Stem cell-based treatment controls blood sugar in people with type 1 diabetes

An innovative stem cell-based treatment for type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results from the University of British Columbia (UBC) and Vancouver Coastal Health (VCH) – a trial funded by Canada’s Stem Cell Network (SCN).

This article was initially published on the Stem Cell Network’s website; you can access it here.

Stem cell-based treatment controls blood sugar in people with type 1 diabetes, demonstrates SCN-funded clinical trial

“This is a significant step toward a functional cure for type 1 diabetes,” said Dr. David Thompson, principal investigator at the Vancouver trial site, clinical professor of endocrinology at UBC and director of the Vancouver General Hospital Diabetes Centre. “For the first time, a stem-cell based device can reduce the amount of insulin required for some trial participants with type 1 diabetes. With further refinement of this approach, it’s only a matter of time until we have a therapy that can eliminate the need for daily insulin injections entirely.”

The findings, published today in Nature Biotechnology, arise from a multicenter clinical trial for an experimental cell therapy that aims to replace the insulin-producing beta cells that people with type 1 diabetes lack. A small medical implant – approximately the size of a Band-Aid and no thicker than a credit card – which contains millions of lab-grown pancreatic islet cells, is implanted just beneath a patient’s skin where it is hoped that they will provide a steady, long-term regulated supply of self-sustaining insulin.

“Each device is like a miniature insulin-producing factory,” said co-author Dr. Timothy Kieffer, a professor within the departments of surgery and cellular and physiological sciences at UBC, and past chief scientific officer of ViaCyte. “The pancreatic islet cells, grown from stem cells, are packaged into the device to essentially recreate the blood sugar regulating functions of a healthy pancreas. This may have tremendous benefits over transplant of scarcely available donor-derived cells, given that we can create a virtually limitless supply.”

Ten participants, each of whom had no detectable insulin production at the start of the study, underwent surgery to receive up to ten device implants. Six months later, three participants showed significant markers of insulin production and maintained those levels throughout the remainder of the year-long study. These participants spent more time in an optimal blood glucose range and reduced their intake of externally administered insulin. One participant, in particular, showed remarkable improvement, with time spent in the target blood glucose range increasing from 55 to 85 per cent, and a 44 per cent reduction in their daily insulin administration.

“The Stem Cell Network is delighted to support this clinical trial and we’re pleased to see the promising results,” said Cate Murray, President and CEO of the SCN. “Moving toward a functional cure for diabetes will require a coordinated and collaborative effort. It takes excellent science by top researchers in world-leading institutions, funders, like SCN, that de-risk research, and innovative biotech companies that can manufacture and scale the technology. SCN is proud to play its part and we look forward to what’s next in game-changing diabetes research.”

The results are the latest in a series of clinical trials funded by SCN and conducted by the UBC-VCH team. Previously, in a 2021 study in Cell Stem Cell, the researchers were the first to show that the approach could produce insulin in the human body. In another ongoing trial, also funded by SCN, the UBC-VCH team is investigating whether a version of the device containing cells that have been genetically engineered to evade the immune system, using CRISPR gene-editing technology, could eliminate the need for participants to take immunosuppressant drugs alongside the treatment.

“We envision a future where people with type 1 diabetes are able to live their lives free from daily insulin injections and free from immune suppressing drugs,” said Dr. Thompson. “That future is now within reach, and Canada is leading the way in efforts to bring these novel treatments to patients.”

Key Facts:

• Approximately 300,000 Canadians are living with type 1 diabetes, which costs the Canadian health care system about $29 billion annually.
• Since 2016, the Stem Cell Network has invested $10.3M in diabetes research in Canada for 18 research projects, including four clinical trials.
• The clinical trial was conducted at Vancouver General Hospital in B.C., Canada, with additional sites in Belgium and the United States.
• The tested stem cell-based therapy is developed by U.S. biotechnology company ViaCyte (acquired by Vertex Pharmaceuticals) and is being clinically tested in Canada by leading researchers and clinicians.
• The trial sought to significantly increase the amount of insulin produced by leveraging two-to-three times more devices per participant, alongside an updated device design with small perforations to allow for blood vessel ingrowth — a feature aimed at improving survival of the lab-grown cells.

These trials are continuing to recruit participants. People who are interested in participating and want to learn more can contact study coordinator Barbara Allan (barbara.allan@vch.ca).

About the Stem Cell Network: The Stem Cell Network (SCN) is a Canadian not-for-profit that supports stem cell and regenerative medicine research; training the next generation of highly qualified personnel; and knowledge mobilization and transfer of stem cell and regenerative medicine research. From the lab to the clinic, SCN’s goal is to power life-saving therapies and technologies through regenerative medicine research for the benefit of all. Created in 2001, with support from the Government of Canada, the Network has grown from a few dozen labs to more than 270 world-class research groups, supporting over 250 research projects and 30 clinical trials. Since its inception, over 25 biotech companies have been catalyzed or enhanced and more than 6,400 highly qualified personnel have been trained. In 2021, the Government of Canada demonstrated its continuing trust and support in SCN with an investment of $45 million for the 2022–2025 period.