ViaCyte, a beta cell replacement company long supported by Breakthrough T1D, has shown—for the first time ever—that its PEC-Direct therapy helps people with type 1 diabetes (T1D) produce insulin again. Preliminary data from ViaCyte’s clinical trial show that when pancreatic precursor cells, called “PEC-01 cells,” are implanted under the skin and properly engrafted, they are capable of producing circulating C-peptide, a biomarker for insulin, in people with T1D. This is a significant milestone for the company, Breakthrough T1D and the T1D community.

“ViaCyte has achieved a number of firsts in this field,” said Paul Laikind, Ph.D., chief executive officer and president of ViaCyte. “Now with the first demonstration of insulin production in patients who have received PEC-Direct, we are confident we can be the first to deliver an effective stem cell-derived islet replacement therapy for type 1 diabetes.”

Breakthrough T1D has been a significant supporter of Viacyte. Early its in history (when ViaCyte was called CyThera), Breakthrough T1D underwrote development of the proprietary line of precursor stem cells used in their treatment. Breakthrough T1D also funded the preclinical and clinical studies of ViaCyte’s PEC-01™ therapies, which are designed to mature into islet tissue in humans, including glucose-responsive insulin-secreting beta cells. This includes the first ever clinical trial to test a stem cell-derived beta cell replacement therapy for T1D, in 2014.

“Today, ViaCyte presented preliminary data from its PEC-Direct therapy clinical trial, which showed that implanted cells, when effectively engrafted, are capable of producing insulin, in people with type 1 diabetes,” said Aaron J. Kowalski, Ph.D., president and CEO of Breakthrough T1D. “Advancing research in beta cell replacement is a core pillar of Breakthrough T1D’s research strategy and we have been a significant supporter of ViaCyte and other promising approaches. This progress is an encouraging milestone that shows the research being conducted today is bringing us one step closer to finding cures for type 1 diabetes.” 

This funding is one of several approaches to beta cell replacement Breakthrough T1D has advanced through research funding and regulatory affairs as part of the Breakthrough T1D Beta Cell Replacement Consortium. In addition, Breakthrough T1D had a major hand in the creation in 2004 of the California Institute of Regenerative Medicine, which is another major funder of the company. Beta cell therapies and immune therapies are the two pillars of Breakthrough T1D’s cures strategy.

Why it Matters:
Beta cell replacement therapies aim to provide insulin on demand from cells implanted in the body. These therapies have the potential to eliminate insulin therapy and liberate people from the burdens of managing T1D for months or even years at a time. The shortage of donor beta cells and the need for strong immunosuppressive drugs, however, make beta cell transplantation an impractical solution for most people. Breakthrough T1D is heeding the call, developing beta cell replacement technologies that are capable of restoring glucose control and delivering long-term independence without suppressing the body’s immune system and the ability to fight infections. ViaCyte is moving another step forward in the development of their system, and the technology will hopefully be approved in the coming years.

To learn more about Breakthrough T1D’s work to advance beta cell replacement, click here.