Weekly reads: RFK Jr. stem cell summit, FDA OKs eye cell therapy, cartilage repair

I had a feeling late last year that I’d frequently be writing about RFK Jr. and his impact on the FDA. However, I didn’t realize some of that would be writing for STAT News.

As I mentioned a few days ago, I now have a new regular column over there. I thought the column would be just a once-a-month kind of thing, but so much has been happening that I put together another column this week.

RFK Jr. and regenerative medicine

Yes, it’s on Kennedy: RFK Jr.’s regenerative medicine roundtable on stem cell deregulation raises red flags. Check it out.

Donna Chang of Hope Biosciences did a video about attending the Kennedy meeting. It provided some info. See a picture from her video above of her and RFK Jr. I also highly recommend this piece from Endpoints, which had the scoop on the meeting: RFK Jr. convened roundtable around ways to reduce stem cell regulation.

Kennedy is not the FDA commissioner, but as head of HHS, he will have major sway. A big question is whether likely FDA Commissioner Marty Makary will go along with Kennedy or stick to rigorous science-based medicine.

Good news: FDA approves new cell therapy for rare eye condition

• FDA approves Neurotech cell therapy to slow a rare degenerative eye disease, Endpoints. This is exciting news. The FDA doesn’t often approve cell therapies. Here’s the company’s announcement: “Neurotech’s ENCELTOTM (revakinagene taroretcel-lwey) Approved by the FDA for the Treatment of Macular Telangiectasia Type 2 (MacTel).” Check out my updated comprehensive, lightly-annotated list of FDA approvals of cell and gene therapies.
• This other piece from Endpoints is also interesting: FDA’s medical review departments remain untouched (for now) by Musk’s cuts. I’m not sure this “untouched” status will continue and as I’ve written before, we’ve already seen some key departures from CBER.

Other recommended reads

• Engineered stem cells automatically deliver arthritis drugs at prescribed times of day, Medical Express. This is cool work from Shriners researchers.
• Decoding functional hematopoietic progenitor cells in the adult human lung, blood. It’s surprising how many blood stem cells and progenitors are present in human lungs. It’s reportedly on a similar scale as marrow: “Here, in the adult human lung, we show that functional hematopoietic precursors reside in the extravascular spaces with a frequency similar to the bone marrow, and are capable of proliferation and engraftment in mice.” Incidentally, why does the journal “blood” use a lower case “b” to start its name?
• 2025 Brain Prize honors pair of cancer neuroscientists. This award went to scientists studying the pediatric glioma-brain interface including Frank Winkler and Michelle Monje. Michelle’s work has focused on H3.3 K27M glioma, which is also a major interest of my lab.
• Clinical relevance of engineered cartilage maturation in a randomized multicenter trial for articular cartilage repair, Science Translational Medicine.

Blast from the past: do some researchers have too much NIH funding?

Stem cell monopoly: do not pass go, do not collect $200,000. This piece is from 14 years ago. It’s interesting to sometimes run across individual PIs now who have 5, 6, or even 7 R01s at once time. How is that possible?

Every so often there’s a debate as to whether NIH should limit the maximum number of R01s that any one PI can have. What do you think?

Some data have suggested that impact per each R01 grant goes down after having three R01s.

As a side note, I think the post mentioned $200K here because of the Monopoly connection and the $200 for passing Go. Unfortunately, NIH modules for R01s have been stuck at a max of $250K for ages, although fewer people submit modular R01 proposals these days.