Blood & The Search For A Universal Drug Delivery System

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. 

We accept the DNA of another to live on, with new Blood, without question and without any harm.

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?

Enter science, as usual, to solve the human evolutionary dilemma - Create it.

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.

Engineered weapons of the vascular system that naturally hone in and destroy invading pathogens.

The below provides a rough summary of how this is coming together from the perspective of ACTC and it's scientific colleagues.

Cheers

Phase 1 - Blastomere Derived Renewable Stem Cell Line
Phase 2a - Clinical Expansion & Banking of Hemangioblast Derived Megakaryocytes
Phase 2b - Engineered Variants for Drug Delivery Requirements
Phase 3a - Differentiation of Platelets, Red Blood Cells & Line Derivatives 
Phase 4a - Biocompatible Unit Preparation & Universal Distribution
Phase 4b - Locally Served Fresh Product via Bioreactor Automated Production

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REFERENCES:

Cell Research Jan 2011 - "Platelets generated from human embryonic stem cells are functional in vitro and in the microcirculation of living mice" - SCRMI, Univ of Illinois Chicago, Cha Univ, Harvard / BWH & ACTC 

Blood July 2014 - "Platelet bioreactor-on-a-chip" - Harvard / BWH, Univ Colorado & Colorado School of Mines, McGill, ACTC

Vector - Boston Children's Hospital Blog March 2014 - "The Platelet Whisperers" 

MedCity News April 2014 - "Biochip mimics how the body produces platelets so they could be made in a lab"

NY Times May 2014 - "Young Blood May Hold Key to Reversing Aging"

UCSF July 2014 - "Key to Aging Immune System Is Discovered" 

Proceedings of the National Academy of Sciences of the USA, June 2014 - Whitehead Institute & MIT - "Engineered red blood cells as carriers for systemic delivery of a wide array of functional probes"

The Scientist Sept 2014 - "Next Generation: Blood-Cleansing Device" - (Engineered MBL Protein use example in fighting Sepsis - with cell engineering it can be done without dialysis)

Advanced Cell Technology's Scientific Advisory Board

Dr. Langer's Lab & Dr. Jensen's Lab of MIT

The Scientist July 2013 - "Narrow Straits - Transfecting molecules into cells is as easy as one, two, squeeze." 

Proceedings of the National Academy of Sciences of the USA, Feb 2013 - "A vector-free microfluidic platform for intracellular delivery"

R&D, July 2013 - "Researchers put squeeze on cells to deliver"

Dr. Daley's Lab - "Hematopoiesis Research" - HHMI / Children's Hospital Boston / Havard  & "CellNet" - Children's Hospital Boston / Havard / Boston Univ & iPS mRNA Tech

Daley / Children's Hospital Boston / Harvard Patent Families: 

Biomechanical Induction of Hematopoiesis
Inhibition and Enhancement of Reprogramming by Chromatin Modifying Enzymes
Methods for Enhancing Hematopoietic Progenitor Cell Engraftment 
Method to Produce Induced Pluripotent Stem (iPS) Cells from Non-Embryonic Human Cells 
Method of Enhancing Proliferation and/or Hematopoietic Differentiation of Stem Cells
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Advanced Cell Technology's Patent Portfolio for the Blood Program:

INEXHAUSTIBLE SOURCE OF RENEWABLE STEM CELLS:

Blastomere Non-Destructive Human Embryonic Stem Cell Technology 

Blastomere Patent Family

5 Granted US Patents: 1, 2, 3, 4, 5

iPS/Reprogramming Renewable Stem Cell Derivation papers 1 & 2

iPS/Reprogramming Renewable Stem Cell Patent Family Portfolio: 1, 2 with examples A, B, C and SCNT Tech

EARLIEST YOUTHFUL BLOOD LINE POSSIBLE

HEMANGIO-COLONY FORMING CELLS - US Patent Granted & Patent Family 

HEMANGIO COLONY FORMING CELLS AND NON-ENGRAFTING HEMANGIO CELLS - App Pub March 2011 & Patent Family 

UNIQUE BLOOD LINE CELL PRODIGY DERIVATION:

