It’s been a crazy week on the cell and gene therapy front. We live in strange times more generally.
I just wrote about a Thursday FDA cell and gene therapy roundtable that generally promoted the idea of less oversight. And far more speed to human use.
RFK Jr. was present at the end and spoke.
Almost all the speakers, often top academic researchers, pushed for the FDA to exercise much less oversight here.
A growing feeling of unease about coming cell and gene therapy deregulation
For example, pioneer Carl June floated the idea that investigational autologous gene therapies should only initially being regulated locally by IRBs and not the FDA. It’d be super risky.
I came away from the meeting initially feeling it was a mostly positive gathering. Although the risks of the speedy or minimal FDA oversight emphasized at the meeting worried me, I tried to focus on the upside of the unique gathering. However, as time has passed, a feeling of deeper concern about the meeting has taken over.
Part of that shift may have been influenced by the revelation that Kennedy received unproven stem cells himself. In the podcast where he revealed his stem cell treatment, he also suggested much wider access to such cells or other similar unproven therapies for Americans would be a good thing.
Big changes are on the horizon for the FDA related to our fields. A risky experiment in deregulation is likely coming.
Recommended reads
- Wondering about what grants have been canceled by the Kennedy HHS? There’s an HHS database for canceled grants. I’m not sure why, but it’s there. It’s also not clear how up-to-date it is. You can search for keywords and see patterns. It’s also easy to find vast amounts of important research that were halted for no good reason. The database does not appear to include other kinds of squashed funding like when grants are simply not allowed to be renewed. As we’ve talked before, this is really shooting ourselves in the foot as a country. It’s also not actually saving money given the overall budget.
- H3.3 deposition counteracts the replication-dependent enrichment of H3.1 at chromocenters in embryonic stem cells, Nat Comm. H3.3 is our main molecular of interest these days in my lab including in normal development and glioma, although we still study MYC protein and other factors as well.
- Speaking of glioma: FGFR inhibition as a new therapeutic strategy to sensitize glioblastoma stem cells to tumor treating fields, Cell Death Discovery.
- Remembering James Till, a pioneer in stem cell research, University of Toronto. Till was a great researcher who made important discoveries related to hematopoietic stem cells. He and Ernest McCulloch had the key finding reporting stem cell colony formation and functionality. Also see: Who discovered stem cells & when? Some fun science history.
- An analysis of stem cell training programs for physicians in the US—A mirage of credibility, Stem Cell Reports. Congratulations to Zubin Master and the other authors on this important piece. Some of you may remember that I published a paper calling for rigorous regenerative and cellular medicine training back in 2013. We still need more such programs. Too many of the trainings offered are run by stem cell clinic folks.
Blast from the past: my interview with Steven Pinker
Steven Pinker interview: case against bioethocrats & CRISPR germline ban.