It’s been a big couple of weeks of chaotic developments at the FDA including the ousting of CBER director Vinay Prasad. The Sarepta rollercoaster was tied to that.
If such recent events are any indication of how the next 3+ years are going to play out at the agency, it’s going to be negative for the field and patients.
First let’s go through some interesting papers on aging including the ties between aging and stem cells.
Recommended reads including aging links to stem cells
- Ageing accelerates around age 50 ― some organs faster than others, Nature.
- Comprehensive human proteome profiles across a 50-year lifespan reveal aging trajectories and signatures, Cell.
- Ageing stem cells in the knees drive arthritis damage Mice with a version of osteoarthritis experienced symptom relief after expression of a protein targeting a stem-cell receptor was blocked, Nature.
- Scientists discover less invasive way to collect babies’ stem cells, WaPo.
Do you agree with the idea that aging is a disease? Take our poll below.
FDA and Sarepta
Quite the rollercoaster for Sarepta and Elevidys over at the FDA. FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients, FDA. Thoughts? Just last week folks were discussing whether Sarepta could survive as a biotech. Then, as I mentioned, Prasad was fired. This may seem like old news given how much has been going on at the FDA, but it just happened a few days ago.
CBER moving away from use of untitled letters?
Also on the agency, you can look through biologics-related untitled letters from the FDA. So far, zero are listed in the subcategory of “IMO/Team Biologics/Internet Surveillance/Other” for 2025 but the page hasn’t been updated in more than a year. In the other category here, “Untitled Letters Regarding Advertising & Promotional Labeling for Approved Biologics” the page hasn’t even been updated in 3 years.
Maybe CBER is just not using this untitled mechanism much anymore or it still is but hasn’t been updating these pages.
Bad stem cell article of the week: “Stem cell therapy developed in Poland could revolutionize osteoarthritis treatment”
TVP World brings us breathless news of research in Poland from Ewa Zuba-Surma that, as the headline claims, “could revolutionize” osteoarthritis treatment. Except, this research is a long long way from that.
I’m not linking to this article because it’s a prime example of weak media on stem cells.
Where to start?
Despite the headline, the text says much more carefully, “But a new therapy developed by a team of researchers at the Jagiellonian University in Poland’s southern city of Kraków is offering a glimmer of hope.” A glimmer of a revolution?
And yet the team hopes “the therapy will become available within the next two years.”
The ‘therapy’ here is some kind of autologous adipose cell prep.
The literature on using adipose cells for arthritis is not particularly encouraging. Even most stem cell clinics in the U.S. no longer claim that adipose cell preps like SVF grow new cartilage once injected. They instead invoke anti-inflammatory functions.
Finally, it’s worth noting that Ewa Zuba-Surma has published many articles with Mariusz Ratajczak on supposed VSELs. I am skeptical that VSELs exist as naturally-occurring cells.
Yet these purported cells have been claimed to be adult pluripotent stem cells. The claims are similar in some ways to MUSE cells, which also seem in doubt.
Dr. Zuba-Surma seems to have shifted gears in recent years away from VSELs and toward adipose and other kinds of cells.