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

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.

Internal FDA Fighting on Sarepta Bad Omen for Stem Cell Oversight?

The FDA caused major controversy by approving the drug Exondys 51 from Sarepta Therapeutics for treating Duchenne muscular dystrophy over the recommendations of a scientific panel it convened that had voted 7-3 against approval.exondys-51-sarepta

Is this contentious Sarepta approval serve as a sign of how the FDA will deal with stem cells moving forward?

Will the FDA approval, which was based on very limited, unclear data and in large part due to pressure from some patient advocates, serve as a warning to those of us hoping the agency will not yield to political or other pressure in weakening oversight of investigational stem cell therapies?

Newly released internal FDA emails show that Sarepta approval was enormously controversial within the agency itself. A top FDA scientist challenged the agency’s decision in strong terms, noting that “Sarepta was unable to reproduce its own findings and there was “no way to reach a rational conclusion” a “reasonably likely” clinical benefit could be predicted.”

I wonder if similar debates within the FDA could be going on right now related to its four draft guidances on stem cells and regenerative medicine. Ultimately could the agency leadership make judgment calls without data in effect green lighting stem cell clinics to continue offering unproven stem cell therapies and putting patients at grave risk?

If I had to guess I would say optimistically that this is unlikely to happen, particularly given the disastrous case studies the FDA heard at the meetings it called on stem cells. For instance, we heard more about the patient Jim Gass developing a spinal tumor and the breaking story of at least three women reportedly being blinded by a stem cell clinic in Florida. A man in the audience reportedly wore a sign around his neck saying he was blinded by a stem cell clinic. I have heard through the grapevine that more bad news is coming from Florida on the stem cell clinic front.

One way or another the FDA should say something about its draft guidances on stem cells by early 2017. The number of stem cell clinics appears to be continuing to shoot up. The FDA can be more efficient in reviewing stem cell products in development, but it all has to remain data-centric.