We recently saw exciting news with the first CIRM-funded therapy garnering FDA approval.
Before we jump into that and other news and new publications, a reminder that my lab is still crowdfunding to support our brain tumor research in these tough funding times. Please consider making a tax-deductible gift to give more hope for kids and adults facing brain tumors. Thanks!
CIRM-funded therapy gets FDA approval
The approved therapy supported by CIRM funding is KRESLADI from Rocket Pharmaceuticals, Inc. It will be used to treat a condition called severe leukocyte adhesion deficiency-I (LAD-I) in children who have no bone marrow donor match for stem cell transplantation.
This is a great milestone for CIRM and I expect more drug approvals related to CIRM-funded research.
Educational videos
There are two reasons I’ve been writing more about peptides in the last few years. First, people are getting hurt by these peptides and more are at risk. Second, the peptides are often claimed to have regenerative medicine-type properties. Stem cell communicator and student Parmin Sedigh and I have a new, growing series of educational videos. The newest video is just a few minutes on peptides as to key things to know.
If you like it, please follow the YouTube channel.
More recommended reads
- Asymmetric histone inheritance regulates olfactory stem cell fates during regeneration, Nat. Comm. When a “mother” cell has two “daughters” those two can be quite different. Those differences can be key to developmental processes. This has been known for quite some time, but the asymmetric histone inheritance seems relatively novel.
- Two States Sue Cord Blood Bank Over False Advertisements, NYT. For-profit cord blood banking is under stress. It’s expensive and almost no one ever needs the cord blood. Some firms have pushed the marketing pretty far. Other banks are trying to diversify into banking additional biologics like MSCs, or even pursue treating people at clinics.
- Distinctive DNA sequence features define epigenetic longevity of inflammatory memory, Science.
- Systemic epigenetic dysregulation as a driver of ageing and a therapeutic target, Nature Rev Mol Cell Bio.
- BMJ retracts cardiac stem cell paper, removes authors months after sleuths flag data ‘mismatch’, RetractionWatch. This paper was in trouble from the get-go.
- RFK Jr. May Reverse a Peptide Ban He Calls “Illegal.” Former FDA Officials Say He Mischaracterized Their Work. “The agency’s 2023 decision to place 19 peptides on the “unsafe” list was supported by numerous documented safety concerns, former officials said. Even though demand for peptide therapies has exploded since then, there’s been little new science.” Propublica. The bottom line is that these are unproven drugs just like any other unproven, unapproved drugs. Popularity doesn’t mean they should be made more available by the FDA.
The peptide topic is so interesting. You have to wonder, if they’ve made $10s of millions and still rely on anecdotes, and won’t even show a 20 person placebo controlled trial… chances are it doesn’t work, or the benefits are marginal.
If you mention this above, the response is “nuh-uh my dad is off his pain meds now”, and the wheel keeps turning.
But, should it be regulated in the way it is is the more interesting question. Are vitamins proven? How many of them have gone through rigorous RCTs?
Martin Shkreli, the pharma bro who went to prison and bought that 1 of 1 Wu Tang album, did a debate with a peptide bro a few days ago. It was more entertaining than informative, I’d like to see an actual peptide research scientist do it over instead of a salesperson.
https://www.youtube.com/watch?v=5ddXfe6H1a8
Iirc, he mentions the person who discovered BPC157 tried to do an RCT, and it failed, yet it’s still profited off of. Becoming a tale as old as time….