(WO2011069127) LARGE SCALE GENERATION OF FUNCTIONAL MEGAKARYOCYTES AND PLATELETS FROM HUMAN EMBRYONIC 
STEM CELLS UNDER STROMAL-FREE CONDITIONS - PCT Pub June 2011 & US App Pub Dec 2012 & Patent Family  
(Note: SCRMI is a JV between ACTC & Cha Biotech of Korea - North American Rights belong to ACTC - Japan/Korea to Cha - ROW split)

(WO2014100779) METHODS FOR PRODUCTION OF PLATELETS FROM PLURIPOTENT STEM CELLS AND COMPOSITIONS THEREOF - PCT Pub June 2014 & US App Pub Sept 2014

Tools & Therapeutics - How the Industry is Lining Up Stem Cells – A Sector Overview from Bio-Europe Spring 2013, Barcelona

It seems odd to debate the merits of scientific progress and value when people are dying and illness remains the single biggest impediment to human advancement. Well that’s just the reason for Biotech – right? To investigate the possibility of new approaches to treating human suffering and to trial them so they are fit for acquisition and release – or at least that’s what the dogma is supposed to suggest. However, as I learned last week in Barcelona all is not clear with the establishment and the dawn of a new era may be beginning to take shape….

What is it that is driving this movement? Well, like all good stories it revolves around plot, and the storyline may well be all about action. It’s happening all around us… The business of health is moving towards a sustainable path, ever so slightly, not so much that you or I can see it – but it’s there, inching its way to a new paradigm. It’s the gradual understanding that our societies can’t manage the onslaught of the sick and elderly of an ever increasing population without adapting and innovating our way out - like we have always done. We’re good at it, in most senses, but it does take time… History has proven that, at least in some ways – so there’s hope – or at least one would like to think so…

One theme at EBD Group’s annual Bio-Europe Spring conference this year was the emerging field of Stem Cell Technology and its application and relevance to today’s Pharma structure. The promise of a revolution over 15 years in the making since ES cells were first discovered has largely fallen on deft ears in the power corridors of Pharma. However, patients still maintain hope for medicines that offer relief, as well as potential restorative and curative benefits without toxicity. The combination of economic incentive, innovation and patient advocacy has thickened the plot – can the real action be that far away now?

I sat in on many related Pharma Positioning speaches, Panel Session Talks and Corporate Presentations and my takeaway was cautious acknowledgement of a Regenerative Medicine revolution. From J&J’s announcement of their London Innovation Center, CIRM’s $30m iPS cell bank initiative and its` European and Japanese counterparts, to Baxter, Teva and Shire’s intent to secure leading positions in the emerging field. Even the handful of top names’ desire to further harness the power of cell technology, were all evident on the scene. What was lacking was a deal announcement in the space to signal the segue.

iPS cells have done what hES couldn’t in less than half the time – entered the protected walls of the establishment. From what the speakers described, that was a hard fought battle in itself, yet four years and a Nobel later the sector has a home within those castles. Cell Therapeutics – the bedside “Apple” to the lab juiced “Orange,” as CIRM described it, is still a bit player, but that may be changing as companies in the space are advancing their proof data and that is what many noted will set the action off and usher in new stars.

Which therapeutic companies are positioned to drive the field? – for it is that progress and those breakthrough programs that were viewed as needed, along with Pharma`s subsequent buy-in. These companies will be the catalysts to the new business dynamic that has not been seen in the Industry since the early days of monoclonal antibodies a generation ago.

Enter two US Stem Cell companies that participated in the conference and were notably featured on related technology Panel Sessions – ImmunoCellular Therapeutics (IMUC) and Advanced Cell Technology (ACTC) – with Novartis and Roche on their Session Panels respectively. Both have great programs and potentially breakthrough commercial potential. One is the natural follow-up to Dendreon’s pioneering science in the cancer space, IMUC, and the other is the leading ES and iPS player, with a curative Eye therapy reaching its inflection point and a multiple cell source derived Platelet trial lining up, ACTC.

I had the distinct pleasure to meet with ACTC’s CEO Gary Rabin and Matt Vincent, the company’s Business Development Director on the final day of the conference. We chatted for an hour and a half on a wide range of topics of interest to current and future shareholders (report here). The rain outside the hotel lobby on the day was heavy and soothing, as was the conversation. To be on the brink of solving such a devastating disease as age related blindness at a time when the ranks of the elderly are swelling and the social burden of maintenance is so high, is worth waiting a few months for until the final action packed Act…

Word of mouth is a powerful tool – let’s hope no one has lost the plot along the way…

Cheers