Type 1 diabetes is an autoimmune disease where insulin-producing pancreatic beta cells (often called just “islets,” because they reside in the islets of the Langerhans part of the pancreas) are destroy by the body’s own immune reactions. People with the condition take insulin via shots or a pump to stay alive and must also regularly measure their blood sugar levels and adjust their insulin.
But some people with type 1 diabetes often have severe low blood sugar (hypoglycemia) and may not feel the symptoms, such as shaking and sweatiness, that signal a blood sugar drop. The only candidates for islet cell therapy, those who are unaware of their hypoglycemia, must additionally take immunosuppressive medicine, which carries risks similar to those of other donated organs like a kidney. Researchers are working to end the need for immunosuppressants.
In clinical trials with Lantidra, 21 out of 30 patients did not need to take insulin for at least 1 year, while 10 were still insulin-independent more than 5 years after the treatment. But in five patients, it didn’t work at all.
Meanwhile, in an early clinical trial of a different type of pancreatic islet cell made from stem cells, Vertex Pharmaceuticals’ VX-880, two people with type 1 diabetes and severe hypoglycemia have been able to completely come off insulin for at least a year, and three others are moving in that direction. Those findings were present on June 23 at the annual American Diabetes Association’s Scientific Sessions.
“For decades now, the promise of pancreatic islet transplantation as a treatment for a small subset of those with the most difficult-to-control type 1 diabetes – and in particular, those with frequent and severe hypoglycemia – has faced two major hurdles,” said David M. Harlan, MD, co-director of the University of Massachusetts Diabetes Center of Excellence.
“One is an insufficient supply of islets to transplant, and two, the sometimes-toxic immunosuppression required to prevent the transplanted islets from immune rejection,” he said.
Harlan, a professor of medicine at the University of Massachusetts Chan Medical School in Worcester, stated that VX-880 recent results hold the promise of overcoming both hurdles. They demonstrate the potential of growing stem cell-derived islets in the lab, opening the possibility of a virtually endless supply.
With Lantidra, side effects included nausea, fatigue, anemia, and belly pain. Most people in the trial had at least one severe adverse reaction, due to either the IV procedure into the portal vein or from the immune-suppressing medications. In some cases, these events required the person to stop using those medications and lose the function of the transplanted cells.
“These adverse events should be consider when assessing the benefits and risks of Lantidra for each patient,” the FDA said in a statement.