Positive preliminary results of an ongoing, first-in-human Phase 1/2 study demonstrating that stem cell-derived therapy can produce insulin in people with severe type 1 diabetes were published today in Cell Stem Cell and Cell Reports Medicine.
“The data from these papers represent a significant scientific advance,” said Cell Reports Medicine study lead author James Shapiro, MD PhD, Canada Research Chair and Director of the Islet Transplant Program at the University of Alberta, Canada. “It is the first reported evidence that differentiated stem cells implanted in patients can generate meal-regulated insulin secretion, offering real hope for the incredible potential of this treatment.”
The Cell Reports Medicine paper reports on 17 patients who were implanted with PEC-Direct devices — from ViaCyte, Inc., a clinical-stage regenerative medicine company focused on developing novel cell replacement therapies to address diseases with significant unmet needs — comprising pancreatic cells (PEC-01) contained within pouches designed for subcutaneous placement. Results indicate positive C-peptide levels (a biomarker for insulin) as early as six months post-implant in some patients. Initial data suggest that pancreatic endoderm cells can be differentiated into, and offer a potential scalable, renewable source of insulin-producing, pancreatic islet cells.
The Cell Stem Cell paper reports on findings from 15 patients at the trial’s largest participating site, the University of British Columbia (UBC) and Vancouver Coastal Health (VCH) in Canada. Six months after implantation, researchers observed that the cells had matured into insulin-producing islet cells. They also detected a rise in C-peptide levels after patients ate a meal, indicating functional insulin production. Furthermore, patients spent 13% more time in target glucose range, with some able to reduce the amount of their injected insulin.
“We’re highly encouraged by our initial success and are now implanting larger numbers of cells in more patients,” says David M. Thompson, MD, principal investigator of the Vancouver study highlighted in Cell Stem Cell, director of the VCH’s Vancouver General Hospital Diabetes Centre, and clinical assistant professor in the UBC Faculty of Medicine’s Division of Endocrinology. “We believe we’re on the right track toward developing therapies that can improve the lives of millions of people with type 1 diabetes worldwide.”
The international Phase 1/2 clinical trial is focused on evaluating safety, tolerability, and efficacy of PEC-Direct (VC-02) in patients with hypoglycemia unawareness or extreme glycemic lability. PEC-Direct devices are comprised of millions of pancreatic endoderm cells designed to mature into insulin-producing islet cells. The devices are configured to enable direct vascularization of the engrafted cells, therefore requiring concurrent immunosuppression.
ViaCyte is advancing PEC-Direct and its portfolio of stem cell-derived therapies with the goal of enabling patients with diabetes to regulate their blood glucose levels, improve health outcomes, and one day achieve independence from the burden of daily insulin injections.
This research was supported by funding from the Stem Cell Network, JDRF, Canadian Institutes of Health Research, and California Institute for Regenerative Medicine.
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