European Clinical Study Reports that Stem Cell-Derived Implants in Type 1 Diabetes Recipients Produce Insulin under Glucose Regulation
– Potential Functional Cure for Type 1 Diabetes Patients –
BRUSSELS, June, 2021
The Center for Beta Cell Therapy in Diabetes, coordinator of an international consortium, announces the first results from its EU-supported clinical study in which a stem cell-derived therapy product from ViaCyte is implanted in type 1 diabetes patients who had lost their insulin-producing beta cells. Six months after implantation, three of the first four recipients produce insulin under glucose regulation, which is needed for control of blood glucose.
This clinical study tests one of ViaCyte’s cell therapy product candidates, called PEC-Direct, which consists of stem cell-derived pancreatic progenitor cells contained in an open encapsulation device. Once implanted under the skin, the cells mature into pancreatic endocrine cells, which includes insulin-producing beta cells and glucagon producing alpha cells that control blood glucose levels. This process is detected and followed by measuring newly formed C-peptide in the circulation, a biomarker molecule that is co-produced with insulin and thus indicative for the formation of this hormone by the implanted cells.
Potential functional cure for type 1 diabetes patients
In preclinical models and initial clinical studies, implants of PEC-Direct have been shown capable of producing insulin and other hormones in response to blood glucose levels. Uncontrolled blood glucose can be life-threatening, and a beta cell replacement therapy could provide a functional cure.
The Center’s clinical study represents European participation in a trial currently being conducted in the U.S. and Canada. The goal of the study is to optimize engraftment and formation of a functional beta cell mass. Patients eligible for enrollment are subject to inclusion and exclusion criteria that are listed on the website of the Belgian Diabetes Registry (www.bdronline.be).
Implantations are carried out at UZ Brussel, the University Hospital of Vrije Universiteit Brussel-VUB, with the PEC-Direct product candidate from ViaCyte. The clinical trial, and associated preclinical studies, are undertaken by the BetaCellTherapy Consortium, with support of a Horizon 2020 grant from the European Commission. The consortium is composed of clinical, industrial, and research teams at VUB, ViaCyte, San Raffaele Hospital Diabetes Research Institute in Milan, Nestlé Institute of Health Sciences in Lausanne, University Medical Center in Leiden, and Institut du Cerveau et de la Moelle Epinière in Paris. These teams have a longstanding collaboration on development of a cell therapy for curing type 1 diabetes.
Type 1 diabetes can appear at any age but is the major form of diabetes diagnosed under age 40. Patients can no longer produce insulin and therefore become dependent on life-long daily insulin administration. Treatment with exogenous insulin does not eliminate risks for complications, some potentially life-threatening. The disease also has a significant impact on quality of life. Beta cell implants prepared from human donor pancreases can restore endogenous insulin production and glucose control, but shortage of human donor organs limits implementation of this form of cell therapy. Human pluripotent stem cells may overcome these limitations as they represent a potential large-scale cell source that can be differentiated into pancreatic cells in the laboratory under highly controlled conditions and applied to cell-based treatments.
About the Center for Beta Cell Therapy in Diabetes
The Center for Beta Cell Therapy in Diabetes is a coordination core for studies and interventions that aim prevention and cure of type 1 diabetes. The objective of the projects is to preserve or to replace a functional beta cell mass that can achieve long-term metabolic control in patients. They are undertaken by an international consortium with partners from research institutions, university hospitals, and bio-industry. Since its inception in 2002, the Center and its consortium have received support from the European Union through FP6, FP7, and Horizon 2020 programs, from JDRF (formerly known as the Juvenile Diabetes Research Foundation) and from Flanders Innovation & Entrepreneurship. The Center is based on the Medical Campus of Vrije Universiteit Brussel. For the present program on stem cell-derived implants in diabetes, it coordinates activities by partners in Brussels (UZBrussel and VUB), San Diego (ViaCyte), Milan (San Raffaele Hospital Diabetes Research Institute), Lausanne (Nestlé Institute of Health Sciences), Leiden (University Medical Center), and Paris (Institut du Cerveau et de la Moelle Epinière). For more information, please visit www.betacelltherapy.org.
Center for Beta Cell Therapy in Diabetes
Brussels Health Campus Vrije Universiteit Brussel
Laarbeeklaan 103, 1090 Brussels
About ViaCyte
ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies based on two major technological advances: cell replacement therapies derived from pluripotent stem cells and medical device systems for cell encapsulation and implantation. ViaCyte has the opportunity to use each technology individually or together to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins. The company’s first product candidates are being developed as potential long-term treatments for type 1 diabetes patients to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. To accelerate and expand the company’s efforts, ViaCyte has established collaborative partnerships with leading companies, including CRISPR Therapeutics and W.L. Gore & Associates. ViaCyte is headquartered in San Diego, California. For more, please visit www.viacyte.com and connect with ViaCyte on Twitter, Facebook, and LinkedIn.
ViaCyte Media Contact:
Lazar-FINN Partners
Glenn Silver
1 (973) 818- 8198