Upbeat prospects for some California clinical trial efforts from CIRM

Over at the California Stem Cell Report, David Jensen is reporting on some good news from CIRM for California on the stem cell clinical trial front.

Stem cell biotechs Asterias and Capricor have stem cell trials supported by 20+ million in CIRM funding each and have been hitting milestones. These trials are progressing and so far have good safety profiles. Asterias and CIRM have mentioned some possibly encouraging early hints at efficacy as well in its trial, and apparently there are hopeful hints from the Capricor trial too.

See the posts from CIRM here (a weekly summary kind of post that begins discussing Asterias) and here. For background, also see past posts I’ve done on both companies here and here in the archives, and see especially my interview with Asterias leadership from a few months back.

It’s early days for these trials and at these phases they are not really designed to look for efficacy so a conservative approach to discussing such trials is in order given the stage, but at this phase of the game for early clinical trials the news has been all one could hope for so far in both cases.


Asterias cells

The Asterias and Capricor trials are for spinal cord injury and Duchenne muscular dystrophy, respectively. The latter trial utilizes the Capricor CAP-1002 product, which is a cool allogeneic cardiosphere technology made from donor human heart tissue. A beating cardiosphere from a different source (IPSCs) can be seen in the video above. Asterias’ trial employs their OPC product made from hESCs, which is also inherently allogeneic. The idea of potentially repairing the injured spine via stem cells is intriguing.

I’m hoping in the next month or so to do a broader update on the stem cell and regenerative medicine biotech arena. By way of disclosure, I do not have any financial stake in either company discussed here.

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.

J&J Bets Up to $325 million on Capricor: Big Pharma Stem Cell Move as Predicted for 2014

linda marban CapricorBig Pharma Giant Johnson & Johnson (J & J; $JNJ) announced today a $12.5 million investment in stem cells & regenerative medicine via the small biotech Capricor ($CAPR) and the potential for the bet to go into the hundreds of millions for the heart disease program.

It was only 8 days ago that I made my stem cell predictions for 2014 and #3 on the list was “Big announcement  from “Big Pharma” on stem cells or regenerative medicine.”

Chalk that up for the crystal ball of this blog at one for one so far for 2014.

According to the FierceBiotech today:

Later this week a little biotech named Capricor Therapeutics is set to begin a Phase II study of its new stem cell therapy with about $20 million in support from the California Institute for Regenerative Medicine (CIRM).

J&J’s role could shoot up to over $300 million depending on how things go.

I’d call that big money. Investors seem enthused as well as the CAPR stock is up nearly 50% today at the close.

The Capricor adult stem cell product is CAP-1002, which is in clinical trial and consists of allogeneic cardiosphere-derived cells. The trial may enroll up to 274 patients, which is impressive.

FierceBiotech quotes Capricor CEO Linda Marban (pictured above) as follows:

“One of the reasons why I was motivated to work on this deal is because of the statement it makes in the field,” says the CEO. “It says, OK, somebody very large and powerful is taking a look at this technology and saying there’s something there, and that’s the most exciting thing for me.”

I’ll be very curious to see what else big pharma might have in store on the stem cell/regenerative medicine front.

Disclosure: To my knowledge, I have no financial interests in the companies discussed in this piece.