Jeanne Loring - Parkinson's, Mice, DNA, hPSC & Rhinos

Jeanne Loring of the Scripps Research Institute in La Jolla California kindly sat down with me at the ISSCR annual meeting for a broad discussion of her history, views on the field and developments in the science.

I found Jeanne a refreshing character, as I did a number of others I was fortunate to met in Sweden. Her style I can only best describe as natural. It must be the Southern California air or something but there is a definite quality of relaxed confidence about her. I liked her a lot and hope to have the opportunity to meet her again sometime - perhaps at the birth of her “to be” Northern White Rhino! If she invites me :) that would be something.

The format of the interview was free flowing and what was clear to me from her long standing scientific focus and deep knowledge of the sector was it takes determination and a varied set of skills to maintain one’s position in today’s fast paced world of cutting edge science and more so even to successfully translate that to the clinic. 

Kudos to Jeanne for her efforts to continue the fight to bring forward a therapy for Parkinson's after near on 30 years and for her passion to help our planet’s most endangered.

Hope you find the interview interesting, as much as I enjoyed it.



M: Tell me a little about your background

JL: So I was trained in embryology and neurobiology, I studied neural crest cells which are actually stem cells and I was fascinated by them. Then when I finished graduate school I got a job as an assistant professor at the University of California Davis and I then realized I could either teach or do research but not both. There were not enough hours in the day, so I took the opportunity to join a biotech company in California called Hana Biologics. There were lots of them around, in 1987. I joined specifically because they were planning a stem cell therapy for Parkinson’s disease. I thought I could use what I thought was practical because I was getting a little bit tired of generating just knowledge. I wanted to generate practical knowledge which made me a little bit different from everybody else at the time.

M: Was that motivated by personal experience?

JL: No I don’t know anybody sick at all it was just I wanted to have more impact than just writing papers. I wanted to do something more important. I probably could have ended up like Rudolf Jaenisch or Austin Smith, in a way, if I was good at it the entire time but when you go into biotech it's guaranteed you'll not be at the same job for more than 5 years. I didn't know that at the time but I've learned now. So the first company I went to work for was doing a cell therapy for Parkinson’s disease but there were no stem cells yet so we were using fetal cells and we were trying to expand them. That was the heyday of the fetal transplants for Parkinson’s disease and it was clear they were working for some people, so why couldn't we take fetal cells and expand them and treat more patients.

M: I attended the pre-meeting symposium @ Karolinska on the aging brain. The eye and brain are related of course but the brain is less accessible.

JL: Yes you just have to pretty much accept that. The eye is accessible therefore it is a good testing ground for therapies. Once you put things in the brain you pretty much don't know what's happened until the person dies.

M: Why did the fetal work not continue?

JL: Yeah I'll tell you, I know exactly why. So there were two groups, sort of areas, there was Andreas Bjorklund of Karolinska/Lund and his offspring who are following in his footsteps and then there were two groups in the US who were funded by the NIH to do clinical trials. I think the people in Sweden will always argue that they had a tremendous success rate. They weren't under the same kind of regulations that were applicable in the US. They had the authority to do this under a physician kind of approval whereas in the US they were required to, or decided to, do a double blind clinical trial. You had to have negatives looking for placebo effects.

M: And surgery?

JL: Yes, I would never do that.

M: Isn't that unethical?

JL: I think it’s unethical now, I would never do it but they were doing it then. They did have the promise of treatment if they didn’t

M: You mean opening them up again?

JL: Yeah, isn't that wonderful, it’s just brain surgery. The people who do brain surgery don't take this issue as seriously as I do, but they know what they're doing. So to continue, the problem maybe 25%/30%, depending on the study, had an adverse effect that was quite dramatic called dyskinesia. So when they got the cells they had Parkinson’s disease, it was treatable with L-Dopa, but then after they had the cells they started having uncontrollable events, like the opposite of that sort of frozen kind of characteristic of people with Parkinson’s disease. They had to be treated separately and it had obviously been caused by the transplant. The question was 

M: Why?

