The Future of Regenerative Medicine




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Video title: The Future of Regenerative Medicine
Released on: January 13, 2009. © PharmaVentures Ltd
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As President of the world’s largest funding source for regenerative medicine Alan Trounson draws on his experience from his long career in research which extends back to the very beginnings of this groundbreaking area. He talks of his personal career and the birth of stem cell research and explains how the Institute will help to ensure that the science fulfils its potential.
Origin of California Institute of Regenerative Medicine.
Fintan Walton:
Hello and welcome to PharmaVentures business review here in Melbourne, Australia. On this show we have Professor Alan Trounson who is the President of the California Institute of Regenerative Medicine based in San Francisco. Welcome to the show.
Alan Trounson:
Hi.
Fintan Walton:
The California Institute of Regenerative Medicine is a relatively new initiative that came out of basically the California politics.
Alan Trounson:
Yes.
Fintan Walton:
Proposition 71 which allowed the state to start funding basic stem cell research effectively and now you head up this institute which has about $3 billion worth of funding
Alan Trounson:
That's right. $300 million a year for around about 10-years and because what we are dealing with alliance it might send a little longer than that say 12 or 14-years.
Fintan Walton:
Right, so what exactly does the California Institute of Regenerative Medicine do?
Alan Trounson:
So it's a funding agency. So it's there to " to really address the mission of the of the institute which is embedded in in the proposition 71 and it really is to take the discoveries that are in stem cell science, the stem cell biology through to the clinic that is to deliver treatments to the patients and so in that time frame which is a very short time frame for those developments you know we are required to establish a number of clinical treatments.
Funding on research facilities in california
Fintan Walton:
Right, now clearly trying to do something like that requires many different tactics required in into developing that strategy. So funding, putting money into research is one thing, creating in actual laboratories in specialized area. So what is what's the money actually been spent on?
Alan Trounson:
So initially it was spent on training grounds so that to get the new people in, before that there was a reticence for young people to go in that area because NIH wasn't supportive even though it was the US Administration. So people were a bit nervous about committing their you know their whole life to an area that may not be supported and so once the once it was established we needed to help people into that area by training grants, by funding established researches in comprehensive grounds and building the faculty, building the personal resources so we did that and I think there is about 168 or 169 scientist that are that are supported. In addition to that we felt that we needed to build space, institutional space and that was limited under proposition 71 to around about 280 million. We couldn't spend more than that on bricks and mortar. So we what we did was we called for grants from the institutions, they applied to us, we " we had selected 12 of them and also said to those institutions that we were looking for a leverage for our dollars which suggested that should be 20% or more turned out to be 200% so they raised $540 million and then the institutions themselves put in 300 millions so they may get a billion dollars for 12 new institutes that have to be built within two years.
Fintan Walton:
And these are based where?
Alan Trounson:
All over California all in the major major cities but in some of the peripheral areas as well.
Fintan Walton:
But in in some of the universities Stanford university [PharmaDeals ID = 31033].
Alan Trounson:
Yes Stanford, the University of California [PharmaDeals ID = 32646], the University of Southern California, Los Angeles, San Diego, Sacramento and San Francisco.
Fintan Walton:
Well relatively speaking in California in the USit's been slight behind, we are here in Australia with where a lot of this early stage work was done, lot of that work goes from the UK and elsewhere. So did that mean also bringing people into California?
Alan Trounson:
Well because when you get a such an initiation of such a major granting opportunity then the institutions go looking for some of the really key personal in the space, so they reached down to Australians and people in other places and took some of the best scientist to really populate this area. So yeah it was it was quite predatory on their capacity of countries like Australia.
Grants for biotech companies by CIRM.
Fintan Walton:
Sure, now clearly California is very famous for it's biotechnology industry.
Alan Trounson:
Yeah.
Fintan Walton:
That is always been venture backed largely venture capital backed some government funds available but largely venture capital funded, this is totally different, this is totally new and one would argue that this is a different commercialization modeled to the one that traditional goes with biotech?
Alan Trounson:
I think that's correct and while we are been initially powering up the not for profit institutions. We're now into the phase of supporting the biotechnology companies so we are entering that translation and clinical team teams programs where we've actually got to take the early discoveries and drive them through to the clinic, best done by the biotech companies, so we are coming in with grants but preferably loans to these biotech companies that that will create the initiative but if commercialized we get some money back to to reinvest in the area.
Fintan Walton:
Right, so so you'll see some of the funding, supporting work in some of the biotech companies
Alan Trounson:
Yes.
