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><u>iPS/Reprogramming Renewable Stem Cell Derivation</u> papers <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2705327/" target="_blank">1</a> & <a href="http://www.ncbi.nlm.nih.gov/pmc/articles/PMC3104759/?tool=pubmed" target="_blank">2</a></span><br />
<br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><u>iPS/Reprogramming Renewable Stem Cell Patent Family Portfolio</u>: <a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2011286978A1&KC=A1&FT=D&ND=&date=20111124&DB=&&locale=en_EP%20&" target="_blank">1</a>, <a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=WO&NR=03018780A1&KC=A1&FT=D&ND=3&date=20030306&DB=EPODOC&locale=en_EP" target="_blank">2</a> with examples <a href="http://appft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PG01&p=1&u=/netahtml/PTO/srchnum.html&r=1&f=G&l=50&s1=20120184035.PGNR.&OS=DN/20120184035&RS=DN/20120184035" target="_blank">A</a>, <a href="http://appft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PG01&p=1&u=/netahtml/PTO/srchnum.html&r=1&f=G&l=50&s1=20130104253.PGNR.&OS=DN/20130104253&RS=DN/20130104253" target="_blank">B</a>, <a href="http://appft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PG01&p=1&u=/netahtml/PTO/srchnum.html&r=1&f=G&l=50&s1=20030044976.PGNR.&OS=DN/20030044976&RS=DN/20030044976" target="_blank">C</a> and</span><span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;"> </span><a href="http://investorstemcell.com/forum/msemporda/23656.htm" style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;" target="_blank">SCNT Tech</a><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<u><span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">EARLIEST YOUTHFUL BLOOD LINE POSSIBLE</span></u><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">HEMANGIO-COLONY FORMING CELLS - <a href="http://patft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PALL&p=1&u=%2Fnetahtml%2FPTO%2Fsrchnum.htm&r=1&f=G&l=50&s1=8017393.PN.&OS=PN/8017393&RS=PN/8017393" target="_blank">US Patent Granted</a> & <a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?CC=US&NR=2012027731A1&KC=A1&FT=D&ND=3&date=20120202&DB=EPODOC&locale=en_EP" target="_blank">Patent Family</a> </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">HEMANGIO COLONY FORMING CELLS AND NON-ENGRAFTING HEMANGIO CELLS - <a href="http://appft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PG01&p=1&u=/netahtml/PTO/srchnum.html&r=1&f=G&l=50&s1=20110064705.PGNR.&OS=DN/20110064705&RS=DN/20110064705" target="_blank">App Pub March 2011</a> & <a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily?page=1&FT=D&CC=MX&locale=en_EP&DB=EPODOC&NR=2010012088A&date=20110728&ND=3&KC=A" target="_blank">Patent Family</a> </span><br />
<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;"><br /></span>
<u><span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">UNIQUE BLOOD LINE CELL PRODIGY DERIVATION:</span></u><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">(WO2011069127) LARGE SCALE GENERATION OF FUNCTIONAL MEGAKARYOCYTES AND PLATELETS FROM HUMAN EMBRYONIC </span><br />
<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">STEM CELLS UNDER STROMAL-FREE CONDITIONS - <a href="http://patentscope.wipo.int/search/en/detail.jsf?docId=WO2011069127&recNum=1&maxRec=&office=&prevFilter=&sortOption=&queryString=&tab=PCT+Biblio" target="_blank">PCT Pub June 2011</a> </span><span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">& <a href="http://appft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PG01&p=1&u=/netahtml/PTO/srchnum.html&r=1&f=G&l=50&s1=20120315338.PGNR.&OS=DN/20120315338&RS=DN/20120315338" target="_blank">US App Pub Dec 2012</a> & <a href="http://worldwide.espacenet.com/publicationDetails/inpadocPatentFamily;jsessionid=F9EA0FA3880249B26278C95E84E1AE2A.espacenet_levelx_prod_3?CC=US&NR=2012315338A1&KC=A1&FT=D&ND=4&date=20121213&DB=EPODOC&locale=en_EP" target="_blank">Patent Family</a> </span><br />
<span style="font-family: 'Helvetica Neue', Arial, Helvetica, sans-serif;">(Note: SCRMI is a JV between ACTC & Cha Biotech of Korea - North American Rights belong to ACTC - Japan/Korea to Cha - ROW split)</span><br />
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<span style="font-family: Helvetica Neue, Arial, Helvetica, sans-serif;">(WO2014100779) METHODS FOR PRODUCTION OF PLATELETS FROM PLURIPOTENT STEM CELLS AND COMPOSITIONS THEREOF - <a href="http://patentscope.wipo.int/search/en/detail.jsf?docId=WO2014100779&recNum=1&maxRec=1&office=&prevFilter=&sortOption=&queryString=WO2014100779&tab=PCT+Biblio" target="_blank">PCT Pub June 2014</a> & <a href="http://appft1.uspto.gov/netacgi/nph-Parser?Sect1=PTO1&Sect2=HITOFF&d=PG01&p=1&u=/netahtml/PTO/srchnum.html&r=1&f=G&l=50&s1=20140271590.PGNR.&OS=DN/20140271590&RS=DN/20140271590" target="_blank">US App Pub Sept 2014</a></span><br />
<br />@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.comtag:blogger.com,1999:blog-2276080694592277707.post-68582664975745750782014-05-09T12:53:00.003-07:002014-05-09T12:55:59.072-07:00Synthetic Biology & Process vrs Product - is this the Stem Cell sector's next big commercialization challenge?<a href="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhCxk-6Iq0RtaOoPICNrffaqI_4bt99R3EmQMnK1Kcy-ve6ubGAl2nspZFEe_D3QZvDgsqK8YdG5GpizY6DeqBsgCwIHDY1EUa21SXUAZ6JpxSS2Fz0svytmLcDVtWp3TvaY9dLGTVbXu73/s1600/dolly+image.jpg" imageanchor="1" style="clear: right; float: right; margin-bottom: 1em; margin-left: 1em;"><img border="0" src="https://blogger.googleusercontent.com/img/b/R29vZ2xl/AVvXsEhCxk-6Iq0RtaOoPICNrffaqI_4bt99R3EmQMnK1Kcy-ve6ubGAl2nspZFEe_D3QZvDgsqK8YdG5GpizY6DeqBsgCwIHDY1EUa21SXUAZ6JpxSS2Fz0svytmLcDVtWp3TvaY9dLGTVbXu73/s1600/dolly+image.jpg" /></a>It has been ruled on by the Supreme Court and Affirmed in Federal Appeals Court and at the USPTO that naturally occurring biological matter cannot be exclusively owned. This was today confirmed again in the matter of Roslin Institute's desire to Patent the "clones" of their SCNT cloning process. After years of examination rulings and a lengthy legal challenge, the Federal Appeals court upheld the decision of the USPTO that no Patents will be issued on the product of the process that created Dolly, as it produced a copy of a nature, and therefore "Dolly's genetic identity to her donor parent renders her unpatentable." Further, Judge Dyk stated that "There is nothing in the claims...that suggests that the clones are distinct in any relevant way from the donor animals of which they are copies."<br />