JL: Exactly. So the whole thing stopped in about ‘92 perhaps. I remember I was at the society of neuroscience meeting when the results were reported and everyone in the room was saying we can't do this anymore

M: But 75% of the patients...

JL: Were either not helped or they were helped for the rest of their lives and went off drugs. So it was clearly a spectacular therapy.

M: This was so early, late 80s early 90s, wouldn’t you think that with more push in the science

JL: Everything sort of dropped off the map at that stage and people just didn't pursue it anymore.

M: Was it ethically charged?

JL: Yes but the question was do you want to do something to people that had a clear probability of an adverse effect because it's not a life threatening disease

M: It is a debilitating disease

JL: Yes it is a debilitating disease, that’s right. So I know that if you talk to patients they are willing to take that chance.

M: I have a relative that has Parkinson's, in my wife’s family, and it's not just the disease it's what it does to you aside from that. Your whole spinal column changes and that makes everything more painful and difficult.

JL: Everything is more challenging and you know you can never predict whether you’re going to have a good time or bad time today or this morning or afternoon. I know I have a friend that has Parkinson's and he lives in Texas and I talk to him on the phone every once in awhile and it has to be at a particular time of day otherwise I can't understand him at all.

M: Devastating disease, all the neuronal diseases are devastating.

JL: So that was the whole basis for my Parkinson's disease focus and there was a resurgence when stem cells were discovered. Obviously because you can turn them into dopamine neurons or just what you want with quality control.

M: This was the question I had at the brain symposium, why wouldn't you test the neural stem cells themselves as a method of action and let them stimulate the environment. 

JL: People have tried that. There was a whole interval where people were trying to treat Parkinson's with cells that were not dopamine neurons and actually it damaged the field because it didn't work. It turned organizations like the Michael J. Fox foundation, an organisation that had supported it, against cell therapy entirely. So they're not funding it now.

M: Were those stem cell originated?

JL: No they were neural stem cells. This was all before pluripotent stem cells. Either adult or fetal derived and those don't make dopamine, they're not dopaminergic neurons.

M: Isn't there a big difference in terms of your understanding of the pluripotent sources in terms of that?

JL: Oh yeah, of course. There’s a huge difference, the cells you get from adult or fetus have not been successfully turned into the same neuronal cell type that dies in Parkinson's. They don't seem to be able to.

M: So what about the pluripotent neural stem cells?

JL: Neural stem cells aren’t pluripotent. I use pluripotent stem cells to make neural stem cells which I then turn into dopamine neurons.

M: Ok

JL: So my next job, because that obviously failed, was to work at a company called GenPharm. This was in the early 90s and I was doing gene knockouts and mouse embryonic stem cells so I got to be at the beginning of one field and the beginning of another field. That company lasted about five years.

M: What technology did you use?

JL: Using homologous recombination which was a brand new idea. Mario Capecchi won a Nobel Prize for it. I like being in the situation where the technology I’m using wins Nobel prizes for people. I kind of think that validates it. We could just about do anything. We could knock-out, we could change genes. This was all pre Crispr-Cas, which is the way people are doing it now. So that was early days for that as well.

I made a lot of mouse embryonic stem cell lines. This was the same time as Austin Smith and Rudolf Jaenisch were using, making and knocking-out genes in mouse embryonic stem cells. That’s where our histories all sort of coincided. We were all around doing this in the early 90s. Although I was at a company. It was a spectacular five years. We did a lot of amazing work. The work I did then is still some of the most highly cited work that I have ever done. It’s amazing, for whatever reason people are still interested in the work we did then. It was really pioneering and it was fun. Then after the company failed I moved on to a company called Incyte Genomics. This was at the peak of the human genome sequencing era, which started around the mid-90s. I worked at Incyte for around 5 or 6 years learning how sequencing worked.

M: Uncovering the map

JL: Yeah and the technology was evolving very fast at that time. So I now had three things - the Parkinsons neural cell transplant idea, stem cells and now genomics/DNA.

M: This all was at the same time as the other groups but your work went unheralded?

