Pancreatic islets derived from stem cells continue to produce insulin after a year

CHICAGO – Ten people with type 1 diabetes. Moreover, who suffered from recurrent severe hypoglycemia and asymptomatic hypoglycemia, were able to do without insulin for more than a year after having followed allogenic treatment based on pancreatic islands derived from stem cells associated with immunosuppression, according to new 1/2 phase data of the multi -pole study. Meanwhile, Pharmaceuticals.

The results were presented at the annual congress of l’American Diabetes Association (ADA 2025)^[1] . Meanwhile, published in the New England Journal of Medicine .

Insulino-producer treatment VX-880, now called Zimislecel, is administered by infusion in the hepatic gate vein. Additionally, Meanwhile, An immunosuppressive treatment without corticosteroids is used. Therefore, including an induction by antithymocytic globulin followed by maintenance treatment by tacrolimus and sirolimus.

“It is really exciting to have a constant. For example, evolving source of insulin producer fabric,” said Medscape Medical News pancreatic islets derived stem cells the Dr Michael R. Moreover, Rickelsresearcher at the Faculty of Medicine of the University of Pennsylvania, in Philadelphia.

Even if immunosuppression is necessary. In addition, many patients could benefit from cell therapy, especially those who suffer from severe hypoglycemia or who have difficulty controlling their blood sugar, or those who are already immunosupplected for organ transplantation, he said.

“It will be important to have a product whose effectiveness is reproducible. Furthermore, whose security is established to test new immunomodlative approaches and, in the long term, other approaches aimed at bypassing the immune system, whether through engineering or genetic editing approaches,” added Dr. Therefore, Rickels. For example,

Questioned on this subject, Jeffrey R. Similarly, Millmanprofessor of medicine. Furthermore, biomedical engineering at the Faculty of Medicine of the University of Washington, in Saint-Louis, which contributed to the development of the technique making it possible to obtain islets from stem cells, indicated pancreatic islets derived stem cells to Medscape Medical News : “This is what we hoped, but seeing him come true is simply incredible. Furthermore, No stem cell therapy has never approached what they have managed to accomplish. Meanwhile, »»

But. Therefore, added Professor Millman, “this will only concern a small part [des personnes] Affected by type 1 diabetes, which is why we need techniques such as encapsulation or genetic engineering to avoid the immunosuppression part, in order to make it a therapy much more applicable to most or to all people with type 1 diabetes. However, “

Pancreatic islets derived stem cells

Data on 1 year

The new data complete the conclusions presented during the ADA congress last year. In addition, continue to demonstrate the feasibility of therapy for people with type 1 diabetes in whom the risk of immunosuppression prevail over the advantages.

The 14 participants (5 men, 9 women) included in the pancreatic islets derived stem cells analysis which ended a year -round follow -up had an average age of 43.6 years and an average duration of type 1 diabetes of 22.8 years. All had an undetectable P peptide at the start, an average A1C rate of 7.8 % and an average insulin daily dose of 39.3 units. All used continuous gluomes, 9 used insulin pumps and 6 used automated insulin administration systems. Despite this technology, the study participants had an average of 2.7 episodes of severe hypoglycemia during the year before screening.

All participants benefited from a transplant and infusion, as shown by the appearance of the Peptide C. Two patients received half a dose of Zimislecel and 12 received a full dose (0.8 × 10⁹ cells) in a single infusion.

After a year, none of the 14 patients had suffered from severe hypoglycemia. The 12 patients who received the full dose presented no episode of pancreatic islets derived stem cells severe hypoglycemia and had an A1C rate below 7 %. They also spent more than 70 % of the time in the target blood sugar range (70-180 mg/dl). and 10 patients were independent of insulin after 365 days.

Fourteen undesirable events have been reported, including diarrhea, headache and nausea. Most were light to moderate and attributed to immunosuppression. Neutropenia occurred at six participants. Two patients died. one of cryptococcal meningitis attributed to immunosuppression and the other of severe dementia with agitation due to the progression of a preexisting neurocognitive deficiency. These deaths led to a temporary interruption of research at the start of 2024.

“The results of this small short -term study carried out with people with type 1 diabetes support the hypothesis that Zimislecel can restore the physiological function of the pancreatic islets. which justifies the continuation of clinical research,” concluded the authors.

The Zimislecel will therefore be subject pancreatic islets derived stem cells to a phase 3 test. with a planned recruitment of 50 patients, which should end at the end of the summer of 2025.

Pancreatic islets derived stem cells

Overcome the need for immunosuppression

Professor Millman said that it was not optimistic. about the potential of the techniques of encapsulation of the pancreatic islets – several of which were discussed at the ADA meeting – to overcome the need for immunosuppression.

“Encapsulation is promising in the sense that it is relatively simple in its concept. its execution, but it has proven to be very difficult to implement in the past,” he said.

“The problem is that it takes a certain amount of islets producing a certain amount of insulin to control. blood sugar in an adult. These cells have specific metabolic needs in glucose for oxygen they breathe. and if you encapsulate them, they can no longer count on blood pancreatic islets derived stem cells vessels to provide them with the nutrients and oxygen they need, “he explained.

He added: “Perhaps there are more advanced methods to overcome these obstacles. but so far, no conclusive evidence has shown that this could be implemented in a manner applicable to patients. Indeed. in March 2025, Vertex interrupted a 1/2 phase test on a product based on encapsulated islets, the VX-264, due to a lack of efficiency.

Professor Millman. other specialists in the field are rather optimistic about the possibility of genetically modifying pancreatic islets to avoid the use of immunosuppressive drugs. “Many interesting scientific works have been carried out by university companies. laboratories on different methods of genetic modification of cells in order to avoid destruction by the immune system,” he said.

Although this research is still at their beginnings. Professor Millman spoke of upcoming programs, such as that announced by Sana Biotechnology, whose six -month results pancreatic islets derived stem cells were presented at the ADA meeting^[2].

Indeed. the company announced the positive clinical results six months of a phase 1 study on the transplantation of cells of the pancreatic islets without immunosuppression.

The technology used is called UP421. It is an allogenic therapy based on modified primary pancreatic islands using Hyaimmune technology (HIP) of Sana.

Thanks to the genetic reprogramming technique. researchers, using molecular scissors technique, cut and eliminate DNA from surface antigens, explained the Pre Laurence Kessler Diabetologist at Strasbourg University Hospitals and researcher in the Inserm UMR 1260 unit, regenerative nanomedicine, at Medscape French edition.

Allogenic therapy has been transplanted in a patient with type 1 diabetes without immunosuppression. The study is carried out in partnership with the Uppsala University Hospital. The results 6 months after cell transplantation are consistent with the clinical results previously reported to four and twelve weeks. They show that modified pancreatic beta cells pancreatic islets derived stem cells survive and operate. The presence of peptide C circulating, indicates that the transplanted beta cells produce insulin. Peptide C levels also increase during a tolerance test at the mixed meal (MMTT). which corresponds to insulin secretion in response to a meal. An MRI-TEP carried out 12 weeks after the transplant also highlighted the presence of cells in the pancreatic islets at the transplant site. a forearm muscle. The study did not identify any security problems. and cells of the pancreatic islets modified by HIP technology have escaped immune responses.

This article was translated and adapted from Medscape.com, supplemented by Aude Lecrubier.