Australian scientists have demonstrated a new way to restore insulin production in pancreatic cells, using a drug that’s already approved for use in humans. The study could mark a major breakthrough towards new treatments for diabetes.
Blood glucose levels are managed by the hormone insulin, which is produced in beta cells in the pancreas. However, these cells begin to die off in patients with type 1 diabetes, resulting in little or no insulin production and a lifelong requirement for supplementary insulin shots to manage the disease.
In a new study, researchers from Monash University have identified a new way to restore insulin production in the pancreas. In lab experiments on pancreatic stem cells from donors with type 1 diabetes, the team was able to activate them to begin expressing insulin by exposing them to a drug compound known as GSK126.
Intriguingly, these progenitor cells don’t normally produce insulin, but the drug let them functionally step into the shoes of the beta cells that had stopped working. In principle, a single course of this kind of drug over a few days could replace the need for regular insulin shots in diabetics.
The team says that the new potential treatment has a few advantages over other techniques currently in use or under development. Pancreas transplants are effective, but subject to organ donor shortages and other complications like rejection. Other teams have converted skin cells into stem cells and used those to produce new beta cells, and although results have been promising in mice, immune-suppressing drugs need to be given to prevent rejection.
The new treatment would work much faster, within a matter of days, and without the need for surgery. But perhaps the biggest advantage is that GSK126 is already approved by the US FDA and elsewhere in the world as a treatment for cancer. Its safety profile is already being assessed in clinical trials, which could reduce hurdles down the road for its use against diabetes.
That said, the scientists caution that it is still very early days. These experiments were conducted on cells in culture – not even in animals yet – so there’s still plenty of work to do. Nevertheless, it remains an intriguing new possible tool.
"Before you get to patients, there are many issues to be resolved," said Dr Keith Al-Hasani, co-lead author of the study. "More work is required to define the properties of these cells and establish protocols to isolate and expand them. I would think therapy is pretty far away. However, this represents an important step along the way to devising a lasting treatment that might be applicable for all types of diabetes.”
The research was published in the journal Signal Transduction and Targeted Therapy. The team describes the work in the video below.
Source: Monash University