This first-of-its-kind therapy cured type 1 diabetes
Industry Buzz
It’s remarkable to see 12 out of 12 patients with baseline HbA1c above 7% and multiple severe hypoglycemic events reach consensus targets for glycemic control by both HbA1c and time in range as well as elimination of severe hypoglycemic events.
—Michael Rickels, MD, endocrinologist
When news broke in late 2024 that a 25-year-old woman in China had stopped requiring insulin injections after receiving insulin-producing cells made from her own body, many in the diabetes community were skeptical. Could this really be a cure? Or just a rare fluke?
A year later, the field has more data and more ambition, and it’s worth stepping back to ask: How close are we to a scalable, durable therapy? And might this success in diabetes hint at what cell therapy could become in Alzheimer’s?
The first patient
In a study led by Peking University and Tianjin First Central Hospital, investigators reprogrammed adipose (fat) cells from a woman with long-standing type 1 diabetes into induced pluripotent stem cells (iPSCs). []
The researchers then differentiated those into islet-like clusters and transplanted them beneath her abdominal rectus sheath. The cells engrafted, grew vasculature, and began producing insulin.
According to the report, by day 75, she no longer required exogenous insulin, and at the 1-year mark, she remained insulin-independent with stable glycemic control.
Before the transplant, her “time in range” (TIR) was nearly 43%, meaning this was the percentage of time her glucose levels were within the target range. Within 4 months, it jumped to over 96%, and she had no more severe hypoglycemic events.
The team emphasized the challenges ahead. “The trial is ongoing and includes three patients in total. After interim analysis of the first patient and the submission of this work, the second and third patients were enrolled," the authors wrote. "Follow-up with these patients is ongoing, as they were sequentially enrolled in accordance with regulatory safety requirements.”
According to lead author Hongkui Deng, PhD, "long-term follow-up of at least two years will be conducted.”
However, scaling individualized iPSC-based therapy and managing immune attack are existing hurdles for this therapy.
Related: New study may help diagnose this rare, fatal disease decades before symptoms appearExciting new trial results
The most significant advance in the past year comes from Vertex Pharmaceuticals and their stem cell–derived islet product, zimislecel (also known as VX-880).
Rather than making patient-specific islets, the approach produces a consistent, allogeneic supply that can be infused into patients under immunosuppression.
At ADA 2025, Vertex presented results from the Phase I/II portion of their FORWARD-101 clinical trial, which were simultaneously published in The New England Journal of Medicine. [][]
Among 12 participants treated with the full dose and followed for ≥1 year:
All 12 achieved HbA1c <7% and >70% time in range.
10 of 12 (approx. 83%) remained insulin-independent at day 365.
Severe hypoglycemia events were eliminated after day 90.
The average reduction in daily insulin dose across the cohort was over 92%.
“It’s remarkable to see 12 out of 12 patients with baseline HbA1c above 7% and multiple severe hypoglycemic events reach consensus targets for glycemic control by both HbA1c and time in range as well as elimination of severe hypoglycemic events," Michael Rickels, MD, a lead investigator, said. []
The success, however, comes with trade-offs. Some adverse events (AEs) were related to the immunosuppressive regimen; neutropenia was the most frequent serious AE, occurring in three participants (in a broader safety set).
There were two deaths in the overall program: One from cryptococcal meningitis (thought to be related to immunosuppression) and another in the Phase I study attributed to dementia progression; neither was adjudicated as directly caused by zimislecel. [][]
Vertex has already advanced to a Phase III trial, aiming to enroll around 50 participants globally by the end of 2025. They plan to track the proportion who remain insulin-independent and free of hypoglycemia at 1 year after achieving insulin independence. Safety will be monitored for up to 5 years. []
Evolving therapies
1. Encapsulation/extra-hepatic niches
Vertex had also been developing VX-264, an encapsulated-cell variant intended to avoid systemic immunosuppression, but that program was terminated in 2025 in favor of focusing on zimislecel. []
Meanwhile, platforms like Sernova’s Cell Pouch (a pre-vascularized, subcutaneous device) have shown promising interim results.
Some patients achieved insulin independence with donor islets placed into the pouch. The idea is to protect the cells locally and limit systemic immune exposure. []
2. Immune-evasive gene editing
One of the field’s biggest challenges is how to deliver functional islets without the need for chronic immunosuppression.
In 2025, reports are also emerging of CRISPR-edited islets that evaded immune detection while producing insulin []
3. Adjunct immunomodulation
Islet transplantation (donor or stem cell–derived) traditionally relies on anti-rejection regimens with calcineurin inhibitors, mTOR inhibitors, steroids, etc.
But newer agents, such as anti-CD40L antibodies (eg, tegoprubart), are being tested in combination with islet transplant settings.
In a recent islet cell transplant trial (not stem cell–derived), two patients achieved insulin independence using an anti-CD40L antibody without tacrolimus, suggesting that refined immunomodulation may enable safer long-term suppression. []
It's also worth noting that the FDA approved Lantidra (donislecel) in 2023, the first allogeneic cellular therapy for type 1 diabetes, although it utilizes cadaveric islet cells (not stem cell–derived) and is limited by donor islet availability. []
4. Immunotherapy and remission extension
While cell replacement is a “fix,” preserving native or newly introduced islets is equally critical. Therapies like teplizumab (Tzield) have gained traction: in high-risk individuals (stage 2 type 1 diabetes), teplizumab has delayed progression to clinical disease. []
In newly diagnosed patients, baricitinib (a JAK1/2 inhibitor) has been shown in a randomized trial to preserve C-peptide and reduce insulin needs. []
Related: A new clue in Alzheimer’s could rewrite what we know about the diseaseA brief note on Alzheimer’s
In contrast to the well-defined endocrine deficit in type 1 diabetes, Alzheimer’s disease involves widespread neurodegeneration with multifactorial pathology.
Small Phase 2a studies of mesenchymal stem cells (e.g., Lomecel-B) have shown promising signals, including reduced hippocampal atrophy and exploratory cognitive improvements. However, effect sizes and reproducibility remain modest. []