Stem Cell Therapies Show Early Promise For Type 1 Diabetes
FORWARD trial and engineered stem cell data presented at ADA meeting in Chicago.
Two studies unveiled at the 85th Scientific Sessions of the American Diabetes Association in Chicago point to progress in developing stem cell-based treatments for type 1 diabetes. One study evaluated a fully differentiated, stem cell-derived islet cell therapy in adults with impaired hypoglycemia awareness. The other examined genetically modified stem cells designed to resist immune system attacks.
Insulin production restored in all patients receiving VX-880
The FORWARD study is a Phase 1/2 open-label trial evaluating the safety and efficacy of VX-880, an allogeneic islet cell therapy developed from stem cells. Twelve adult participants with type 1 diabetes received a full dose of VX-880 islets through infusion into the liver via the portal vein. All participants were on standard immunosuppressive therapy.
Treatment led to measurable restoration of endogenous insulin secretion in all individuals, determined by C-peptide detection. None experienced severe hypoglycemia events during the study. Participants achieved recommended glycemic control targets with mean insulin use reduced by 92%. Ten out of 12 participants no longer required external insulin.
Reported adverse events were consistent with those expected from the infusion procedure and immunosuppressive drug use. No additional safety concerns emerged. The study is now in Phase 3, which aims to enroll around 50 participants through 2025. A separate study is also planned to investigate VX-880 in patients who already receive immunosuppressive treatment following kidney transplant.
Immune-resistant SC-islets tested with built-in safety mechanism
The second study presented preliminary data on the use of genetically engineered human embryonic stem cells to produce islet cells resistant to immune attack. The researchers inserted eight protective genes into these stem cells, which were then differentiated into insulin-secreting islet cells. The modified cells included a safety switch that can be triggered by the antiviral drug ganciclovir to eliminate unwanted or rapidly proliferating cells.
In vitro tests showed the engineered cells consistently produced insulin and resisted killing by immune cells. The safety switch was also effective in destroying non-desirable cells in culture. Current studies in animals aim to evaluate whether this immune resistance and safety mechanism perform as expected in a living organism.
Reference: Reichman TW, Markmann JF, Odorico J, et al. Stem cell–derived, fully differentiated islets for type 1 diabetes. N Engl J Med. 2025. doi:10.1056/NEJMoa2506549
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