JL: The thing if you're going to be heralded you have to stick at one field for a long time and my approach has been to learn something then learn something else and try to put the two things together.

M: and its all coming together now?

JL: It is, everything I've done is coming together now.

M: Tell me about that, because that’s why your back.

JL: Yeah, that's why I’m back, right. Parkinson’s disease - we now have human pluripotent stem cells and I can turn them into dopamine neurons, so all the things that were wrong with the early work I was doing in the late 80s I can now fix. I'm in full control - I can make the cells I want to.

M: Your independent in an academic setting now.

JL: Yeah, I'm an academic with my own lab. That is what I'm directing my lab to do and our areas of expertise. So I have the Parkinson's history and then I have the mouse embryonic stem cell history, which Austin and Rudolf share. You know it’s interesting people who come from a mouse embryonic background and then started working on human ES or iPS cells have been really pissed that the human cells don't act like the mouse cells. They're much harder to grow. So both Rudolf and Austin have been trying, and this is the focus of their lives now, to turn human pluripotent stem cells into cells like the mouse.

M: Is this kind of an internal debate?

JL: Yes whether it’s important or not to do that. Austin & Rudolf are making their careers on this. This is what they're focused on now & intensely competing with each other over whether you can make a human cell with the quality of a mouse cell. That’s because where they grew up from, and for me, also growing up like that, I just went straight to human pluripotent stem cells and realized they were not going to be like mouse and lived with it.

M: Is that because of the quality and body of evidence in the mouse?

JL: Yes, their history with the mouse. The kind of things you can do with the mouse ES cells. They're much more robust than human pluripotent stem cells. Human embryonic stem cells have to be babied, mouse ES cells you can pretty much leave in an incubator and they're fine. So that’s what they missed about it and wanted hESCs to be like that. They’ve actually moved into the direction of trying to make hESCs be like that.

M: To perform better?

JL: Yes, so that then can use all those techniques they used in the mouse and now apply them to human. So I've just bypassed that. My cells become what I want them to becomes and that’s it - I'm done. They don’t need to be mouse cells, they're human cells. To continue, while I was still at Incyte I started a company and derived a whole bunch of hESCs which were on George Bush’s original list.

M: From IVF donations?

JL: Yes, that’s right. I started a company, so I did it in my own company. There were two employees, actually there were 3 employees, as we had a manager. I was the main scientist and I had an assistant.

M: You had a number of lines. What ever happened to those lines?

JL: Well they got acquired by another company and I have no idea, they're probably still in the freezer somewhere. They never did anything with them. Its not important anymore as there are so many lines out here. There are 130 lines or so.

M: And they're sufficient to do the science work?

JL: Yeah, oh yeah. But I don’t work on hESCs anymore. I work on iPS cells because they are equivalent but you can get them from individual people. This is where my genome stuff comes in because I studied and learned all the sequencing and genomic stuff when I was at Incyte, so I had an appreciation and understanding of the methods by which you study the genome of cells, along with all the stuff I learned at different companies.

M: The translational aspect of what you're working on is focused on Parkinson's as a primary program?

JL: Yes there are a lot of reasons for it. The person who came to me with the idea to do a therapy for Parkinson's disease is actually the head of the movement disorders clinic at Scripps Health, which is across the street from me. She thought there ought to be a stem cell therapy for Parkinson's and together we did a bunch of fund raising and we've gotten enough data now. We want to do a personalized therapy, an iPS therapy, and genomics is real important as we want to have quality control

M: This is your approach to it?

JL: Yes one of the things we've learned from studying pluripotent stem cells is that they acquire mutations if they spend a lot of time in culture. So you don't want to put cells with dangerous mutations into people, so that’s where my genomic expertise comes in. Essentially the quality control of the cells we want to transplant.

M: Is that the same risk factor in regard to hESCs?

JL: Yeah they will do the same thing. In fact everything we've done shows to-date that iPS and ES cells are identical and eventually I think that will be accepted. The approach I've taken has been because I worked in genomics and have analysed lots of cell lines not just one.

