Could a pill cure diabetes? A U of A researcher is working on it

A simple pill to treat or even cure diabetes could be possible, thanks to University of Alberta research — and funding that is laying vital groundwork.

A team of scientists has identified a certain gene in crucial insulin-producing cells, and the discovery is a step toward creating a set of drugs that could effectively cure diabetes.

The research, if eventually proven effective in clinical trials in humans, could be “one of the most significant discoveries in the field of diabetes,” says lead researcher Jean Buteau, a professor of human nutrition in the Faculty of Agricultural, Life & Environmental Sciences and member of the Alberta Diabetes Institute at the U of A.

“It would pave the way for an entirely new class of medications for diabetes treatment, especially in Type 1 diabetes, where options are extremely limited. Our hope is that it could lead to better glucose control, less insulin use, and ideally, diabetes remission.”

Over the past few years, the team’s work involved characterizing what is known as the Lyn kinase gene and the molecule that targets it. The gene regulates beta cells, which are insulin-producing cells located in the pancreas that are responsible for the cells’ life or death. If beta cells fail, blood glucose levels rise, causing diabetes.

Triggering the gene through a pill containing the molecule could protect surviving beta cells and even regenerate those destroyed by the immune system, Buteau explains.

“The goal is to have more beta cells to increase the body’s capacity to secrete insulin and better regulate glucose.”

The therapy has already proven effective against Type 1 and Type 2 diabetes in mice model experiments conducted by Buteau and his team.

“With Type 2 diabetes, we discovered that the molecule we want to use in a pill was doubling the number of beta cells after just seven days of treatment.”

The results were similar against Type 1 diabetes, with “an expansion of beta cell mass and regeneration, and also a protection of beta cells,” he adds.

“These findings were extremely exciting,” he adds. “We now know that we can cure or prevent Type 1 and Type 2 diabetes in mice with this molecule, and we are cautiously optimistic for what it could mean for people with diabetes.”

The promising findings have paved the way to now conduct the first-ever clinical testing of the therapy in humans with Type 1 diabetes. Working with a partner in the pharmaceutical industry, the U of A team is preparing to test an oral drug with 12 patients.

The pilot study will help provide key baseline data to develop and refine the protocol for a larger clinical trial.

Buteau’s work is among the cutting-edge research being supported by the Defeating Diabetes campaign, publicly launched today on World Diabetes Day. As a partnership between the Alberta Diabetes Foundation, the Diabetes Research Institute Foundation of Canada and the U of A’s Faculty of Medicine & Dentistry, the campaign funds a trio of key research areas: finding a cure, eliminating the impact of diabetes in people’s lives, and increasing early diagnosis to mitigate the harmful conditions associated with the disease.

The fund, which has reached $8 million of its $10-million goal to date thanks to support from community leaders and donors, was started in 2020 to support vital research at the Alberta Diabetes Institute.

Buteau’s progress from basic research to the clinical trial stage is a significant achievement, notes Peter Senior, director of the Alberta Diabetes Institute and Charles A. Allard Chair in Diabetes Research at the U of A.

“Typically, our clinical research unit within the Alberta Diabetes Institute conducts trials of drugs developed by other researchers or industry partners. The opportunity for vital basic research conducted in-house by Buteau and his team, to take the next step to the clinical testing stage — all within the institute — is a huge achievement,” notes Senior.

“It’s a project that happened right here in Alberta, translating all the way from the discovery stage to the cusp of clinical research. The project is not only an example of groundbreaking work that signals hope for people with diabetes, but is also a homegrown success story that continues the U of A’s long history of major diabetes research discoveries.”

The support provided through the Defeating Diabetes campaign is crucial to advancing his team’s work, Buteau says.

“Without it, I’m not sure we would have been able to bring this research to fruition.”

The contribution of the campaign “goes far beyond its monetary value,” he adds. “It reminds us as researchers why we have chosen to study diabetes and keeps us motivated.”

The Defeating Diabetes campaign supports innovative research throughout the Alberta Diabetes Institute, including the work of four more U of A researchers all based in the Faculty of Medicine & Dentistry. James Shapiro is working on using engineered stem cells that can produce insulin in the body and eliminate the need for immunosuppressants.

Gregory Korbutt and Andrew Pepper are tackling the supply issue of islets used for transplant in diabetes patients, where an islet transplant is their only hope in controlling the disease. Due to limited supply and risk of disease and complications from lifetime immunosuppressants, they are working on a reliable source of islets and a safer way to transplant them.

Andrea Haqq, who is also cross-appointed to the Faculty of Agricultural, Life & Environmental Sciences, is looking at using a unique combination of therapies to prevent Type 2 diabetes in children.

In addition to the Defeating Diabetes campaign, Buteau’s work with the Lyn kinase gene was supported by a Canadian Institutes of Health Research grant.