The researchers at the Harvard Stem Cell Institute (HSCI) believe that the hormone might also have a role in treating type 1, or juvenile, diabetes.
The hormone, called betatrophin, causes mice to produce insulin-secreting pancreatic beta cells at up to 30 times the normal rate. The new beta cells only produce insulin when called for by the body, offering the potential for the natural regulation of insulin and a great reduction in the complications associated with diabetes, the leading medical cause of amputations and non-genetic loss of vision.
The researchers who discovered betatrophin, HSCI co-director Doug Melton and postdoctoral fellow Peng Yi, caution that much work remains to be done before it could be used as a treatment in humans. But the results of their work, which was supported in large part by a federal research grant, already have attracted the attention of drug manufacturers.
"If this could be used in people," said Melton, Harvard's Xander University Professor and co-chair of the University's Department of Stem Cell and Regenerative Biology, "it could eventually mean that instead of taking insulin injections three times a day, you might take an injection of this hormone once a week or once a month, or in the best case maybe even once a year."
"Our idea here is relatively simple," Melton said. "We would provide this hormone, the type 2 diabetic will make more of their own insulin-producing cells, and this will slow down, if not stop, the progression of their diabetes. I've never seen any treatment that causes such an enormous leap in beta cell replication."
Though Melton sees betatrophin primarily as a treatment for type 2 diabetes, he believes it might play a role in the treatment of type 1 diabetes as well, perhaps boosting the number of beta cells and slowing the progression of that autoimmune disease when it's first diagnosed.
While Melton was clear about the need for more research before the hormone could be available as a drug, he also said that betatrophin could be in human clinical trials within three to five years, an extremely short time in the normal course of drug discovery and development.
The work was published by the journal Cell as an early on-line release.