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