Expanded FORWARD Trial Shows Promise of Stem Cell Islet Therapy in Eliminating Insulin for T1D

At the 84th Scientific Sessions held by the American Diabetes Association (ADA) in Orlando, the latest data from the Phase 1/2 FORWARD clinical study of VX-880 islet cell therapy was unveiled. The data showed remarkable results suggesting that VX-880 can significantly reduce or even eliminate the need for insulin in patients with type 1 diabetes (T1D). This marks a potential breakthrough in restoring natural islet function and achieving better glycemic control through stem cell-derived islets.

A considerable population of type 1 diabetes patients continues to suffer from recurrent severe hypoglycemic events (SHEs) despite advancements in diabetes management technologies. Around 6% of these patients face such life-threatening events due to impaired hypoglycemic awareness, which can lead to serious complications like confusion, seizures, or even death if left untreated. Current treatment options are limited, emphasizing the need for innovative therapies.

The FORWARD study, a phase 1/2 open-label trial, included adults with type 1 diabetes who had impaired hypoglycemic awareness and had experienced at least two SHEs in the previous year. The study focused on VX-880, an investigational allogeneic, stem cell-derived, fully differentiated insulin-producing islet cell therapy. Participants had an average age of 44, a mean HbA1c of 7.8%, and used about 40 units of insulin daily. All participants had undetectable C-peptide levels at the start, indicating a lack of insulin production.

The results were promising. Participants who received a full dose of VX-880 via a single infusion showed successful engraftment of islet cells, began producing endogenous insulin, and eliminated severe hypoglycemic events. The treatment significantly improved glycemic control and reduced or entirely eliminated the need for exogenous insulin. All 12 participants achieved an HbA1c level below 7.0% and maintained a target blood glucose range over 70% of the time.

Among the 10 participants who completed the 180-day visit, seven no longer needed exogenous insulin, while two others reduced their daily insulin usage by approximately 70%. All participants with over a year of follow-up met the primary study endpoint of eliminating SHEs and achieving an HbA1c level below 7.0% at 12 months. The therapy also reached the secondary endpoint of insulin independence, underscoring the potential of VX-880 to function like natural islets and provide substantial benefits to patients.

Dr. Piotr Witkowski, a study investigator and Professor of Surgery at the University of Chicago, highlighted the significance of these findings. He stated that the data supports the potential of VX-880 to revolutionize type 1 diabetes treatment by offering an alternative to exogenous insulin. The trial's expansion to approximately 37 participants aims to generate comprehensive clinical data to support the future availability of VX-880 for type 1 diabetes patients.

The ADA's 84th Scientific Sessions, an event that brings together over 11,000 experts from around the world, provided a platform to present these findings. The symposium encourages the exchange of groundbreaking research and advancements in diabetes care, propelling the fight against this widespread disease.