The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development. 2 FDA-approved gene therapies for sickle cell Friday’s news follows on recent UK approval for …
For about as long as I’ve been writing The Niche, people have been asking about stem cells for MS. There’s a huge need for new therapies. While a chemo-based approach with autologous hematopoietic stem cell transplantation (HSCT) looks to work for certain cases of multiple sclerosis (although not yet approved in the US), other cell …
Weekly reads: stem cells for MS, good news x2, extending dog years, Neuralink updates Read More »
Let’s start this week’s digest with a discussion of clonal expansion and mutations during cell and gene therapy development. Here’s the article that brought this to mind. Sickle cell gene therapy process may cause cancer-linked mutations in blood stem cells, Fierce Biotech. Clonal expansion This is a story about a non-CRISPR gene therapy using a …
Some folks can view data from early, small clinical trials too skeptically or overly enthusiastically, and maybe that’s going on with some preliminary results from Verve Therapeutics. Good news? Bad news? I’m going to start with two articles about the same news that have very different vibes. What do I think? On the whole, I …
Weekly reads: Verve Therapeutics, Casgevy UK OK, GDNF, MYC Read More »
For me, this was another week of heavy grant writing but also taking on Google again. How? Over how badly its search engine often performs on stem cell queries. I have a new piece at MedPage Today. It’s focused on the problem of Google Search promoting unproven stem cell clinic websites. Google still prefers stem …
Weekly reads: Google, CIRM, young Swedish bone marrow Read More »
What’s more important than Woolly Mammoth de-extinction research in the stem cell arena? Only maybe a 10,000 other things. Still, the mammoth de-extinction efforts capture people’s attention much more than the average research story. Mammoth De-extinction update Is de-extinction only a pipette dream? This startup has a big, expensive plan to find out, Popular Science. …
Paolo Macchiarini is one of a small group of people in the stem cell universe whose misconduct has blown up in the press. Piero Anversa, Haruko Obokata, Hwang Woo-Suk, and some operators in the unproven stem cell clinic sphere come to mind. Macchiarini published quite a few seriously problematic papers, some of which just hung …
Weekly reads: late Macchiarini retractions, stem cells & Lululemon Read More »
There’s been much discussion about issues related to stem cells in the reproductive space such as human embryo models and differentiating stem cells into gametes, but there’s also interest in stem cell-assisted IVF. In this kind of method, stem cells are employed to make tissue cells such as ovarian stromal cells to support production of …
Weekly reads: stem cell-assisted IVF, Takeda MSC trial, reporters getting stem cells Read More »
Most of us have heard of organoids but what about the related model called an “assembloid”, which is a term growing in use including from organoid researcher Sergiu Pasca of Stanford? Before we jump into that, be sure to enter The Niche stem cell image contest for your chance to win $100 or some swag. …
People often ask me what are the most promising stem cell therapies in development and new data suggest we should add stem cells for epilepsy to the list based on work by Neurona Therapeutics. I’ll start off the weekly recommended reads with this new mouse paper on cell therapy for epilepsy. Neurona Therapeutics: stem cells …