The FDA is holding its first 2016 stem cell meeting today and you can read about some impressions of the morning session of this meeting here.
In this post, I’m focusing on the afternoon session, which has been mostly on policy and ethics, including on stem cell clinics.
Jonathan Kimmelman from McGill University got the afternoon going with his excellent talk “Ethics, Evidence, and Regulatory Approval for Cell-Based Interventions“. Jonathan started his talk addressing the stem cell clinic situation in the U.S. and asking more broadly how regulatory authorities should establishment a benchmark for making experimental interventions such as stem cells available to patients.
He asked if there is a zero sum game between innovation and oversight? He argues there isn’t. They can actually work together. With oversight you maximize the amount of data per patient put at risk.
He used gene therapy taking decades (and still no approved therapy) as an example of the timeline for experimental stem cell therapies.
What about the idea of patients vs. bureaucracy? Is that a genuine dynamic? Are the most vocal patients democratic representatives of patients more generally?
In talking about the idea of safety vs. efficacy, he discussed risks of cell therapies.
He asked, “How do we as a society want to distribute the costs and burdens of medical uncertainties?”
How does this all tie in with the new 2016 ISSCR guidelines on stem cell clinical translation?
Key issues include the primacy of patient welfare and social justice. There should not be exposure of patients to unproven therapies outside of true clinical trials or in other unique circumstances (I’m thinking expanded access).
Jonathan also talked about concerns with pay-to-participate trials for stem cells.
Above is a summary slide from Jonathan’s talk.
Massimo Dominici, MD next gave his talk “Dissecting Unproven Cellular Therapies: The International Society for Cellular Therapy (ISCT) Position.”
Dr. Dominici talked about global stem cell clinic locations, the costs, and the global challenge here. He showed data from the important John Rasko Cell Stem Cell paper on global stem cell clinics. He also cited the example of the Stamina problem in Italy and the “weird” protocols involved there in that treatment. One patient died. See his slide above on Stamina, where UCT means unproven cell therapy. To sum up he indicated that a problem is confusion between unethical versus innovative cell-based approaches.
Peter Rubin, MD wrapped up the session on “Clinical Adipose-Based Therapies.” Dr. Rubin is a plastic surgeon who studies adipose (fat) stem cells. He talked about autologous fat transfer versus adipose stem cell treatment. As to the former, he discussed the strong safety profile and good results with fat transfer outcomes (for instance with facial deformities from wartime injuries). There’s about 63% retention of volume induced with fat transfer in their experience. He reported that the # of cells in the fat tissue correlates with better outcomes. Tissue remodeling (not just volume) occurs. He did also mention how fat stem cells might be able to promote cancer recurrence or progression.
What about adipose stem cell treatments? Rubin called the basis for this as being “bioactive” cells from fat or the stromal vascular fraction, which someone from Wisconsin he says apparently calls colloquially “sushi”. I’m not sure I get that. Rubin uses an automated machine to make SVF. Lots of heterogeneity in SVF cell types. He talked about IFATs (a federation for fat tissue/cells). These cells make growth factors. They are using these cells in pre-clinical studies now (e.g. in rodents). Their clinical strategy under an IDE is to mix the SVF with the fat graft for traumatic amputation. He advocated for taking a responsible, evidence-based approach.
Overall, I found these talks to be really fascinating together linking together real-world experiences with stem cells in clinical use (responsible or not or even in the middle gray zone in some cases depending on one’s perspective) along with ethical and policy considerations as well as guidelines.
Was at the hearing on Mon and Tuesday, was looking for Professor Knopfler earnestly, however unable to find. Maybe he would be open to being interviewed in Webinar format? Bill
Dr. Knopfler was hoping to meet you at the FDA hearings.
Appreciate the reports. Regret unable to make the meeting.