M: I remember you putting out a paper on this recently that was very exhaustive - 2 years of work

JL: Yeah, 2 years of cell culturing. The scientist I worked with on that is here actually - I'm going to take him to lunch tomorrow. So the whole idea was to try and come up with things that were generalizable to cell lines and different conditions. So almost everything we've done was done on 100s of cell lines so we can come up with general principles instead of for one cell line but for 100s.

M: So if you use 1 cell line that is on the edge of a uniform grouping that will be different than if you select and productize something else within that sequence?

JL: Yes

M: and that’s where we're at?

JL: Yes, right now. Here’s the challenge. If you have different cell lines we know they'll have some diversity. They act a little bit differently, a lot due to the personal genomes of course. These are actually people that have been translated into a culture dish and they'll all act a little bit differently. The challenge we have is to develop technologies and quality control methods to allow us to know how every one of those cells becomes the same thing every single time.

M: Is that a far reaching goal?

JL: I think it's actually going to work pretty well. A lot of this has been developed already. That’s been a high priority all along. Some people think it's never going to work but I don't believe that.

M: Is RNA a part of that process?

JL: Yes absolutely.

M: So the work presented recently by Yamanaka will factor into this.

JL: Yes. So the way we make the cells is important - we’ve investigated that. How you reprogram the cells. The methods we use is harmless to the cells.

M: Is that Sendai?

JL: Yes, that’s right. It turns out we've done whole genome sequencing on a lot of cell lines and we've discovered that it’s benign. The other ones are also benign which is also important to keep in mind.  

M: So looking at this from a cost perspective, autologous patient specific treatments are highly personalized but are highly expensive. Is that part of the process strategically?

JL: Yes, that’s right. It’s part of the process. There’s a lot of discussion on personalized therapy and whether it's worth the cost.

M: Of course, if it costs $1 million dollars how many people can actually be treated?

JL: Well, more than you think. It turns out for cancer treatments the amount of money that people are paying is similar.

M: Is this about annuities, is that where we’re going?

JL: No. Somebody needs to figure out how to have insurance reimburse for stuff. Right now we're not worried about that yet. I can see how it’s going to work because the work that’s being done now with T-Cells, this is CAR-T therapeutics they’re calling it.

M: I wrote about that 3 years ago.

JL: There you go. That’s about how much it costs for what I'm doing and yet there are even companies developing this technology.

M: There are many now but there were none before.

JL: Well I remember the first time I heard about it I thought I had no idea you could do that. I didn't know the immune system could do that.

M: The immune system is a powerful force to employ.

JL: Yes it is and that's one of the reasons we’re using autologous therapy. We want the cells to be matched and don't want them to be rejected.

M: Isn't there a movement towards Allogeneic?

JL: It’s hard to say. Yes, probably because of this effort in characterizing cells but let me put it this way. As soon as we demonstrate we can make the same cells from 8 different patients and they all work then the story’s over. Autologous therapy and the price point will be worth it. It will be like CAR-T therapy. It will be what you have to do for the best possible therapy.

M: Wouldn’t you want to test an Allogeneic source?

JL: I don’t want to as others are doing it. I’m going to let them go ahead and do it.

M: and who are those others?

JL: Studer Lab in the US. Actually there’s one other Autologous therapy which is being done in Japan by Jun Takahashi.

M: Coming out next year.

JL: Yeah, his and my project are very similar. We’re trying to collaborate but it’s not going to work I don't think, as it's almost impossible to exchange things between the US and Japan.

M: Really I thought we were friends in many ways.

JL: We are but when it comes to scientific IP, it’s very hard.

M: They do want to license a lot of their underlying technology, Japan Academia, Healios.

JL: and I would be licensing it if I were a company but I don’t have to and that’s another strategy of mine.

M: Don’t they have underlying patents?

JL: Oh yeah and if I decide to commercialize what I develop I’m going to have to license patents from Japan but I'm going to worry about that later. I know a lot about patents too.

M: I remember that.

JL: So therefore I think I have a good strategy. You know the thing is you can never be certain, the science makes sense, the strategy makes sense.

