Now, cancer drug holds out hope for diabetes treatment

An Australia-based study from Monash University in Melbourne this month has claimed a “major breakthrough” in Type 1 diabetes treatment protocol as researchers restored insulin expression in the damaged pancreas cells of a deceased 13-year old child using a cancer drug.
Published in The Nature’s “Signal Transduction and Targeted Therapy” journal, the researchers took pancreatic cells with classic signs of silencing of the insulin-producing cell progenitor genes with barely detectable insulin. The drug, GSK126, which is not authorised for Type 1 diabetes and is a cancer remedy otherwise, was used in the cells, resulting in the expression of core insulin-producing cell markers. It was also found to reinstate insulin gene expression despite absolute destruction of the insulin-producing cells.
The study has essentially discovered a novel pathway to the regeneration of insulin in pancreatic stem cells. Using the pancreas stem cells of the Type 1 diabetic donor, researchers were able to effectively reactivate them to become insulin-expressing and functionally resemble beta-like cells through the use of a drug. In principle, this means that the said drug will allow insulin-producing cells (beta-cells) that are destroyed in Type 1 diabetics to be replaced with newborn insulin-generating cells.

The authors claim that this discovery can translate to a major breakthrough in new therapies to treat Type 1 and Type 2 diabetes, especially leading to a potential treatment option for insulin-dependent diabetes.
Significance of this proof-of-concept study
Type 1 diabetes is a chronic condition in which the pancreas produces little or no insulin, leading to glucose build-up in the bloodstream instead of going into the cells and in turn causing hypoglycemia. Symptoms are usually not apparent until around 80 per cent of the insulin-producing cells, that is the beta-cell mass, are destroyed. Ultimately, this leads to patients relying on external insulin adminration for survival.
Two strategies currently ex that focus on replacing the damaged beta cell mass in diabetic patients, which involve either whole pancreas or islet transplantation. However, both these methods become a challenge given the acute shortage of organ donors in most countries, combined with the associated side-effects of immunosuppressive drugs. The current research has thus focussed on the replacement of the lost beta cell through infusing descendants of stem cells which can further differentiate into specialised cells, effectively making new functional beta cells, which generate insulin.

Limitations
Only an isolated case of a 13-year old Type 1 diabetes child with hallmark islet damage and significant destruction of ß-cells was taken with insulin expressing cells taken from two adult non-diabetic brain dead donors, which, the authors acknowledge, makes it “unknown if the results will generalise.”
The study also notes that it is unclear whether silencing of progenitor genes can be restored in long-standing diabetes, given that the child had diabetes for the past 4.5 years.
The authors also acknowledge that further studies are required for “due consideration to the potential pharmacological interactions and unforeseen synergic benefits,” and it is also unclear what other effects of the GSK126 (a cancer treatment drug) can have.
Moreover, the study was conducted ex-vivo, that is not in live humans. making the genetic modification of cells outside the body to produce therapeutic factors, the authors acknowledge that the results from this study need to be confirmed with larger studies.