Trials on new islet cell therapy offer potential to reduce or stop insulin use for people with type 1 diabetes
Patients reported fewer hypoglycaemic episodes and insulin independence.
Initial trials for a new islet cell therapy for people with type 1 diabetes have revealed promising results – with a possibility that use could lead to eliminating the need to take insulin.
Vertex presented the findings on their VX-880 treatment at the recent American Diabetes Association 83rd Scientific Sessions.
The Diabetes Research Institute (DRI) at the University of Miami made a significant contribution to the presentation, led by Camillo Ricordi, M.D., Director of the Cell Transplant Center and Director Emeritus of the Diabetes Research Institute, who serves as steering committee chairperson of Vertex’s VX-880 and VX-264 trials in type 1 diabetes, and principal investigator of the University of Miami – Diabetes Research Institute Site.
Professor Ricordi said: “This is the first time that durable insulin independence has been obtained with stem cell-derived insulin-producing cells. These findings are potentially groundbreaking, and they bring us closer to a future where type 1 diabetes management can be transformed.”
VX-880 is a stem cell-derived, fully differentiated islet cell therapy, for people with type 1 diabetes with impaired hypoglycaemic awareness and severe hypoglycaemic events (low blood glucose levels).
All six patients treated with VX-880 had undetectable fasting C-peptide (endogenous insulin secretion) at baseline, a history of recurrent severe hypoglycaemic events in the year prior to treatment and required an average of 34.0 units of insulin per day.
Following treatment, all six patients demonstrated endogenous insulin secretion, improved glycaemic control as measured by HbA1c, improved time-in-range on continuous glucose monitoring, and reduction or elimination of insulin use. Patients with greater than 90 days of follow-up had elimination of severe hypoglycaemic events in the evaluation period.
VX-880 has been generally well tolerated in all patients dosed to date, with mild or moderate adverse reactions, such as dehydration, diarrhoea, hypomagnesemia and rash.
As a result of these safety reports a third trial period is anticipated, with dosing of patients at the full target dose of VX-880.
Felicia Pagliuca, Ph.D., Disease Area Executive, Type 1 Diabetes at Vertex, said: “These data represent a foundational advance in the potential treatment of T1D, bringing us one step closer to a potentially curative therapy for patients who are waiting.”
Trevor Reichman, M.D., Department of Surgery, University of Toronto, said: “The normalisation of HbA1c without the need for exogenous insulin one year after therapy with VX-880 is historic and offers hope that the transformative therapies the type 1 diabetes community has been waiting for may finally become reality.”
Professor Ricordi said: “In addition to the VX-880 trial we are happy that the VX-264 trial is now enrolling. This is an investigational cell therapy in which allogeneic human stem cell-derived islets similar to those used in the VX-880 trial are encapsulated in a channel array device designed to shield the cells from the body’s immune system. VX-264 is designed to be surgically implanted and is currently being evaluated for patients with T1D, without any anti-rejection drugs (immunosuppression).”
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