M: To be clear on this patent issue - you don’t need a license to do clinical trials.

JL: No. I don’t need a license to develop anything, as long as I’m an academic and I’m not commercializing it,

M: The concept is Japan Academia is licensing for research purposes because they deliver a package.

JL: Yes - and I don’t need it because I’m a non-profit. They will license their technology for research purposes to companies but they won't let anybody sell the iPS cells. They will just let people generate iPS cells but they charge a lot of money

M: The process of development can happen independently using Sendai without a license.

JL: Yes that’s correct. This is something I know a lot about as I was in biotech and I was affected rather negatively by patents at times. I remember this so clearly I was arguing with one of the WARF patent attorneys, so my friends wanted to challenge the WARF patents. I think we were pretty much challenging for the whole time and the patents finally ran out. They expired just before we lost. The fundamental patents are gone and I challenged the patents because they essentially closed my company. I became an academic, as I like to say to them as many times as I can, so that I would have the freedom to operate and not require a patent from them to do embryonic stem cell research. So as a result of our challenge, very interesting and very educational, we got them to change the claims of their patents. Their fundamental patent on all embryonic stem cells. Originally the wording would have also included iPS cells, so they could have retroactively…

M: There are a few words in there which are specific to embryo derived.

JL: We made them put those in. What happened was we challenged the patents, the patent office rejected all of them, and in order to get the patent office to reinstate them they had to change the language for their primary patent.

M: It was their intent in the first place.

JL: Oh yeah absolutely, that was the strategy but as a result of that they lost any claims to iPS cells so this is a subtle victory and it was a lot of fun. I met some really great people as a result of the patent challenge. The attorney that was with me was the same attorney that brought the challenge to the Myriad patents. He was at the Supreme Court for that and I went to the Supreme Court and got to watch them.

M: Interesting isn’t it

JL: It was fascinating

M: The legal system is a world apart

JL: It’s so bizarre

M: Somewhat like the science world?

JL: No, well in theory yes. What really struck me was the fact that the Supreme Court justices, and I think lay people, need metaphors in order to understand science.

M: They do and communication from science to the world is vital as the boxed view of old school scientists just doing experiments to publish needs to change.

JL: I don’t know why people would do that to tell you the truth.

M: I think there is a value. My curiosity brought me here. So I think there’s a great value in curiosity and the maintenance of that throughout your life

JL: I agree.

M: I like that analogy the Salk Professor, Rusty Gage, presented. He spoke of a running experiment where brain cells develop due to vitality, even in disease states. I’m not sure how a Parkinson's patient can actually get on a treadmill but.

JL: They actually can, this is the cruel thing. Our funding mechanism is patient advocacy based, the people raise money for us.

M: I’m not aware of your funding mechanism.

M: I remember that now - you just recently you did a drive. How did that go?

JL: It’s gotten enough money to get us to the pre-IND stage.

M: Did CIRM ever come in?

JL: CIRM will come through the next round, I believe. We're certainly going to apply for CIRM money. We've been working up to this for a long time. The patients have been going to CIRM meetings so we can educate the panel about the importance of this.

M: How far are you away from the IND

JL: About 2 to 3 years

M: That’s pretty similar to some of the others - Malin Parmer for instance mentioned 2018. Of course the Japanese are coming on fast next year.

JL: They’re on a fast track - there are some positives and negatives about that.

M: Tell me about that. There is I guess a Japanese societal push, an industrial push. They have a tendency to like to do that in industry and it’s been beneficial in the past. Do you see that as a mechanism to dominate?

JL: Oh yes absolutely - that’s why they're doing it.

M: and will it open up things potentially or will there be a downfall?

JL: The good thing as far as I’m concerned, if Jun Takahashi gets his therapy through the regulatory agency, he´ll get to transplant his cells to people and they'll be doing that before I do. It’s quite clear our FDA will not let me do it sooner. So if nothing bad happens to the patients then that will help me. However, if somebody else, because of this fast track, gets on this and there are scientists that are not as careful as Jun Takahashi there could be issues. Stem cell therapy, just like everything else, if it is strongly promoted can have setbacks. In Japan we saw what happened with the STAP problem. As soon as there’s a lot of pressure from the Japanese society and Government to move forward there are going to be people that make mistakes. There will be people that are not careful.

