How is research looking on stem cells for diabetes? I’m feeling more encouraged about the clinical research in that area.
Before we jump into that let’s talk about two other things. The big news of the week was the felony plea deal by Liveyon’s John Kosolcharoen to charges related to marketing an unapproved cell therapy drug. This seems historic so check it out if you haven’t already.
Also, September 1 is when I usually start thinking about The Niche’s science hype award, The Screamers Award. As far as I know, The Screamers is the only science hype award out there. That’s surprising given the massive amount of science hype continually raining down on us. Send me your nominations for news articles or headlines that you felt were particularly full of hype or hypeful as I like to say. I guess “hypeful” may be a neologism. I enjoy such new words and phrases and have coined others like “health flexing“.
Okay, on to some encouraging news.
Stem cells for diabetes
Diabetes took over her life, until a stem cell therapy freed her, WaPo. I’ve been following this Vertex work for many years including in its previous incarnation of a sort at Semma. Vertex acquired both Semma and ViaCyte, two key small biotechs working on stem cells for diabetes. I like this article by Carolyn Johnston. It does a good job of mentioning that this is still a clinical trial and the overall results are not yet known, but it gives real hope.
The trial participant at the focus of the article, Amanda Smith, must still take immunosuppressive drugs to avoid rejection. The transplanted pancreatic cells are made from embryonic stem cells so they aren’t a match to patients like Smith. For that reason, the immune system would likely reject them.
Let’s say a cell therapy for diabetes therapy is proven safe and effective, yielding FDA approval. The cost is likely to be as much as a million dollars per treatment. That may seem like a lot, but the cost of the traditional approach to treating diabetes and its related health problems is also extremely high. A cell therapy for type 1 diabetes may also be a permanent fix.
By comparison, stem cell therapy cost at unproven clinics is often in the $10,000-$20,000 range, but there is almost zero chance of benefit and often substantial risks.
Stem cells or gene therapy for sickle cell disease
‘Low-intensity’ blood stem cell transplants for sickle cell appear safe for lung health, NIH. If stem cell transplants for sickle-cell can be made safer they provide another broader option, which is important. Using gene editing for sickle cell is an exciting new, FDA-approved approach. However, costs and access are likely to be huge issues.
Stem cell patches
Advanced Cardiac Patches for the Treatment of Myocardial Infarction, Circulation. The idea of using tissue patches to repair damaged organs is exciting. These patches are often engineered from stem cells and are sometimes referred to as stem cell patches. Such patches or strips of tissue may prove just as useful as injections of suspensions of stem cells. I am more skeptical about Band-Aid-like stem cell patches that are claimed to increase the function of endogenous stem cells.
More recommended reads
- Where Are They Now: Jake Javier, CIRM Blog. This piece also makes me think of arguably the top stem cell patient advocate ever, Roman Reed.
- World-first study to identify stem cell super donors launched, The Irish Times.
- Science had a brief item that invoked storing iPS cells from various species on the moon, which I thought was fun. See the image above.