Fintan Walton:
Some going back into the research. So clearly the commercialization it's still a very early stage, a lot of the work is still in the discovery pre clinical level haven't yet moved into clinical trials?
Alan Trounson:
Well some of it's already in clinical trials but it's the more advanced cell types, these are bone marrows cells, (indiscernable)stem cells, the code blood cells. They are all in clinical trials and so are some of the neuro stem cells, adult stem cells are being used for clinical trials. The work on the embryonicstem cells or the pluripotential stem cells, that is still coming forward in the clinical trial but the company Geron has an application into the FDA they have been considered and they are addressing some of these issues that the FDA, FDA have had and so they are on the move and other companies in California are also proposing to make applications for clinical trials.
Fintan Walton:
So as part of the remet within the California Institute of Regenerative Medicine clearly one thing is to fund research but other is to make sure that the regulatory environment is there as well presumably that is as we just talked about the FDA doing this.
Alan Trounson:
Yes, so that's a really important component part and it's being difficult to, to find out for cell therapies you know what are the what are the principle issues that you have to deal with and the companies really have to work very hard with the FDA to do this and since there has been a culture of working with adult stem cells some of it's known but because these are embryonic and they contain cells that can produce Teratomas there is certainly some some considerations which are broader than using the more developed adult stem cells. So they have to be solved and they will be solved. one would think that the the incoming president will be much more supportive and I think we will get traction on what's needed there.
Network of bringing innovations to california
Fintan Walton:
Right, now we've already eluded to the fact that the science is not just only based in California it is elsewhere. So what about the other regions of the world? where how does the California Institute of Regenerative Medicine ensure that those technologies, those inventions, those development of innovations are brought back into California?
Alan Trounson:
Well I think the you know we because it's bond money that that it's driving the initiative in California we are required to invest that money in California. But that means that we can't fund outside the California yet there are very good scientist as you know all over the world but particularly here in Australia. So what we decided to do was to invite if there was an interest to invite other countries that have a funding arrangement to join us so that if their scientist say Victorian scientist applied with the California scientist and it was ranked high in the assessment a round of evaluation and recommended for funding that we would pay for the Californian and then the Victorian Government [PharmaDeals ID = 32815] for the Victorian component and that's what's actually happening at the moment and we are also now because this is it's such a strongly motivating opportunity for Victorians suddenly to be partners now with the front line of American Research plus the biotech technology companies that are often linked in there this is just a fantastic opportunity to really push you know push the opportunity for for what ever region in the current case it's for Victoria I think it will spread to Australia. The Federal Government is really very interested in seeing if they can pursue that but we've also got arrangements with Canada in, Cancer, stem cells with the United Kingdom through their medical research council and we've also got foundation such as the Juvenile Diabetes Research Foundation already signed up another countries in discussion. So it's kind a big network if you like of researches who trust one another, work together with one another, who will accelerate this I believe, will really accelerate the opportunity but here is also a very good opportunity for the biotech companies to be part of that network.
New paradigm in driving opportunity for pharmaceutical industry
Fintan Walton:
Right, and traditional pharmaceutical companies stem cell therapy based therapy is something that's not quite normal pharmaceutical activity. Are we seeing the pharmaceutical industry itself responding to this opportunity at all?
Alan Trounson:
They are responding to the use of stem cells for drug drug design, so they are already drawing that component in-house.
Fintan Walton:
Sure.
Alan Trounson:
And they are also using you know, where scientist are using it for stem cells for identifying new drug candidates so they are in that but when it comes to thecell therapies it's very difficult for them and the investors to see what the business model is.
Fintan Walton:
Sure.
Alan Trounson:
I mean if you are working to a cure which is simply an injections of cells for curing diabetes as a once injection in an outpatients clinic it's hard to see how you can recover you know half a billion dollars of investment to take it through the clinical trials. So there is a real problem here and and this one that we've recognized and we are working towards finding the solution.
Fintan Walton:
And what is that solution? Is that driven by? Other drivers have to drive this through?
Alan Trounson:
Yes, look I think you have to look at who are the beneficiaries for cures and they are the the government if they are funding that funding health in Australia for example. But there are also the health insurance agencies inside the US who are the big that they're funding a lot of the healthcare there, so so it's in their interest to to have cures, if they were still looking out to patients with polio that would pay be paying $24 billion a year just to look after those poor people in (indiscernable) lungs or whatever that was, that doesn't exit so you can " they can recognize there is benefit here if we for example cure diabetes. So the idea I am hoping, we hope the idea is that they will form a fund a fund that they were investing to drive that. So they so what are you asking is the beneficiaries, the government and health insurance agents to drive that. And I I think there is a lot of interest in that and certainly no negative, we haven't received really any negative news about that.