M: Masayo Takahashi is trialing iPS cells.

JL: Yes and she has published preclinical work. Essentially she is trying to show equivalency of iPS to ES cells. I think that´s very important, as the FDA is still very worried about iPS cells.

M: They are it seems. There was some planning to file an IND for Platelets.

JL: Yes.

M: Do you know that story?

JL: I do, yes.

M: Will you tell it?

JL: I probably can. I can publicly say I was a consultant for them [Ocata/ACT] and that I attended their pre-clinical meeting as a consultant. So I know a lot about the reaction of the FDA about that.

M: This was back 3 years ago?

JL: Yeah I think 2 to 3 years

M: 2013

JL: Yeah, and I wasn’t really necessary. I didn't say a word but it was fascinating to see the FDA’s response.

M: The concerns they had were GMP compliance related.

JL: Yes, but they’re getting over that. They’ve approved ES cells which were accidentally made. I mean nobody thought about using them for therapy. So one of the cell lines that they approved was derived in 1998 using bovine serum. They’ve approved something that is far less qualified than the iPS cells we're making now.

M: Yes even the Ocata/ACT cell line comes from some time ago on MEF.

JL: Yeah and I’m not concerned about that. There are some issues with using xeno reagents but they are really related to whether you’re making the cells make the wrong kinds of sugars. That’s essentially all it is. If you were to put mouse embryonic cells or mouse iPS cells on MEF they would get viruses and would be transferred into the cells but since they’re not human viruses they don’t affect human cells. Nobody has ever shown any kind of viral induction.

M: That’s why the safety is intact.

JL: Yeah exactly. Even if that should happen and it doesn’t, you just have to accept that it doesn’t.

M: It’s only a transitory process as well.

JL: Yeah you’re not putting mouse cells into people. But you’re also not infecting the cells so there’s no lasting change.

M: There are a lot of other technologies that are far more dangerous - virus delivery for instance.

JL: Oh yes of course and people are using viruses and they’ve actually been dangerous and demonstrated to be dangerous.

M: There have been a lot of adverse events and people have actually died in the CAR-T trials and no one talks about it.

JL: Yes that’s right.

M: My feeling is the xeno movement is a good thing for standardization and some of the work shown here, the BioLamina work and the Thermo Fisher work, these are very good protocols that need to be adopted and the expansion occur.

JL: That’s fine as long as they are necessary and they work well I don’t really care. I’m not too worried about the xeno issue because the cells won’t be inter species  

M: Are they requiring it now, is that the new standard?

JL: Not yet, no. We just had a meeting with our regulatory consultant a couple of weeks ago and of course you never know.

M: So when the Israelis and Biotime and others in the field are touting the xeno-free, it’s just marketing?

JL: I think so, mostly yeah, don’t let's pull it too far, I mean the FDA is never really predictable and they could suddenly think it’s dangerous to have xeno reagents.

M: But the products will be approved without a line switch.

JL: Right, that’s right.

M: What are the programs in the pluripotent space you think will reach the market within the next 5 years?

JL: Obviously there’s a lot of interest in the reagent business and there are a lot of companies that are joining in, especially in Japan. I really didn’t realize how much was involved in Japan in creating reagents for taking the cells to the clinic.

M: ReproCell?

JL: Yeah, ReproCell and there’s this other one.

M: Takara?

JL: Yeah. I’m doing an innovation showcase tomorrow. One of my friends asked me to do it for him because he can’t make it here and it's for another Japanese company. I still have to do my research on them but apparently they’re a very large chemistry company. They’ve been in business a long time and now they want to start to apply what they’ve done to stem cells.

M: Interesting how some of the non-scientific power houses in Japan are involving themselves now. A change in strategy perhaps. Digital is affecting their main lines of business and there’s an opportunity.

