A single infusion of a stem cell-based treatment may have cured 10 out of 12 people with the most severe form of type 1 diabetes. One year later, these 10 patients no longer need insulin. The other two patients need much lower doses.
The experimental treatment, called zimislecel and made by Vertex Pharmaceuticals of Boston, involves stem cells that scientists prodded to turn into pancreatic islet cells, which regulate blood glucose levels. The new islet cells were infused and reached the pancreas, where they took up residence.
The study was presented on Friday evening at the annual meeting of the American Diabetes Association and published online by The New England Journal of Medicine.
"It's trailblazing work", said Dr Mark Anderson, professor and director of the diabetes centre at the University of California in San Francisco. "Being free of insulin is life changing", added Dr Anderson, who was not involved in the study.
Vertex, like other drug companies, declined to announce the treatment's cost before the Food and Drug Administration approves it.
A Vertex spokeswoman said the company had data only on the population it studied so it could not yet say whether the drug would help others with type 1 diabetes.
About two million Americans have type 1 diabetes, which is caused when the immune system destroys islet cells. A subset of islet cells, the beta cells, secrete insulin. Without insulin, glucose cannot enter cells. Patients with type 1 must inject carefully calibrated doses of the hormone to substitute for the insulin their body is missing.
Type 1 is different from the more common type 2 diabetes, which usually occurs later in life.
Controlling insulin levels is a constant, often costly effort. Elevated blood sugar levels can damage the heart, kidneys, eyes and nerves. But if levels fall too low, people can feel shaky, pass out or have seizures. The patients in the study are among the estimated 30 per cent with a complication of type 1 diabetes — hypoglycemic unawareness. Those with the condition have no warning when their glucose levels are falling precipitously.
New York Times News Service