This week marked the release of new draft FDA guidance on cell and gene therapies. Is the FDA kind of like the guidance counselor at school or more like the principal? It probably depends on many factors. I also find it funny how pretty much everyone in academia and biotech industry works to be compliant even with draft guidance, while many in the unproven stem cell clinic sphere strategize how to get around it or at least not get caught.
We’ll start with this FDA guidance and then move on to MSCs and recommended research pubs including brain evolution.
But before I start, don’t miss the new piece out in Vice this week on the troubling Duke Cryo-Cell mega-million deal to make revenue off of injecting perhaps thousands of autistic and other kids with unproven cord cells. Dark stuff but an important read. I’m going to do a post reacting to the Vice article in a few days.
Also, check out this week’s Stem Cell Podcast with me as the guest.
Draft FDA guidance
Fresh from the agency we have new cell and gene therapy draft guidance entitled: Studying Multiple Versions of a Cellular or Gene Therapy Product in an Early-Phase Clinical Trial. Enjoy. What are the key points?
My very brief take on this is that if a sponsor is submitting two versions of one product, they still ideally would submit two separate INDs but the INDs can in a sense be functionally linked together for efficiency. This is just draft guidance so things could evolve. Weigh in in the comments.
Would an MSC by any other name smell as sweet?
Cell Stem Cell has tandem review articles on MSCs (whatever you believe the acronym should stand for…the authors of these 2 pieces differ on that it seems):
- Multipotent stromal cells: One name, multiple identities. Hesham Soliman,Marine Theret,Wilder Scott,Lesley Hill,Tully Michael Underhill,Boris Hinz,Fabio M.V. Rossi
Mesenchymal stromal cells: Putative microenvironmental modulators become cell therapy. Mauro Krampera,Katarina Le Blanc.
Other recommended reads
An inducible p21-Cre mouse model to monitor and manipulate p21-highly-expressing senescent cells in vivo, Nature Aging. I’ve written before about how researchers are hoping to improve health and maybe slow aging by targeting senescent cells with so-called “senolytic” molecules. If you can identify the senescent cells easily it could help with development of senolytics.
From my old haunting grounds we have from Ahmad K and Henikoff S. (2021) The H3.3K27M oncohistone antagonizes reprogramming in Drosophila. PLoS Genet. 17(7):e1009225. Great stuff. My lab is also studying H3.3 K27M but in human glioma.
Comparative cellular analysis of motor cortex in human, marmoset and mouse, Nature. You can see in the image above from a different paper just how much these brains vary in size and morphology overall.