ViaCyte (CA, USA) has announced that the University of Alberta (AB, USA) is to be the second site of the Phase I/II clinical trial investigating its VC-01â„¢ Combination Product for Type 1 diabetes, and will be led by pancreatic islets transplant pioneer Dr James Shapiro
Type 1 diabetes, which comprises 10% of all cases of diabetes, is characterized by deficient insulin production and requires daily administration of insulin. The transplantation of pancreatic islets, known as the Edmonton Protocol, has been used to successfully globally treat patients for Type 1 diabetes, in some cases for several years. However, supply of the islets from donors is limited and the procedure requires recipients to take chronic immunosuppressive drugs in order to prevent destruction of the transplant by their immune system. Therefore, the islet transplant has been limited to Type 1 diabetes patients with significant difficulty in establishing glycemic control with pharmaceutical approaches, including patients with hypoglycemia unawareness.
This procedure was developed by the laboratory of Dr James Shapiro, who holds a Canada Research Chair in Transplant Surgery and Regenerative Medicine at the University of Alberta’s Faculty of Medicine & Dentistry (AB, USA). Dr Shapiro will be the lead investigator of the second site of ViaCyte, Inc.’s (CA, USA) Phase I/II STEP ONE clinical trial investigating VC-01â„¢ Combination Product for Type 1 diabetes: the first stem cell-derived islet replacement therapy for the treatment of diabetes in clinical trials.
Dr Paul Laikind, President and CEO of ViaCyte, comments: “Dr Shapiro is one of the world’s leading experts in the field of islet transplant technologies … We welcome his participation in ViaCyte’s STEP ONE clinical trial and we are confident that his extensive experience in the field will prove to be a valuable resource as we continue the development of the VC-01 product candidate as a transformative therapy for patients with Type 1 diabetes.”
The VC-01 combination product candidate is a macroencapsulation device containing human pancreatic progenitor cells — PEC-01â„¢ cells. The cells are manufactured from pluripotent stem cells, addressing the supply issue, and the Encaptra® drug delivery system protects the cells from patients’ immune systems, addressing the immune response issue that results in islet transplant patients requiring immunosuppressants.
“Clinical islet transplantation has transformed many lives, being critical for those patients hardest hit by the disease and for whom insulin injections are insufficient,” Dr Shapiro explains. “But the fact remains that new treatments are sorely needed, not only for the high risk patients but for all patients suffering from this life-altering disease. The remarkable promise of the VC-01 product candidate is that a virtually limitless source of appropriate human cells can be transplanted without the need for lifetime immunosuppression. Should this treatment be approved, we will be far closer to a robust cure for diabetes than we have ever been in the past. I am excited to join with ViaCyte as we seek to translate the exciting results demonstrated in preclinical studies of the VC-01 product candidate into a novel new therapy that has the potential to help millions of patients.”
Launched in late 2014, the STEP ONE trial has been enrolling Type 1 diabetes patients at the University of California San Diego Health System, with the support of the UC San Diego Sanford Stem Cell Clinical Center.
The first cohort, providing a sub-therapeutic dose, has been designed to assess the safety of the product and optimize the engraftment procedures. If the first cohort meets its aims, a second cohort, receiving a therapeutic dose, will provide further safety data, as well as an initial evaluation of dosing and efficacy.