JL: Oh yeah, absolutely. They have a lot of bandwidth. Fuji Film just bought Cellular Dynamics. I mean you’re not making film anymore so you might as well make stem cell reagents.

M: I don’t want to press on the point of who’s going to come to market soon but success needs to be translated and your view on early access as being a component of adoption, proof of clinical concept, is that in your view an essential part of regulatory review & language?

JL: Yes, I think so absolutely. I think one of the problems with the way that Japan has fast tracked is that our FDA has not been fully appreciated. I think they’re not going to fully adopt because the Japanese regulatory authorities have decided to trust their scientists a lot more than the FDA trusts us.

M: In my view Japan also has a symbiotic relationship with the other parts of the system, you know, it works all together - the insurance, the legal, the funding, the university/academic and business community.

JL: I know, it’s really amazing. I’m really envious.

M: Anything else on the program front? I wanted to talk about the Zoo work.

JL: I know that’s what you actually wanted to talk to me about.

M: Yes, it was fascinating that you’re doing that work.

JL: Yeah, we decided that we could reprogram endangered species cells so in 2011 we published a paper to say we can - Rhinos, using human technology. That sort of just sat there for awhile.

M: The egg/sperm combination?

JL: All that stuff is coming. At that time we just made iPS cells from the animals and it sort of lay dormant for awhile because the zoo we were working with thought it was a little bit creepy, too Jurassic Park. They've now they decided to embrace it.

M: Weren’t there a number of international groups working on that before?

JL: Yes but not a lot, not in this particular thing. There was a group in Australia that was trying to reprogram other animals.

M: Interestingly in Spain also

JL: Yeah that was different though, replacing one animal with another.

M: Using an animal to host?

JL: Well they’ve done that too. Yeah, the essence of our approach with endangered animals is to take the pluripotent stem cells and to use the technologies that has been developed for mouse and for humans and make gametes out of the cells. Then use IVF technology, that is also in development for these animals, and have a surrogate host that we’ll be able to put the embryos into and regenerate the species. Just a small thing! I’m doing it with the Northern White Rhino, as there are only 5 of them left. There’s only one male and the females I think, with one exception, are beyond reproductive age and they’re dying.

M: So how would you approach that?

JL: The Southern White Rhino. It has a very similar reproductive cycle. The Northern White Rhino are a different species but they’re very similar. No one really knows if you can cross them yet, which is kind of interesting as I would have thought they would have figured that out by now but no. So we want to make the gametes from the Northern White Rhino, inseminate the eggs in a culture dish and transfer the embryos to a Southern White Rhino.

M: The egg would come from the endangered species also?

JL: Yes, from the pluripotent stem cells.

M: Both gametes.

JL: Yes both gametes would be produced from iPS technology.

M: Have you proven that yet?

JL: No, we had no money, so we essentially have been generating more iPS cells and we’re getting better at it. But now there’s going to be an investment, that’s what they tell me. The zoo has decided.

M: Which zoo is that?

JL: The San Diego Zoo Wild Park

M: Wonderful.

JL: They’ll be announcing that when they feel like it, it’s not up to me.

M: Very good, I wish you luck with that.

JL: Thanks, it’s one of those things which stuck out.

M: Well worth doing.

JL: Yeah, it is worth doing and it was just a matter of timing. I think a lot of this is like that. If you’re too ahead of people’s understanding of what you’re doing then it will just sit there dormant until they understand it.

M: I think in most fields if you’re pioneering something you have a responsibility to educate.

JL: Yes but people also have to accept it. I’ve seen a lot of changes in the stem cell world since we started this in 1998 so it’s been awhile.

M: The %s are way higher now.

JL: Yeah they're higher, that’s right, and it does have to do with education and our patient advocacy approach means we educate patients and the patients educate other people because they’re motivated. They may have not wanted to be scientists but they’re driven into it because they want to get cured, they want to get treated.

M: Thank you Jeanne

JL: Ok 


Ref: Parkinson's review by EuroStemCells