The breakthrough uses stem cells to treat type 1 diabetes
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VX-880 stem cell therapy led to 92% reduction in insulin use, with 10 out of 12 patients eliminating injections entirely in a Phase 1/2 study.
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Genetically engineered islet cells with immune evasion and a kill switch show promise for safer, longer-term treatment of type 1 diabetes.
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Studies presented at the American Diabetes Association's 85th Scientific Sessions underscore groundbreaking advances in potential cures for insulin-dependent diabetes.
A pair of studies presented at the 85th Scientific Sessions of the American Diabetes Association (ADA) have detailed major breakthroughs in the use of stem cell therapies to treat type 1 diabetes without the use of insulin shots.
The findingsone featured as an oral presentation and simultaneously published in the New England Journal of Medicine, the other as a late-breaking posterdemonstrate the latest progress toward viable alternatives to insulin injections, a century-old standard of care.
The FORWARD clinical study, led by Vertex Pharmaceuticals, evaluated VX-880, an allogeneic stem cell-derived islet cell therapy aimed at restoring the bodys own insulin production. In a Phase 1/2 open-label trial involving 12 adults with T1D and impaired awareness of hypoglycemia, all participants saw significant improvement in glycemic control. Tn subjects completely eliminated the need for exogenous insulin, while all patients achieved target A1C levels and spent more than 70% of time in range.
These findings indicate the potential for a novel cellular therapy that restores endogenous insulin secretion to improve outcomes for type 1 diabetes patients who have been struggling to achieve glycemic control, said lead investigator Dr. Michael Rickels of the University of Pennsylvanias Perelman School of Medicine.
How it works
Participants received the VX-880 product via a portal vein infusion to the liver, along with a standard immunosuppression regimen. Safety outcomes aligned with those typically seen in islet cell transplant procedures, and no unexpected adverse events were reported.
The therapy is now progressing to a Phase 3 trial, targeting 50 participants, with plans for a specialized sub-study involving kidney transplant recipients already on immunosuppressants.
In a separate late-breaking study, researchers produced promising results from genetically engineered human embryonic stem cell (hESC) lines that have been modified to evade immune detection. By adding eight protective genes to the stem cell-derived islet (SC-islet) cells and incorporating an inducible kill switch, scientists have developed a treatment designed to resist immune attack while remaining safely controllable.
Researchers said the specialized cells successfully produced insulin in lab settings and exhibited robust resistance to immune system activation and cell death. The kill switchtriggered by the common FDA-approved antiviral drug Gancicloviradds an important safeguard against unwanted or abnormal cell growth, they said.
These early results show potential for safer, longer-term cell therapy for diabetes as we look to provide patients with solutions without the need for harmful immune-suppressing drugs, said Dr. Jia Zhao, postdoctoral researcher at the University of British Columbia and lead presenter of the study.
Posted: 2025-06-24 11:19:45
