Are stem cell clinics going to somehow get the FDA to approve their stem cell interventions? To get recognized as legit? It seems they are trying now more than ever.
Remember the REGROW Act that would have greatly reduced regulations on investigational stem cells and then how the 21st Century Cures Act passed with some REGROW-related provisions, but more moderate ones?
The status quo of how the FDA regulated stem cell-based regenerative medicine therapies wasn’t working so great in past years so some reforms were (and still are) needed, but the key was hitting the “Goldilocks” sweet spot of just the right amount of regulatory change.
REGROW concerns
I was worried about REGROW in particular because I viewed it as having overshot by a mile by going for too much reduction in oversight (particularly in its original language, although it moderated somewhat over time) and I had mixed feelings on Cures, but it was at least relatively more balanced. Cures passed, was signed by Obama, and is now law. One of its main provisions on stem cells is so-called Regenerative Advanced Therapy (RAT, an unfortunate acronym choice that it seems the FDA wants to change to RMAT) designation that could speed up vetting of stem cell therapies.
My concerns about the legislative efforts were mainly stemming from the possibility that stem cell clinics would try to take advantage of the less stringent regulations. Could they get their interventions to be designated as RATs? Could some of the clinics get FDA approval?
Now some of the clinics are trying to make that a reality.
Clinics too?
I’m hearing from multiple sources that a number of stem cell businesses running clinics have already applied to the FDA for RAT/RMAT designation that puts therapies automatically on the accelerated pathway. Most of these businesses have not publicly announced their applications, but US Stem Cell, Inc., a publicly-traded stem cell clinic company disclosed that it has applied for RAT status. You might recall this businesses as it was associated with the blinding of three of its patients. I’m hearing that other businesses are already applying for RAT too. US Stem Cell wrote:
“Following the passing of the 21st Century Cures Act, U.S. Stem Cell, Inc. has applied to the FDA for RAT Designation. We have recently heard from the FDA, who has requested additional information regarding the MARVEL Phase II/III trial. We have provided all requested information to the FDA and are hopeful that the FDA will continue their expeditious review of our MyoCell product. Thanks to the REGROW component of the Cures Act, the FDA will grant RAT designation for a regenerative medicine therapy that is intended to treat, modify, reverse, or cure a serious or life-threatening disease and demonstrates preliminary clinical evidence that the product has the potential to address unmet medical needs for a disease. We believe that our MyoCell product meets these requirements, as we have demonstrated clinical efficacy in both preclinical and clinical studies…”
Presumably, some of those working on investigational stem cell therapies from non-clinics are going for RAT designation too and some of those are probably not going to get approved. On the other hand, some stem cell clinic and non-clinic applications to the FDA may get the RAT stamp of approval.
Looking ahead
Overall, how will the FDA decide what to approve as a RAT and what to reject as not a RAT? How effective will the FDA be able to be on vetting the host of RAT applications it is getting?
Showing how fast this could all happen, the FDA has already granted the now-re-named RMAT designation to some companies including Humacyte, which is in part funded by CIRM and this RMAT designation seems like a positive move.
Hold on to your hat (or chair, or pet rat, or whatever) as we will now all witness a high stakes regulatory experiment unfold in front of us in the coming months and years due to Cures. The outcome is likely to be mixed overall, but we can hope it’ll be on the whole a positive for the field and for patients. There’s no crystal ball on this though.
Dear Admin:
Thanks for alerting your readership to the new RMAT designation from the FDA for bringing oversight to private stem cell treatment activities. I’m encouraged by this development. It could provide a means for private stem cell clinics to share their clinical experience for third party expert review. By requiring “Preliminary clinical evidence” for the designation, submitting clinics would have to organize their prior clinical experience into a format accessible to scrutiny by FDA regenerative medicine experts. That development alone could improve patient care and accelerate progress in stem cell medicine. Since private clinics do not have proprietary issues to consider like cell therapy companies that pursue conventional clinic trial approval, the FDA could identify best practices for developing beneficial recommendations for all private stem cell clinics…and cell therapy companies for that matter.
Despite such potential, I am not optimistic that many clinics will seek the RMAT designation. Their reluctance will be due to the FDA’s plan of forcing them into the existing IND box, which is already a significant deterrent for their practice. Instead of relying on outdated approaches that do not fit active medical practice, the FDA needs to create a new “co-investigatory medical” approach that addresses the special needs of developing safety and efficacy information from routine clinical practice. Importantly, the greater burden of achieving and maintaining such a sea change in medical discovery must be taken on by the FDA (or a new agency!) instead of clinic physicians. If treating physicians could remove the current shadow from over their stem cell medicine clinics by reasonably modifying their practices for treatment planning, documentation, and reporting as prescribed by an oversight agency, many would probably consider it. But they are not going to go for being forced into a conventional clinical trials format to which they are required to conform in order to provide treatment to their patients.
James at Asymmetrex
If you can find out who lobbied the most for the 21st Century Cures Act, you will be able see who will be taking advantage of this as a loophole in FDA oversight. You are right that there may be benefit for legitimate therapies, but I fear that they’ll be lost in the weeds with the onslaught of companies whose motivation is money, not safety or helping patients.