Diabetes could be cured by an anticancer drug

In 2016, University of Pittsburgh researchers Farzad Esni and Jing Hu did an experiment on mice in which they deleted one of the two copies of the gene that codes for an enzyme called focal adhesion kinase (FAK). They were interested in the role of FAK in pancreatic cancer, but a surprising discovery took the research in a very different direction.

“The pancreas looked strange, almost as if it was trying to regenerate after an injury,” Esni said.

Even more strangely, a group of cells in the pancreas secreted both insulin and amylase. In normal mice and humans, insulin, the hormone that regulates blood sugar, is produced by beta cells, while amylase, a digestive enzyme, is produced by acinar cells. The functions of acinar cells and beta cells are very distinct, so it didn’t make sense for this group of cells to look like a combination of the two.

“There were three possible explanations for what we saw in the mice,” Esni said: “It could simply be an artifact of our experiment, the beta cells could start making amylase, or the acinar cells could start making insulin, which would be the Holy Grail.”

In a new study, researchers showed that a FAK-inhibiting drug studied in cancer treatment transformed acinar-derived insulin-producing (ADIP) cells and helped regulate blood sugar in diabetic mice and a primate.

To study the effects of ADIP cells, the researchers partially or completely eliminated the animals’ beta cells with a low or high dose of a compound called streptozotocin, which mimics diabetes. They then treated the mice for three weeks with an oral drug that inhibits FAK or a placebo.

Mice treated with FAK inhibitors regained approximately 30% of their initial beta cell mass, and the treatment partially ameliorated hyperglycemia. These results persisted until the end of the experiment several weeks later, suggesting that a single treatment may have long-term benefits for diabetes control.