In what could be a breakthrough in diabetes research, investigators at the Harvard Stem Cell Institute have discovered a hormone with the ability to produce beta cells--the insulin-producing cells found in the pancreas that are lost in diabetes--that could treat Type 2 diabetes more effectively. The hormone also has the potential to treat Type 1 diabetes, or juvenile diabetes.
In mice, researchers found that the hormone betatrophin increases production of insulin-secreting pancreatic beta cells at up to 30 times the normal rate. Not only that, but the new beta cells generated from the betatrophin only produce insulin when the body needs them--a property that could help regulate insulin in the body and thereby offset many of the complications associated with diabetes. The research was published online April 25 in the journal Cell.
Betatrophin has caught the eye of the biopharma industry, and the compound has already been licensed to Johnson & Johnson's ($JNJ) Janssen Pharmaceuticals, which is working to move betatrophin toward the clinic. The Harvard team has also drawn up a collaborative agreement with Evotec, a German biotech firm that now has 15 scientists working on betatrophin.
"If this could be used in people," said Doug Melton, Harvard's Xander University Professor and co-chair of the University's Department of Stem Cell and Regenerative Biology, in a Harvard Medical School article, "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." Melton and colleague Peng Yi, a postdoctoral fellow, discovered betatrophin.
Beta cells play a role in both Type 1 and Type 2 diabetes, suggesting that betatrophin could be used as a therapeutic to treat both variations of the disease. In Type 1 diabetes, beta cells--which synthesize insulin--are destroyed by autoimmune processes. In Type 2 diabetes, people's bodies become so insensitive to insulin's actions that their beta cells eventually become unable to produce enough insulin.
Melton believes that the hormone could stop the progression of Type 2 diabetes by spurring the creation of insulin-producing cells in diabetics. In patients with Type 1 diabetes, betatrophin might work by boosting the number of beta cells in the body and slowing the progression of autoimmune disease when it's first diagnosed.
While more research needs to be done before the hormone could be available as a drug, Melton says he's optimistic that betatrophin could be tested in human clinical trials within three to 5 years, a very short turnaround on the timeline of drug discovery to development.
- get FierceBiotech's take
- here's the abstract in Cell
- read the Harvard Medical School article