Today was the first part of the FDA’s 2-day meeting on stem cell draft guidances. It was a great meeting I thought.
A few consistent themes and notable things emerged as raised by participants.
- Some argued for middle ground on regs. Some speakers advocated for a “middle ground” between “nothing” in the way of regulation for instance of the dubious clinics and the perceived “mountainous” extreme of the current FDA oversight system. The middle ground was, for example, argued for by Janet Marchibroda of the BPC and then also in the next talk by CIRM President Randy Mills (pictured above). Mills, who is a very powerful speaker, argued that there is a harmful binary system now and we need a middle ground between the extremes of little-to-no regulation and the current in his view constricted pathway.
- Functional versus structural homology. Several speakers argued that the FDA should take into account not just structural homology, but also functional homology. As to the latter, paracrine signaling was cited several times. The point being argued seems to be that MSCs for instance can be functionally homologous to diverse tissues even if they are not structural homologous to them, and therefore the FDA should have a broader definition of homologous use such as for fat stem cells used for non-adipose-related tissue conditions.
- What is “main function” and is it too limiting a term? There was also some debate about what “main function” means as the FDA uses it in regard to homologous use and whether it is a new or long-standing terminology. For instance, ARM asked for clarification on “main function” as did some other parties at the meeting.
- Clarity, examples, and flowcharts needed from the FDA. Many parties asked the FDA for more clarity and consistency on oversight. Also, a number of speakers asked for concrete, real world examples of how definitions would apply. I’ve been pushing the FDA for increased clarity for years including lay abstract summaries of their main guidelines and statements of current thinking. I really like the idea of flowcharts.
- Assumptions of safety & efficacy. Several parties stated unequivocally that the stem cell “treatments” that they market definitely work and are safe, but I haven’t seen convincing peer-reviewed data to prove that. Most speakers overall seemed to realize that there are going to be risks of negative outcomes.
- Patient rights versus clinic profits. Several speakers invoked patient autonomy and patient rights, and argued that they are doing what they are doing as practitioners of stem cell therapies simply for the betterment of patients. There’s the pesky problem there with this argument of probably millions of dollars in profit being made from the patients by some clinics though. If it’s all about patients, I challenge the clinics to do the transplants strictly “at cost” with no profit margin.
- Fat transfer including to the breast should be homologous use, some argued. Some speakers including surgeons were concerned about the possible definition of fat transfer including to the breast as non-homologous use. I have to give this more thought, but off-hand I agree that lactation is not the only function of the breast and of course there is ample adipose in the breast normally. However, should there be a regulatory distinction between reconstruction and augmentation for cosmetic reasons in this regard?
- REGROW not mentioned much. There were unexpectedly few mentions of the REGROW Act. I noted one from Dr. Allan Mishra. Unless I missed it, interestingly I didn’t hear the BPC mention REGROW.