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Cure & Advancements/August 13, 2025/4 min read

Stem Cell-Derived Islets Show Promise in Early Type 1 Diabetes Studies

Recent clinical trials reveal that transplanted insulin-producing cells grown from stem cells can help some people with Type 1 diabetes achieve better blood sugar control. Researchers are testing different approaches to make these treatments safer and more practical.

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Key takeaways

  • Early-stage studies show that stem cell-derived islet transplants can help people achieve insulin independence and near-normal blood sugar levels for at least one year.
  • Immunosuppressive medications remain necessary for most stem cell transplant approaches, though researchers are working on gene-edited versions that may not require them.
  • These are promising first-in-human and early clinical trials with small numbers of participants; larger, longer studies are needed before wider use.
  • Different stem cell approaches are being tested, including chemically induced pluripotent stem cells and genetically modified donor cells.

What Are Stem Cell-Derived Islets?

Stem cell-derived islets are insulin-producing cells created in the laboratory from stem cells. Rather than relying on donor pancreases—which are in short supply—researchers grow these cells from biological starting material that can be expanded and refined. The goal is to create a renewable source of functional islets that can be transplanted into people with Type 1 diabetes to restore natural insulin production.

Several approaches are being explored. Some use chemically induced pluripotent stem cells, which are reprogrammed adult cells converted back into a primitive state and then guided to become islet cells. Others involve genetically modifying donor islet cells to reduce the risk of rejection. Each method aims to solve the core problem: helping the body maintain healthy blood sugar levels without daily insulin injections.

What Do Early Trials Show?

A groundbreaking case report published in Cell tracked one patient who received a transplant of chemically induced pluripotent stem cell-derived islets. Starting 75 days after the procedure, this patient achieved sustained insulin independence. Four months post-transplant, their time in the target blood sugar range jumped from 43% to over 96%, and their glycated hemoglobin (a marker of long-term glucose control) reached non-diabetic levels around 5%. After one year, these improvements remained stable with no signs of transplant-related complications.

A phase 1-2 trial published in the New England Journal of Medicine examined zimislecel, an allogeneic stem cell-derived islet therapy, in 14 participants who completed at least 12 months of follow-up. The study tested whether the transplant could prevent severe low blood sugar episodes and improve glycemic control while avoiding immunosuppression. Results showed evidence of engraftment and islet function, though detailed outcome breakdowns were still under analysis at the time of publication.

Another study published in the New England Journal of Medicine explored a different strategy: genetically edited donor islet cells designed to avoid immune rejection. One participant received this transplant directly into forearm muscle without any immunosuppressive drugs. At 12 weeks, the transplanted cells showed stable, glucose-responsive insulin secretion, and the patient developed no immune response against them. Four adverse events occurred, but none were serious or drug-related.

What Challenges Remain?

Most stem cell-derived islet approaches still require immunosuppressive medications—drugs that dampen immune system activity to prevent the body from rejecting foreign cells. These medications can cause significant side effects and require ongoing monitoring. Researchers are actively working on gene-editing strategies to create cells the immune system will tolerate without suppression, as shown in the forearm transplant trial.

These remain early-stage studies with very small numbers of participants and relatively short follow-up periods. The one-year outcomes are encouraging, but long-term durability, safety, and effectiveness remain unknown. Larger, longer trials are needed to understand how many people might benefit, which approach works best, and whether the benefits persist over many years.

What's Next?

Multiple clinical trials are ongoing to test stem cell-derived islet transplants in more people with Type 1 diabetes. Researchers are refining the cells, optimizing transplant locations and techniques, and exploring whether immune tolerance can be achieved without long-term immunosuppression. The goal is to develop a practical, safe therapy that can be offered more widely than current islet transplantation, which is limited by the scarcity of donor organs.

These advances represent meaningful progress in Type 1 diabetes research. While stem cell-derived islet therapy is not yet a standard treatment, the early clinical data suggest the approach warrants continued investigation. Anyone interested in these trials or considering participation should speak with their healthcare team about current opportunities and what these studies can and cannot yet guarantee.

Evidence label

Origin: YouTube / Taking Control Of Your Diabetes® (Video report). Evidence: Video report, corroborated with 5 indexed studies. Type1Cure is an information and intelligence hub, not a medical advice service. This article summarizes published research and does not provide diagnosis, treatment, or personal medical guidance. Always talk to your own care team before changing anything about your Type 1 diabetes management.

Type1Cure is an information and intelligence hub, not a medical advice service. This article summarizes published research and does not provide diagnosis, treatment, or personal medical guidance. Always talk to your own care team before changing anything about your Type 1 diabetes management.

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