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Stem Cell-Derived Islets and Type 1 Diabetes: Restoring the Body’s Ability to Make Insulin

Can the Body Make Insulin Again?

For people with type 1 diabetes, daily insulin is essential because the immune system damages the pancreatic cells responsible for producing it. Researchers have been exploring whether regenerative medicine could someday replace some of those lost insulin-producing cells.

A first-in-human Phase I study published in Cell took an exciting step in that direction. Researchers created new insulin-producing cells from a patient’s own reprogrammed cells and transplanted them back into her body.

Turning a Patient’s Own Cells Into Insulin-Producing Cells

The process started with cells collected from the patient’s own tissue. Researchers used small chemical molecules to reprogram those cells into chemically induced pluripotent stem cells, or CiPSCs.

Pluripotent stem cells have the ability to develop into many different cell types. In this case, the research team guided them into becoming islet-like cells, similar to the cells in the pancreas that produce insulin.

The finished cells were then transplanted beneath the abdominal muscle covering, an area that allowed researchers to monitor the transplant while giving the new cells a place to survive and function.

From Daily Insulin to Insulin Independence

The study reported results from the first patient enrolled in the ongoing clinical trial. Before treatment, the patient had long-standing type 1 diabetes that was difficult to control and had experienced episodes of dangerously low blood sugar. After the transplant, her insulin needs gradually began to decrease.

By 75 days after treatment, she was no longer using outside insulin. Even more encouraging, that insulin independence continued through the study’s one-year follow-up.

Blood Sugar Control Improved Dramatically

Researchers also used continuous glucose monitoring to see how often the patient’s blood sugar remained within the desired range.

Before treatment, she spent about 43% of her time in the target blood sugar range. Four months after the transplant, that number had increased to approximately 96%.

For the final eight months of follow-up, her time in range stayed above 98%. Her HbA1c, a common measurement of average blood sugar over time, also dropped from 7.57% before treatment to around 5%, remaining in the non-diabetic range later in follow-up. No severe low-blood-sugar events were reported during the year following transplantation.

Why Using the Patient’s Own Cells Is Interesting

One of the most fascinating parts of this approach is that the transplanted cells were created specifically for the patient.

Rather than using insulin-producing cells from an outside donor, researchers created new islet-like cells from cells originating from her own body. This personalized approach could offer researchers another path toward developing replacement cell therapies for diabetes.

There is an important detail, though. The patient had previously received a liver transplant and was already taking immune-suppressing medications, which she continued after the stem-cell-derived islet transplant. Because of that, this study cannot tell us whether similar cells could be transplanted into other patients without immune-suppressing treatment.

What Did the Study Show About Safety?

At the one-year follow-up, the patient had met the study’s planned safety and effectiveness goals, and researchers reported no signs of transplant-related abnormalities.

That is encouraging for a first-in-human study, but the findings still need to be viewed in context. This report describes the results of just one patient, making it far too early to know whether other people would experience the same outcome.

More participants and longer follow-up will be essential for understanding the treatment’s safety, consistency, and durability.

A Remarkable First Step

The most striking part of this study is easy to understand: researchers created insulin-producing cells from a patient’s own reprogrammed cells, transplanted them into her body, and 75 days later she stopped needing insulin injections.

One year after treatment, she remained insulin-independent, spent more than 98% of her time within the target blood sugar range, and maintained HbA1c levels around 5%.

It is only one patient, so much more research is needed before this could become an established treatment for type 1 diabetes. Still, the study provides an exciting example of what regenerative medicine is working toward, using specially developed cells to restore a function the body had previously lost.

Source

Wang S, Du Y, Zhang B, et al. Transplantation of chemically induced pluripotent stem-cell-derived islets under abdominal anterior rectus sheath in a type 1 diabetes patient. Cell. 2024;187(22):6152–6164.e18. doi: 10.1016/j.cell.2024.09.004. Available from: https://www.cell.com/cell/fulltext/S0092-8674(24)01022-5

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