In a healthy pancreas, beta cells play a crucial role in regulating blood sugar levels by producing insulin. However, in individuals with diabetes, these cells are either damaged or dysfunctional, leading to insufficient insulin production.

As a result, people with diabetes require regular insulin injections to manage their blood sugar levels and maintain control.

Research has been increasingly focused on restoring the function of beta cells. Some methods involve using stem cells to generate new beta cells, which are then transplanted into patients, a strategy described as a potential “functional diabetes cure.”

Scientists at Mount Sinai and City of Hope have now made a significant advancement. Unlike previous studies that required growing new beta cells in a lab before transplanting them, this new study has achieved the growth of insulin-producing cells directly within the body within a few months.

The therapy combines two drugs: harmine, a natural compound from certain plants that inhibits the DYRK1A enzyme in beta cells, and a GLP1 receptor agonist, a class of diabetes drugs that includes Ozempic, known for its weight loss effects.

This therapy was tested on mouse models of both type 1 and type 2 diabetes. Researchers implanted a small number of human beta cells into the mice and treated them with harmine and GLP1 receptor agonists.

The result was a 700% increase in beta cells within three months, with the signs of diabetes reversing and remaining improved even a month after stopping the treatment.

Dr. Adolfo Garcia-Ocaña, the corresponding author of the study, highlighted that this is the first time a drug treatment has been shown to increase adult human beta cell numbers in vivo.

He stressed that this research brings hope for future regenerative therapies that could potentially treat millions of diabetes patients.

“We’ve made a groundbreaking discovery, successfully developing a drug treatment that boosts adult human beta cell numbers in the body. This breakthrough offers new hope for regenerative therapies that could potentially treat the millions of people worldwide living with diabetes,” he said.

Although the results are promising, the animal-based nature of the study means more research is needed before this can be applied clinically in humans.

Harmine alone has recently completed a phase 1 clinical trial to test its safety and tolerability in humans, and other DYRK1A inhibitors are planned for human trials next year.

Importantly, the researchers aim to combine beta-cell-regenerating drugs with immune system modulators. This could help overcome the major challenge of the immune system attacking newly produced beta cells.