This is a summary of a Phase 1/2 trial of a cell therapy meant to stimulate insulin production in diabetes patients. The top line is that it met its clinical endpoints and several of the patients no longer require external insulin dosing at all and their blood sugar is well managed. Vertex also has a number of follow-up therapies in the pipeline meant to improve upon this therapy.
I personally don’t have much experience in cell therapy, but the potential of a treatment like this is clear. We will see what the data looks like as this goes through the clinical trial pipeline and expands the patient population in Phase 3.
Unfortunately, this requires immunosuppressives in order to function, which is far worse than having t1 diabetes and a good cgm with auto mode.
There have been plenty of promising studies for making cells produce insulin again, but the crux of all treatments is getting the immune system to stop attacking the insulin producing cells; until that happens, any treatment outside of regular insulin injections is moot.
That is true. Vertex claims that some of the follow-up therapies to this do not require immunosuppressants, so time will tell.
From a strategic perspective, I wonder if they will proceed with Phase 3 or not. I have worked on several programs in the past where we pushed through to Phase 2 to get a proof of concept in humans before scrapping the program because we have a better version of the molecule (using the same mechanism of action) getting ready right behind it. This therapy from Vertex may have proven the concept to allow a better version to come next.