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The issue at hand is pertinent and important to note in that cells themselves are natural biological products, aren't they? Therefore they themselves, if unaltered scientifically, wouldn't be Patentable themselves therefore. The Process yes but the cell itself, if identical to the naturally occurring original, wouldn't?<br />
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Processes in the stem cell field are seemingly never ending...<br />
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The "product-by-process" standards that have applied to determining patentability by limiting duplicate product patents will need to be revisited to narrow the "product" window to only unique synthetic constructs. This developing legal framework of patent scope now calls into question old technology product claims and may give rise to a freedom to operate latitude the Courts are indicating on natural biological ownership.... or am I missing something here?<br />
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Are cells which are innovatively derived also being pushed to be genetically altered as a synthetic variant of the original as a requirement to secure a firm path to market?<br />
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Is an RPE cell derived from different innovative methods, such as hESC, iPS, SCNT, hpESCs, subject to a novelty test against an existing natural RPE cell in order to secure product claims? Will there be a race to alter the cells to create genetically unique versions in relation to the natural standard and the competitive versions? <br />
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Cheers <br />
<br />
__________________<br />
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Dolly the Sheep-type clones ineligible for patent: appeals court<br />
BY BERNARD VAUGHAN - Thu May 8, 2014 6:45pm EDT<br />
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(Reuters) - The method for cloning animals such as the famed Dolly the Sheep can be patented, but the resulting animals themselves cannot, a U.S. federal appeals court has ruled.<br />
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"Dolly's genetic identity to her donor parent renders her unpatentable," Judge Timothy Dyk wrote Thursday for the U.S. Court of Appeals for the Federal Circuit in Washington, D.C.<br />
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Pilar Ossorio, a professor of law and bioethics at the University of Wisconsin Law School, called the decision a victory for people who thought cloning animals was morally wrong.<br />
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"This ruling is taking away an incentive for research organizations to pursue more research into cloning, at least on the margins," she said.<br />
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Scientists Ian Wilmut and Keith Campbell of the Roslin Institute of Edinburgh, Scotland, generated international headlines and intense ethical debates in 1996 when they created Dolly the Sheep, the first mammal to be cloned from an adult cell.<br />
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Dolly, named after country singer Dolly Parton, was euthanized six years later after she was diagnosed with a progressive lung disease.<br />
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The institute, which owns a patent to a method of cloning called somatic cell nuclear transfer, applied for a patent over the clones themselves but was rejected by a U.S. Patent and Trademark Office examiner in 2008.<br />
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In February 2013, the USPTO affirmed the examiner's decision, saying the clones did not possess "markedly different characteristics than any found in nature."<br />
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In affirming the USPTO, the Federal Circuit said that nature, natural phenomena and abstract ideas were not eligible for patent protection.<br />
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Salvatore Arrigo, a lawyer for Roslin, said he was disappointed with the ruling.<br />
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"There's no doubt in anyone's mind that Dolly is man-made," he said.<br />
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Roslin had argued that its clones were distinguishable from their donor mammals, in part because environmental factors could make their shape, size, color and behaviors different than their donors.<br />
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The Federal Circuit disagreed, noting that Roslin itself had said that such differences were produced "quite independently of any effort of the patentee."<br />
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"There is nothing in the claims...that suggests that the clones are distinct in any relevant way from the donor animals of which they are copies," Dyk wrote.<br />
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The USPTO declined to comment.<br />
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The case is In re Roslin Institute (Edinburgh), U.S. Court of Appeals for the Federal Circuit, No. 1407.<br />
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<a href="http://www.reuters.com/article/2014/05/08/us-ip-dollysheep-idUSKBN0DO1ON20140508" target="_blank">http://www.reuters.com/article/2014/05/08/us-ip-dollysheep-idUSKBN0DO1ON20140508</a><br />
<br />@msemporda - Michael Ceahttp://www.blogger.com/profile/11414825287616436008noreply@blogger.com