Vertex Pharmaceuticals has a lot going for it right now on the cell therapy front. For instance, they have the strongest type 1 diabetes cell therapy pipeline after some recent acquisitions. Still it’s not a simple matter to succeed in the cell therapy space even with one therapy for one targeted disease. Trials are tough to run, expensive, and groups of participants can be complex with many other health conditions or comorbidities.
Let’s start with a trial development in January for Vertex.
Vertex pause
Vertex Pauses Islet Cell Study After Patient Deaths, Medscape. I’m a little behind on this news from early January. From the article, “Vertex Pharmaceuticals, Inc. has paused a study of its investigational allogeneic stem cell–derived, fully differentiated pancreatic islet cell replacement therapy (VX-880) following two patient deaths.”
The deaths include that of Brian Shelton who was the first to get lab-grown cells for diabetes. He had been doing very well after receiving the cells. I’m not clear on the cause of death for him or the other participant who died, but the company said it was unrelated to VX-880. They are continuing another trial too.
“Vertex is continuing with a phase 1/2 clinical trial of a different product, VX-264, which encapsulates the same VX-880 cells in a device designed to eliminate the need for immunosuppression.”
Perhaps the pause has now been lifted? So far, I haven’t been able to find out. I don’t see anything on the company news page. I’m still cautiously optimistic that Vertex will ultimately get a diabetes cell therapy approved.
More recommended reads
- “The Kardashian Of Longevity”: Is Bryan Johnson Good For The Nascent Longevity Biotechnology Industry? Forbes by Alex Zhavoronkov. While the headline is funny, it does highlight the superficial nature of a lot of the dialogue and the “science” here. As readers of The Niche probably recall, I’m skeptical of many of the things Bryan Johnson reportedly is trying on the anti-aging front. He definitely raises awareness for anti-aging research, but all kinds of risky things seem to end up in the mix too. Snorting stem cells. DIY gene therapy. And, of course, “young Swedish bone marrow, which seems to have followed on from young blood from his son.
- Searching for information about stem cells online in an age of artificial intelligence: How should the stem cell community respond? Stem Cell Reports. This is a great piece so check it out. Searching online about stem cells is not easy and not even very safe for patients. There’s so much noise and marketing of risky injections. Recently, I wrote about the zoo that is stem cells on Reddit. The amount of sneaky marketing on there is huge. I’ve also repeatedly covered the ongoing challenges with Google search about stem cells including recently in a piece I did on MedPage Today. Google Search often does a bad job with knowing who has expertise in this space. Or it knows the unproven clinics aren’t experts but ranks them highly anyway. In either case, as a result, it ranks unproven stem cell clinics at the top of many important searches.
- Most MS patients stay relapse-free 6 years after stem cell transplant, MS News Today. Here’s the original research article: “Autologous hematopoietic stem cell transplantation for multiple sclerosis: Long-term follow-up data from Norway,” in the journal Multiple Sclerosis Journal. This is more good news that in certain types of MS, the combination of chemo and HSCT can be very effective. Note, that this is not what most unproven stem cell clinics are selling the way of sketchy stem cells for MS. The FDA has also not yet approved any HSCT for MS in the US.
- Athersys files for bankruptcy, sells assets to Healios in wake of stroke cell therapy’s struggles, Fierce Biotech. This unfortunately fits with my stem cell predictions for 2024 of cell therapy biotechs having another rough year.
- Ever feel impatient while a paper is in review? It could be worse. A paper took 900 days in review at Nature: How humans lost their tails — and why the discovery took 2.5 years to publish. We actually don’t entirely lose our tails as during early development, human embryos have a rather normal-looking tail. It just doesn’t continue developing and/or persist.
- A versatile CRISPR-Cas13d platform for multiplexed transcriptomic regulation and metabolic engineering in primary human T cells, Cell.
Athersys going bankrupt was predicted by you if I remember correctly !!