A Madison company started by a UW Health transplant surgeon aims to develop a gene therapy for type 1 diabetes, with the goal of regulating glucose metabolism without the need for daily insulin shots.
Endsulin’s experimental DNA injection is being tested in naturally diabetic dogs after showing promise in tests in rats and mice, but the road to studies in people, let alone approval, could be long. The Food and Drug Administration has approved only a few gene therapy treatments and last year rejected one for hemophilia that uses technology similar to Endsulin’s.
But Dr. Hans Sollinger sees hope for a treatment that could provide long-lasting help to the more than 1 million Americans with type 1 diabetes. The former chairman of transplantation at UW-Madison founded Endsulin in 2016 and retired as a transplant surgeon last year.
Sollinger did more than 3,500 kidney or pancreas transplants in his long career, roughly half on patients with diabetes, and saw the need for a different approach, he said. The quest is also personal, as his adopted brother died at age 31 after a kidney-pancreas transplant for complications of type 1 diabetes in Germany, where Sollinger grew up.
“We have to find something that is simple, that doesn’t cost a lot, which doesn’t need a big operation,” Sollinger said. “If we just get a little bit better (glucose) control with a simple injection, if we just make life a little bit easier (for diabetics), we are doing good.”
More than 30 million Americans have diabetes. About 1.25 million have type 1 diabetes, in which the body stops producing insulin, and must take insulin to survive. The rest have type 2 diabetes, in which they can’t use insulin properly. Some need to take insulin to stave off complications such as kidney disease, nerve damage and vision loss.
Insulin, released by the pancreas, helps turn glucose, or blood sugar, into energy and store glucose in cells.
Endsulin’s experimental gene therapy involves a string of DNA that includes insulin and a “glucose inducible response element,” or GIRE, patented by the Wisconsin Alumni Research Foundation. The DNA is packaged in and delivered by the adeno-associated virus AAV8, which is not known to cause disease, to the liver where it is designed to trigger cells to produce insulin.