I have a new Cell Stem Cell piece out today that aims to take stock of today’s stem cell regulatory oversight in the U.S., Japan, and elsewhere including Australia, Canada and India. I use a carrot and stick metaphor for encouraging legit research and taking regulatory action on serious non-compliant activities, respectively.
Where is stem cell oversight today?
Too much stem cell carrot and not enough stick? Some would probably say too little carrot and too much stick?
Given how the stem cell arena can be dizzying with so many players and developments that seem positive or negative in a simple binary kind of sense, how are regulators doing in overseeing this difficult sphere?
The answer depends on who you ask, but for me as a stem cell scientist who has been watching the field for a long time and blogging about it here for more than 8 years, I believe that regulators aren’t in a sweet spot at this point in either encouraging the development of legit evidence-based stem cell and regenerative medicine therapies or on the other hand in dealing with for-profit, often predatory stem cell clinics. I discuss this in the Cell Stem Cell article entitled, “entitled, “Too Much Carrot and Not Enough Stick in New Stem Cell Oversight Trends”.
It’s been remarkable to see the FDA approve up to 20 regenerative medicine advanced therapy(RMAT) designations in just over a year. However, I think there’s a strong possibility the agency has swung too far from the too slow review of stem cell and regenerative medicine investigational therapies in the past to now going at warp speed. Since none of the current 20 designated RMAT products had any kind of prior expedited review designation given, is it reasonable to think all 20 now meet rigorous enough standards and all because of new data? It’s hard to say, but there’s likely a spectrum of existing data behind these RMAT designated studies.
We won’t have an overall RMAT verdict for years as the RMAT trials play out. However, I predict that the agency has lowered the bar too far. There are also concerns that the conditional approval system in Japan is too liberal, as evidenced by discussion over the approval of a recent IPS cell cardiovascular study. Taken together this is what I mean by “too much carrot”. Another issue with RMAT is that the criteria by which the designations are given (or not) are not clear.
On the other hand as to “too little stick”, despite the FDA doing a bit more on clinics so far in Commissioner Scott Gottlieb’s tenure (and I expect more is to come), with as many as 700 unproven clinics in the U.S. and maybe 1,000 globally, even the FDA’s tick up in activity is still not amounting to enough to make a real dent. That could change, but that’s my sense of where we are at now. It will be interesting to see how the two DOJ suits against clinics proceed.
So, check out my Cell Stem Cell piece and let’s think about stem cell regulatory sweet spots.