Breakthrough in Type 1 Diabetes: Stem Cell-Derived Islets Offer Hope for a Cure

In a major milestone for type 1 diabetes treatment, a groundbreaking clinical trial has shown that stem cell-derived islet cell transplantation can effectively eliminate the need for insulin in patients. This advancement marks a turning point in diabetes care and highlights the efforts of leading researchers, including Dr. Piotr Witkowski at the University of Chicago, and the collaborative work of Vertex Pharmaceuticals (VRTX) and Eledon Pharmaceuticals (ELDN).

Historically, islet cell transplantation involved harvesting pancreatic islets from deceased organ donors—a limited and logistically challenging source. Patients often required multiple transplants to see long-term benefits, and donor scarcity severely limited access to the therapy. But now, lab-manufactured islets derived from stem cells are changing the game, offering a consistent, scalable, and potentially off-the-shelf solution to treating the most severe forms of type 1 diabetes.

In the ongoing multicenter clinical trial, presented at the American Diabetes Association’s Scientific Sessions and published in the New England Journal of Medicine, 10 of 12 patients remained insulin-independent for over a year after just a single infusion of stem cell-derived islets. This level of success, particularly from a single dose, is unprecedented in the field and demonstrates the power of scientific innovation to transform chronic disease management.

Dr. Witkowski, a lead investigator and transplant surgeon at the University of Chicago, has been at the forefront of this effort. His involvement traces back to the early development of the clinical protocol in 2019 with Andrea Vergani at Semma Therapeutics. After Vertex Pharmaceuticals acquired Semma, Dr. Witkowski continued his work as a member of the Scientific Advisory Board and as a principal investigator performing transplants. As of last week, his team has successfully infused their 10th patient with Vertex’s stem cell-derived islets.

While these results are promising, patients receiving the treatment still require immunosuppressive therapy to prevent rejection—typically involving drugs like tacrolimus, which can be toxic over time. That’s where Eledon Pharmaceuticals (ELDN) enters the picture. In a parallel pilot study, researchers are evaluating Tegoprubart, Eledon’s novel immunosuppressive agent designed to reduce toxicity while maintaining immune protection. If successful, Tegoprubart could offer a safer path forward and expand access to islet transplantation for a broader population.

The future is bright. Trials are now underway to test the therapy in kidney transplant recipients—patients already on immunosuppressants—providing an ideal setting to further prove the therapy’s safety and effectiveness. With continued success, stem cell-derived islets could soon become a standard-of-care treatment, immediately benefitting the 40,000+ patients in the U.S. suffering from the most unmanageable forms of diabetes.

Thanks to the visionary work of scientists, clinicians, and companies like Vertex and Eledon, what was once a highly experimental and limited therapy is on track to become a scalable medical solution. As Dr. Piotr Witkowski and his team continue their work, the path to a functional cure for type 1 diabetes is finally within reach.

Learn more about Eledon Pharmaceuticals here.

Eledon Pharmaceuticals (ELDN) – Meeting Highlights Tegoprubart Data Milestones and New Indications


Thursday, July 10, 2025

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

R&D Day Highlighted Science, Current Trials, Future Indications. We attended the Eledon R&D Day on July 9 to hear and evaluate the progress in tegoprubart development. The presentations focused on the current clinical indications in renal transplantation, islet cell transplantation, xenotransplants, and plans for liver and other solid organ transplants. Conference presentation dates for upcoming data announcements were also announced.

Phase 1b Data Update Is Planned For August. The Phase 1b open-label trial has been expanded to enroll up to 36 patients, an increase from the original 9 patients. Data is scheduled for presentation at the World Transplant Congress on August 9, 2025. Previous data presentations have included 13 patients. We expect to see follow-up data from more patients treated longer, with data from additional patients beyond the initial 12-month trial duration.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision. 

Eledon Pharmaceuticals (ELDN) – 1Q25 Reported With Phase 2 Results Expected In 4Q25


Monday, May 19, 2025

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

1Q25 Had Lower Expenses Than We Projected. Eledon reported a loss of $6.5 million or $(0.08) per share, including a gain of $10.1 million related to fair value of warrant liabilities. Without this one-time item, the loss would have been $16.6 million or $(0.21) per share, as Research and Development expense increased less from the previous quarters than we anticipated. We expect R&D to be relatively steady as the Phase 1b and Phase 2 BESTOW trials conclude later in FY2025. Cash on March 31, 2025, was $124.9 million.

Phase 1b Trial Data Expected Mid-Year. The Phase 1b open-label trial is testing an anti-rejection regimen using tegoprubart in kidney transplantation. We see this as important data, since the Phase 2 BESTOW trial is testing this regimen against the standard regimen with tacrolimus. Since historical data for the tacrolimus regimen has been published, we believe the Phase 1b data has some predictive value for the Phase 2 BESTROW trial.


Get the Full Report

Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.

This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).

*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.