8 key arguments, take-homes on day 1 of FDA stem cell meeting

Today was the first part of the FDA’s 2-day meeting on stem cell draft guidances. It was a great meeting I thought.

randy-mills-cirm-fda

Screenshot from video of FDA stem cell meeting of CIRM President Randy Mills speaking.

A few consistent themes and notable things emerged as raised by participants.

  1. 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.
  2. 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.
  3. 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.
  4. 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.
  5. 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.
  6. 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.
  7. 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?
  8. 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.

4 thoughts on “8 key arguments, take-homes on day 1 of FDA stem cell meeting


  1. Why should the clinics do these procedures “at cost”? Why, when you have private hospitals, drug companies, and clinics? Why should they do this in a time when you can’t get life saving drugs “at cost”? You can’t assume that for profit companies are malicious or greedy. Those exist in every field but it is not grounds to call for this. How do you know these clinics are really even profiting? The cost of regulatory compliance is sky rocketing in the midst of all of this. They still have expensive equipment and supplies to buy and staff to pay. I think this is a very mute point in consideration of small clinics and biotech start ups. Let me know when you can get epi pen at cost, then tell me how these clinics should be obliged to be non-profits.


    • @SE,
      I get what you are saying, but I was more making the point that the clinics are not just in it for the patients. They do want to make money. There’s nothing wrong with making a profit of course overall, but there is if what you are selling is not proven to work or be safe.

      The clinics that keep invoking patient rights and empowerment especially have a responsibility to back up their words with keeping patient well-being at the forefront. From talking to clinics themselves and other experts I have seen every indication that these clinics are highly profitable. I don’t know why you mention regulatory compliance since most of these clinics don’t spend money on that. As to the epi pen, yes it is way too expensive today, but there’s key difference compared to stem cell clinic offerings: epi-pens are proven rigorously to work (and in fact save lives) and generally be safe in controlled studies.
      Paul


      • Respectfully Paul, some of these doctors (many I have met at least) do charity work and strive to make cell therapies affordable especially for those who need it most. They often work under other names than their clinic’s namesake and these things don’t show up on their books. You just can’t put forth the image that these doctors are putting out a bunch of snake oil. Clinical trials aren’t a requirement for these therapies. There is strong clinical evidence, such as that presented today, that these therapies are very effective. I don’t believe a (actually several) clinical study is demerited because a small firm didn’t pay the millions of dollars needed to perform a clinical trial. In fact clinical trials are being done despite not seeing much coverage of them. There is very strong evidence for efficacy of cell based therapies for many applications. I wish you would stop taking the low road and jabbing at firms for the fact that they are private sector, and assumed to be in all aspects highly profitable.


  2. One point that you have left out which I believe is very important. The FDA should have a system for registering the Clinics and Doctors and Patients. The Clinics should also be inspected like the blood labs, sperm clinics etc. The Clinics should have to meet cGTPs for patient safety! This reminds me of the old blood labs in doctors offices, looked like the kitchen sink and then along came CILA..thank God! and then 5 years ago Office Operating Rooms where a mess too and now the Office Operating Rooms need to be certified registered and follow strict SOPs. These Doctor Clinics need the same, while the FDA wrestles with the language of the guidelines they need to get a handle on safety of the Clinics out there. Many speakers addressed this.

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