Fintan Walton:
But it's a it's a new paradigm.
Alan Trounson:
It's a totally new paradigm and I think it's one which otherwise will fund all this work up to the Phase I clinical trial and then I have no capacity to take all of these new developments through the clinical trials. So it's an important road block if you like and it's one of the things that an institution like us has to foresee and has to work through it.
Founding of stem cell international.
Fintan Walton:
Yeah, I mean clearly stem cell therapy will be a major player in the future has to happen, now you as an individual got into this many, many years ago based here in Melbourne and people often ask you know what was it that helped to create the opportunity here in Melbourne and here we have you know developed a good presence in the cell area, tell us tell us your story?
Alan Trounson:
Well I was involved in the development of IVF In vitro fertilization and that was just Serendipity where there I was asked to come back to help Professor Carl Wood develop that area and I had some skills which translated very well and so it worked really well that the current IVF procedures are based on those things that we developed. So I took a further I had further interest as a scientist in understanding how a group of undifferentiated cells become a whole organism like human being, how does that happen? So I wanted to I wanted to look at to integrate this whole process. So I got interested in stem cells, in human embryonic stem cells because I thought they would tell me how this undifferentiated cells may neuron, muscle, liver and so was really about, at that point of time is about you know looking at it is is a way to understand the development of process and maybe give you some answers on birthday fix and childhood cancers as it turned out it was a lot larger than that, and so I did a sabbatical leave in Singapore with colleagues there, Professor Eric Bongso and we developed it with colleagues in Melbourne that I one of them I brought from Oxford who joined me and one from Israel who joined us and they made it all all work and so we were in a we were in a very advanced position when when the Americans it was Thompson, Jeremy Thompson had that now made this announcement and so we, we then delayed our publication while to sort of take the work a little further but we were right up there in the front. So so that was just in a way Serendipity but because we are interested as a scientist it can happen.
Fintan Walton:
Right and out of that came stem cell international is that right?
Alan Trounson:
That's right, so well I didn't have any in there " that there were nobody was a very big believer in this is a " as a possible therapeutic. So I was basically doing this work with my colleagues without any funding. So so I asked a group of builders friends of mine who are here in Melbourne here if they would raise a million dollars which they did but the Singaporean Government where we were doing some of the work they wanted to be part so suddenly we had $5 million and a new company ESI and suddenly we're had some funds to do the kind of work we are doing and it of course it lifted off and eventually three of us primarily Dianna DeVore, Robert Moses and myself argued the case for astem cell Centre for Excellence which initiated the Australian stem cell centre.
CIRM and the role of Independent Citizen Oversight Committee.
Fintan Walton:
Right and now you are a President of this California Institute of Regenerative Medicine which is got $3 billion to spend over the next 10-years?
Alan Trounson:
Yeah.interesting.
Fintan Walton:
Did you think that would happen?
Alan Trounson:
No, never. now but I think part of it is luck isn't it then part of it is you are interested in and the work takes you places and things happen around just because you are interested.
Fintan Walton:
Right, I mean I think one of the things that you know by the California Institute of Regenerative Medicine is sometimes we forget that actually in America there is very strong governmental support or at a state level.
Alan Trounson:
Yeah.
Fintan Walton:
You can get that basic research of the ground and that then gives rise to commercial success later on?
Alan Trounson:
I think that's right and there is a very can do attitude in California, I can't really speak for some of the other states. But " but if they really want to do something they are able to persuade government, they are able to persuade the community and those who are interested in you know in technology to sort of come together and I think they help one another out in this process and at the time when you know you build through this whole of the silicon valley you know wonderful huge development really came as a process of people helping one another to do that but believing in it when others said well, wow, but no thank you and so so there is a can do attitude in California and for me with a bit of rebellious attitude and the caring for wanting to do something's that " that really matter, there is a terrific opportunity so when Bob Cline offered the job to me first of all I couldn't understand why it had come to get in Australian but hey something like that only happens once in the life time and it would be silly to say no.
Fintan Walton:
Exactly and the is also you got this say Independent Citizen Oversight Committee.
Alan Trounson:
Yeah.
Fintan Walton:
Which which is made up of 29 peoples so that's that should, should also make some of your work job interesting at times?
Alan Trounson:
Well it is because a 29 member board for which 20 are required to be there for Quorum and they are board making all the decisions so all I can do is put the recommendations to them seeking them to support what we want to do and there all those meetings are held in the public so you know that there is the Media and the and the public are there and they can make a three minute comment on anything so so it's a very different kind of relationship that I ever heard of before.
