Cure for Type 1?

An amazing paper appeared today in The New England Journal of Medicine.

For many years, scientists have been trying to transplant pancreatic Beta cells to cure Type 1 Diabetes. The problem is that Type 1 is an autoimmune disease, and the patient’s immune system is primed to target the transplanted Beta cells. Most protocols that have been employed use high doses of powerful immunosuppressive drugs; these drugs have many risks and side effects.

In this paper, they use genetic methods to make the donor Beta cells “hypoimmune,” meaning they will not be detected or destroyed by the immune system. They used CRISPR to inactivate two genes that are required for a cell to be recognized and destroyed by the immune system, and they also engineered the cells to overexpress a gene called CD47 that signals the immune system to “don’t eat me.”

These genetically engineered Beta cells were transplanted into a 42-year-old man with a 37-year history of type 1 diabetes. After 12 weeks, the transplanted islet cells are still active, making insulin, and the patient no longer requires insulin injections. Sounds to me like a cure.

I read online that six-month patient follow-up results were presented at the 85th Annual American Diabetes Association (ADA) meeting. So still good after 6 months.

Of course, this is only one patient. But it is very promising. Importantly this study used “Allogeneic" Beta cells, meaning the Beta cells are genetically unrelated to the recipient. Thus, they could be used for any patient.

Here is a link to the paper: https://www.nejm.org/doi/full/10.1056/NEJMoa2503822

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Now this is promising.

I feel like I’m one of the few who isn’t completely jaded on a “cure”. I’ve always believed it would happen in my lifetime. Yeah, I’ve heard the “five years away” for decades, too, but I knew that was said in ignorance when we barely even understood what diabetes was, let alone the mechanics. I knew we weren’t there yet, but that we could get there.

The first beta cell transplants were promising, but not earth shattering to me. Just felt like another stepping stone. Post Covid, especially mast cell activation and it’s long-covid symptoms, we’ve seen a lot of new research into autoimmune conditions… But this is the first time it’s really felt tangible. I’m finding myself really emotional about this one. It’s astounding, not just promising.

This is the first I’ve actually wondered what it would be like to not be consumed with this anymore? It’s such a big part of who I am after all this time.

Totally realistic, though, I feel like this is when the ten year timer starts.