Groundbreaking Findings on Stem Cell-Derived Insulin-Producing Cells Presented at the 2023 American Diabetes Association Scientific Sessions
The Diabetes Research Institute (DRI) at the University of Miami made a significant contribution in a clinical trial presented at the American Diabetes Association 83rd Scientific Sessions in San Diego, California, where groundbreaking results were presented from the VX-880 Phase 1/2 Clinical Trial (Vertex Pharmaceuticals Incorporated).
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, this Phase 1/2 study focused on an investigational therapy utilizing fully differentiated stem cell-derived islet cells in patients with type 1 diabetes with impaired hypoglycemic awareness and experienced severe hypoglycemic events.
Vertex Pharmaceuticals’ VX-880 trial treated a total of six patients who had undetectable fasting C-peptide (a marker of endogenous insulin secretion) at the beginning of the trial, along with a history of recurrent severe hypoglycemic events and a requirement for an average of 34.0 units of insulin per day. After the treatment, all six patients demonstrated a restoration of endogenous insulin secretion, improved glycemic control as indicated by HbA1c levels, enhanced time in the target glucose range according to continuous glucose monitoring, and a reduction or elimination of the need for external insulin. Patients who were followed for more than 90 days experienced no severe hypoglycemic events during the evaluation period.
“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 T1D management can be transformed. I am grateful to the DRI-Clinical Cell Transplant Team, led by Dr. Rodolfo Alejandro and Dr. David Baidal, that played a crucial role, diligently screening potential candidates to identify those suitable for the study, and to Dr. Raffaella Poggioli MD, who provided exceptional clinical support”, stated Dr. Ricordi.
The study was divided into two parts: Part A involved administering a half-target dose of VX-880 and monitoring patients for approximately nine months, while Part B involved administering the full target dose of VX-880 as a single infusion. Based on the favorable safety and efficacy data from Parts A and B, the study is now progressing to Part C, which allows for concurrent dosing of patients at the full target dose of VX-880.
The patient treated at the DRI who was allocated into Part B and was the first patient receiving the full dose of stem cell-derived islets who achieved insulin independence, successfully meeting the one-year primary endpoint, with reduced HbA1c levels of 6.0%, and improved glucose control within the target range.
The findings offer hope for individuals with type 1 diabetes, potentially paving the way for a transformative approach to managing the disease. Further research and clinical trials are needed to validate these initial results and ensure the long-term safety and effectiveness of this innovative therapy.
For more information on the Vertex results, visit https://news.vrtx.com/news-releases/news-release-details/vertex-presents-positive-vx-880-results-ongoing-phase-12-study.
About the Diabetes Research Institute (DRI) and Foundation
The Diabetes Research Institute (DRI) and Foundation were created for one reason – to cure diabetes – which is and will continue to be the singular focus until that goal is reached. As one of the largest and most comprehensive research centers dedicated to curing diabetes, the DRI is working to restore natural insulin production and normalize blood sugar levels without imposing other risks. Researchers have already shown that transplanted insulin-producing islet cells allow people with type 1 diabetes to live without the need for insulin injections. Some of the DRI’s islet transplant patients have been free from insulin for more than 15 years. Funding provided by the Foundation is the driving force that allows DRI scientists to pursue new and innovative ideas, and to bring these discoveries to patients. The Diabetes Research Institute Foundation is the organization of choice for those who want to Be Part of the Cure. For more information, please visit DiabetesResearch.org or call 800-321-3437.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency.
Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters is now located in Boston’s Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry’s top places to work, including 13 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.