Fintan Walton:
Sure.
Alan Trounson:
And I was kind of scary in the first place but it works really very well because the board has a lot of passionate wickets on it. It has a lot of, a number of you know Deans of established medical schools, with business people, film people, people out of the-the biotech industry there who are not in stem cells but you know they are they are very interested people. There is a diversity there so there is no small group you know getting control and then taking it somewhere that you don't really want to go
Fintan Walton:
Sure.
Alan Trounson:
So they they usually very (indiscernable) and but receptive to all arguments including the public at the time.
Fintan Walton:
Okay.
Alan Trounson:
So I think it's a very healthy way of doing it.
Stem cell research in next 10 years
Fintan Walton:
Right, what is the vision? It's a 10-year vision?
Alan Trounson:
Was ten-years of raising the bond money
Fintan Walton:
Certainly, yeah.
Alan Trounson:
So that the proposition allows them to raise that for 10-years.
Fintan Walton:
Right, so what could you do you see in the next 10-years? What what's your vision for the outcome from all this work?
Alan Trounson:
Well I think some of it is totally unpredictable because things are moving so quickly. I didn't think you could ever take a skin cell and turn into equivalent of an embryonic stem cell I didn't think that was possible, two-years ago, it is now and clearly that's changed everything again. But what I " what I can see is that there will be new drugs that will come on to the market, there will be they will come because you know we've made stem cells from patients with (indiscernable) or made this iPS these new iPS cells from patients with Alzheimer's and so on. They will find new drugs because they've got new ways of testing. So that will be, that would be a big lift I think with the pharmaceutical industry but I think we'll see I think we'll see spinal repair, I think we will see repair of retinas or retinal repair I think people who are who really basically can't see any more will be able to see again. I think we will have cardiac function repaired, the heart muscle repaired differently to to the way I thought it was going to be done but it will be done. I think well it will be helpful from MS the Multiple sclerosis and you can go to a raft of things which I think will all exceed be clinical treatments if you look 10-years down the track.
Fintan Walton:
Right.
Alan Trounson:
But being
Alan Trounson:
The cell therapy will be it will become common place in ten years?
Alan Trounson:
I think it will be. And that's a very short time and well I am an Optimist. most people would say there is a 25-year time frame but there is so much energy, so much money, some much, it's good science the biotechnology industry really being in there that I think if it has potential it will show up in that time.
Fintan Walton:
Professor Alan Trounson, thank you very much indeed for coming on the show.
Alan Trounson:
It's a big pleasure. Thank you.
Alan Trounson
President
Alan Trounson is President of the California Institute of Regenerative Medicine in San Francisco, California. Prior to joining California Institute of Regenerative Medicine in January 2008, Dr. Alan Trounson was Professor of Stem Cell Sciences and Director of the Monash Immunology and Stem Cell Laboratories at Monash University, where he retains the title of Emeritus Professor. Dr. Alan Trounson founded the National Biotechnology Centre of Excellence 'Australian Stem Cell Centre'. Alan Trounson held various positions at Monash University beginning in 1977 and was appointed Director of the Centre for Early Human Development in 1985. He was awarded a Personal Chair in Obstetrics and Gynaecology/Paediatrics in 1991, and in 2003 was awarded a Personal Chair as Professor of Stem CellSciences. A Fellow of the Royal College of Obstetricians and Gynaecologists and an Honorary Fellow of the Australian and New Zealand College of Obstetricians and Gynaecologists, he was awarded an honorary doctorate by the faculty of medicine at the University of Brussels. Dr. Alan Trounson has been a pioneer of human in vitro fertilization (IVF) and associated reproductive technologies, the diagnosis of inherited genetic disease in pre-implantation embryos; the discovery and production of human embryonicstem cells and of their ability to be directed into neurons, prostate tissue and respiratory tissue.
California Institute of Regenerative Medicine
California Institute of Regenerative Medicine (CIRM) was established in early 2005 following the passage of Proposition 71, the California Stem Cell Research and Cures Initiative. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, was approved by California voters on November 2, 2004, and called for the establishment of a new state agency to make grants and provide loans for stem cell research, research facilities and other vital research opportunities. The mission of California Institute of Regenerative Medicine is to support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury. To date, the California Institute of Regenerative Medicine governing board has approved 229 research grants totaling more than $614 million, making California Institute of Regenerative Medicine the largest source of funding for embryonic and pluripotentstem cell research in the world.