Stem cell therapy could be breakthrough against type 1 diabetes

An experimental stem cell therapy can essentially cure type 1 diabetes by restoring insulin production in some patients, early clinical trial results show.

Seven out of 12 patients no longer needed daily insulin shots after receiving a full dose of the gene therapy, dubbed VX-800, researchers reported Friday at the American Diabetes Association’s annual meeting in Orlando, FL.

Another two needed about 70% less insulin daily to keep their blood sugar stable, results show.

“This positive data adds to the growing body of evidence for VX-880’s potential to revolutionize the treatment of type 1 diabetes,” said researcher Dr. Piotr Witkowski, director of the pancreatic and islet transplant program at the University of Chicago.

People with type 1 diabetes aren’t able to produce enough insulin to keep blood sugar levels stable. Type 1 diabetes occurs when the immune system mistakenly targets and attacks the islet cells in the pancreas that generate insulin.

VX-880 works by introducing fresh islet cells that have been derived from stem cells, with the aim that those cells will restore pancreatic function.

For the early-stage clinical trial, researchers recruited 12 people with poorly controlled type 1 diabetics. All had average hemoglobin A1C levels of 7.8%, a level at which there’s an increased risk of complications from diabetes. They’d also experienced severe hypoglycemia two to four times in the prior year, and used about 40 units of insulin a day to try and stabilize their blood sugar.

A single infusion of VX-880 eliminated severe hypoglycemic events in all 12 patients and drove their hemoglobin A1C levels below 7%, results show.

The findings indicate that the VX-880 stem cell-derived islet cells function like a person’s own islet cells, researchers concluded. The trial has been expanded to enroll a total of 37 participants, researchers said.

“We hope to see this treatment become a pivotal development in type 1 diabetes care,” Witkowski said in a meeting news release.

Still, because these findings were presented at a medical meeting, they should be considered preliminary until published in a peer-reviewed journal.

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