A stem cell-based treatment may have cured 10 out of 12 patients with the most severe form of type 1 diabetes — during a study trial.
The experimental drug, Zimislecel, developed by Boston-based Vertex Pharmaceuticals, uses stem cells engineered to become pancreatic islet cells — the cells responsible for regulating blood sugar levels.
In the study published online by The New England Journal of Medicine, researchers recruited 14 people with type 1 diabetes complication called hypoglycemic unawareness.
People with hypoglycemic unawareness do not have any outward warning signs when their glucose (blood sugar) levels are falling, and can end up fainting, having seizures, or dying.
Advertisement
Once the drug was infused into patients, these new islet cells migrated to the liver and established themselves there.
Within a few months of receiving the treatment, participants in the study began requiring less insulin. By around six months, most had stopped needing insulin entirely.
A year after treatment, 10 of the 12 patients no longer require insulin, while the remaining two now use significantly lower doses.
Advertisement
During the study period, two people died, one due to a fungal infection that spread to the brain and another from complications of dementia that were from a pre-existing condition.
The study has been presented at the annual meeting of the American Diabetes Association.
The scientists said if the study continues to show positive results, the company will apply to the United States Food and Drug Administration in 2026.
Trevor Reichman, director of the pancreas and islet transplant programme at University Health Network, a hospital in Toronto, and lead study author, said there is a “huge unmet need” in patients who have diabetes.
Advertisement
“Current therapies like islet cell transplant is dependent on decreased donors, which are a big limiting factor. Stem-cell-derived islets provide an off-the-shelf, unlimited source of islets to treat these patients,” he said.
