Are stem cell clinics going to somehow get the FOOD AND DRUG ADMINISTRATION to approve their stem cell interventions? To get named legit? It seems they are trying now more than ever.
Remember the REGROW Act that would have greatly reduced regulations on investigational stem cellular material and then how the 21st Century Cures Act passed with some REGROW-related provisions, but more reasonable ones?
The status quo of how the FDA controlled stem cell-based regenerative medicine therapies wasn’ t operating so great in past years so some reforms had been (and still are) needed, but the key was striking the “ Goldilocks” sweet spot of just the right amount of regulating change.
I was worried about REGROW specifically because I viewed it as having overshot with a mile by going for too much reduction in oversight (particularly in the original language, although it moderated somewhat over time) and am had mixed feelings on Cures, but it was a minimum of relatively more balanced. Cures passed, was signed simply by Obama, and is now law. One of its main provisions upon stem cells is so-called Regenerative Advanced Therapy (RAT, an unfortunate acronym choice that it seems the FDA wants to change to RMAT ) status that could speed up vetting of stem cell therapies.
My concerns about the legislative efforts were generally stemming from the possibility that stem cell clinics might try to take advantage of the less stringent regulations. Could they obtain interventions to be designated as RATs? Could a few of the clinics get FDA approval?
Now a few of the clinics are trying to make that a reality.
I’ m hearing from multiple sources that a number of originate cell businesses running clinics have already applied to the FOOD AND DRUG ADMINISTRATION for RAT/RMAT designation that puts therapies automatically for the accelerated pathway. Most of these businesses have not publicly announced their particular applications, but US Stem Cell, Inc., a publicly-traded stem cell clinic company disclosed that it has applied for RAT status. You might remember this businesses as it was associated with the dazzling of three of its patients . I’ meters hearing that other businesses are already applying for RAT as well. US Stem Cell wrote:
“ Following the passing of the 21st Century Cures Act, U. T. Stem Cell, Inc. has applied to the FDA regarding RAT Designation. We have recently heard from the FDA, that has requested additional information regarding the MARVEL Phase II/III trial. We now have provided all requested information to the FDA and are positive that the FDA will continue their expeditious review of our own MyoCell product. Thanks to the REGROW component of the Cures Respond, the FDA will grant RAT designation for a regenerative medicine therapy that is intended to treat, modify, reverse, or even cure a serious or life-threatening disease and demonstrates first 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 effectiveness in both preclinical and clinical studies… ”
Presumably, some of those working on investigational stem cellular therapies from non-clinics are going for RAT designation too and a few of those are probably not going to get approved. On the other hand, some come cell clinic and non-clinic applications to the FDA could get the RAT stamp of approval.
General, how will the FDA decide what to approve as a VERWEIS and what to reject as not a RAT? How efficient will the FDA be able to be on vetting the particular 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 simply funded by CIRM and this RMAT designation seems like an optimistic move.
Hold on to your hat (or seat, or pet rat, or whatever) as we will now every witness a high stakes regulatory experiment unfold in front of all of us in the coming months and years due to Cures. The end result is likely to be mixed overall, but we can hope it’ lmost all be on the whole a positive for the field and for patients. There’ s no crystal ball on this though.