The FDA’s Stem Cell “Workshop” just ended, and if you’re a person who wants continued access to stem cell therapies outside of clinical trials (or a transplanter of “unproven stem cell therapy”), it was a very depressing day. It was way worse than I had feared in my post yesterday. http://rickjaffeesq.com/2016/09/07/fda-stem-cell-meetings-close-hand-stakeholders-sides-worried-one-side-really-worried/
Sure the two ethicists said that it was wrong and unethical to provide unproven stem cell therapy except in FDA sanctioned clinical trials. But the real punch came from two of the last speakers who discussed some horror stories of the severe harm caused by “unregulated” and “unproven” stem cell transplanters.
Stem Cell Horror Stories
Unregulated Stem Cell Therapy Causes Cancer
A Harvard fellow who co-wrote the recent oft-quoted case study of the stroke patient who traveled the world doing stem cells only to develop a stem cell treatment induced CNS tumor throughout most of his spinal column gave the gory details of the case, including a painstakingly detailed complete histology of the tumor. The only good news (if it can even be called such) is that, if I understood correctly, he concluded that the stem cells causing the tumor were not the patient’s but were the result of an allogenic transplant the patient received.
Unregulated Stem Cell Therapy Causes Blindness
The other presenter, an eye doctor, talked about three patients who received stem cells for macular degenerative disease. The procedures were performed in a South Florida stem clinic by a nurse practitioner, not under the supervision of a licensed physician. In each of the three cases, there was severe harm, requiring emergency surgery with catastrophic negative results to the eye sight of the patients. Of course these might be unusual and exceptional cases, but they will no doubt be used by the FDA to demonstrate the need for exactly the draft guidance documents which it is proposing to stop these medical horror stories from reoccurring.
FDA Implored to Stop the Maiming by Snake Oil Stem Cell Transplanters
Other members of the panel including leaders of disease advocacy/research entities all decried “unproven stem cell therapy” and implored the FDA to put these unethical stem cell purveyors of false hope out of business.
One panel member pointed out that the problem was just not in the U.S. and that there were these kinds of clinics in many parts of the world and because of the internet, it was easy to find out about them. One clinician lamented that when prospective patients call him and he has to tell them that he has no treatment options for them, the patients get mad and argue with him.
They should all be delicensed!
The day’s moderator was Irv Weisman, who is one of the biggest names in academic stem cell research. In his summary, he suggested that U.S. physicians who perform unregulated stem cell transplants abroad to skirt U.S. regulations be delicensed.
To listen to these guys, it would appear that stem cells given outside of clinical trials have never helped anyone. In one sense that might be true. If all you care about is scientific data from well-designed FDA clinical trials, then they are right. Anecdotal evidence is not science, and all seemed to agree that all the non FDA trial data which is published is not worthy of consideration because of design flaws.
There is a Consensus
The absolute, universal consensus of today’s workshop was that stem cells should only be available in the U.S. in FDA approved clinical trials, until such trials prove that there is a safe and effective non homologous use for them. And anyone who provides “unproven stem cell therapy” to patients outside of clinical trials is a greedy, unethical charlatan who should lose their medical license…That’s a tough message, but no doubt a welcome one for the FDA since the elimination of non-homologous autologous stem cell therapy outside of clinical trials is the purpose and end result of the four draft guidance documents.
One thing I am clear on: the positions taken today by the people the FDA considers the thought leaders in the field really, really need to be addressed by the stakeholders on the other side next week, head on.
I’m going to give this a good think over the next day or two and maybe make some suggestions to my presenter friends.
Rick Jaffe, Esq.