October 20, 2020

The Niche

Knoepfler lab stem cell blog

Interview with Robert Klein on CIRM’s Future

Bob Klein photoWhat does the future hold for CIRM? I asked former CIRM Board Chair and Prop 71 leader, Robert Klein, about that and more in the interview below. He also says some particularly exciting clinical trial and other developments in the stem cell field are coming soon.

What got you excited for the very first time about stem cells & regenerative medicine?

Klein: In 2002, I was talking to the Director of the NIDDK, the source of the NIH funding on Diabetes Research. My son, who has Type I Diabetes, was 12 at that time and my mother was dying of Alzheimer’s. Bush was going to shut down some crucial funding for Diabetes and we managed to successfully preserve and extend that funding with a $1.5 billion Mandatory Supplemental Appropriation for Type 1 and Type 2 Diabetes, to cover 5 more years of supplemental research. The Director said to me, “This is great, but ultimately we need new beta cells from ES cells. An ES cell based cellular therapy may present the best potential to cure Type 1 Diabetes.” I did a substantial amount of reading and I felt there were phenomenal opportunities for stem cell therapies to mitigate or cure Type 1 Diabetes. As a father, I felt I had to commit myself to pursue this scientific frontier.

What do you see the future holding for CIRM, particularly past 2017? How can we help CIRM continue beyond 2017?

Klein: I am extremely optimistic. The key is to report back to the public, which needs to evaluate CIRM’s achievements before making a decision on additional funding via another bond issue. For example, one tremendous milestone is that as of August 2013, CIRM had funded work that led to something like 1,750 publications of scientific discoveries and the scientists are publishing around a paper a day at this point. NIH is under significant pressure and we need a platform outside of NIH. I think California has and can continue to serve that role. Something like 50% of all the US biotech research capacity (including Academic, Non-Profit, and Private organizations) is in California alone.

I also am optimistic because we are at the crest of a wave of CIRM-funded research heading to the clinic. There is a broad spectrum of work that will extend into Phase I and II human trials using stem cells in the near future. I think the 2nd half of 2015 will be the critical time to make a decision about another bond initiative based upon the milestones of achievement; essentially, the California public will have to be convinced that the milestones of progress on therapies merit an extension of this critical funding

Are there specific trials that have grabbed your attention and will resonate with Californians?

Klein: Let me point out 3 examples of trials that hopefully will provide important milestones for California voters. The deal between J&J and Capricor, on a cellular therapy for victims of a major heart attack, is an example of a major third party validation of what CIRM is doing. Also, Viacyte will be starting a human trial in 6 months for Type I Diabetes. In addition, I’m excited about ACT’s work on RPEs (a validation of cellular therapies beyond CIRM funding). Peter Coffey’s team at UCSB is also working on Dry Eye Age Related Macular Degeneration; this human trial will start later this year as well, based upon CIRM funding. We have to be careful, but I’m extremely encouraged.

It’s a great organization, but are there ways that CIRM could improve? For example, what might it focus on if it received another round of funding?

Klein First, CIRM will be able to fund more of the highly successful Disease Teams to move potential therapies to Phase 1 human trials, and then through the Phase I and Phase II trials. Second, CIRM is already partnering with many international groups, in fact with 15 foreign governments, funding the California scientists on teams exploring basic, translational, and clinical research. I imagine in the future CIRM will expand that kind of global collaboration and we’ll have more opportunities for major breakthroughs through those collaborations, as we already have seen. I can see more collaborations with Canada, for example; these have been highly successful, with a major cancer discovery in the final reviews for a Nature article and another cancer breakthrough headed towards a human trial. Third, I’m also very excited about the interface between stem cells and genomics. In the near future we are going to see some major philanthropic announcements in this area. I believe that genomics will help CIRM answer the key question of its global mission: What is science telling us is best for the patient?

Any thoughts on the search for a new CIRM President? Ideally would this person stay on and transition beyond 2017?

Klein: That is the Board’s responsibility. I’m confident in them. I’d say ‘Get the very best person no matter where they are coming from’.  The Board has to decide whether this new President would be transitional or not.

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