An experimental stem cell therapy has shown promising early results in helping people with type 1 diabetes achieve insulin independence, according to findings from a recent clinical trial led by researchers at the University of Toronto.
The therapy, known as zimislecel, aims to replace the insulin-producing beta cells destroyed in type 1 diabetes.
In this early-stage trial, 14 adults with long-standing type 1 diabetes and a history of dangerous hypoglycaemia received a one-time infusion of the lab-grown islet-like cells.
The treatment was delivered directly into the portal vein of the liver and followed by an immunosuppressive regimen that did not include glucocorticoids.
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All participants had impaired awareness of low blood sugar and had experienced at least two severe hypoglycaemic events in the previous year.
They were monitored for at least 12 months following the procedure at sites across North America and Europe.
Among the 12 people who received the full dose of zimislecel, all remained free from severe hypoglycaemia for the entire year.
They also achieved significant improvements in glucose control, including average HbA1c levels under 7% and more than 70% of their time spent within the target blood sugar range of 70 to 180 mg/dL.
Notably, 10 of these participants were able to stop using insulin entirely, while the remaining two dramatically reduced their daily insulin needs.
The most common serious side effect reported was neutropenia, a temporary drop in white blood cells, seen in three participants.
Two deaths occurred during the trial—one linked to a fungal infection following off-label steroid use, and the other due to progression of a pre-existing neurological condition. Neither was attributed directly to the treatment.
Zimislecel uses stem cells to generate fully differentiated islet cells capable of sensing blood sugar and producing insulin.
If proven effective in larger and longer-term studies, this therapy could offer a scalable alternative to current transplant methods, which rely on scarce donor organs and often require multiple procedures.
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While these initial results are encouraging, researchers caution that the trial involved a small number of participants, was open-label, and lacked a control group.
A more advanced clinical trial is currently underway to better evaluate safety, long-term outcomes, and the potential for broader use.
For people living with type 1 diabetes, this therapy may eventually provide a path to greater freedom, improved quality of life, and relief from the burdens of constant insulin management.
If future studies confirm its success, zimislecel could represent a major step forward in the effort to move beyond insulin therapy and toward a functional cure for type 1 diabetes.
Read the full study in the New England Journal of Medicine.
