Discovery of New Hormone May Impact T1D

The media has come alive, exciting the type 1 diabetes (T1D) community, following the publication of findings of a new hormone in Cell on April 25. The previously unknown hormone, called betatrophin, was found in a mouse study to have the unique ability to promote the natural replication of insulin-producing beta cells. While media reports have been quick to label this a breakthrough in the study of treatments for type 2 diabetes, its value to T1D research has yet to be determined.

JDRF is keenly interested in the potential for this work as a leader in the area of beta cell regeneration pathways. Another cause for interest is JDRF’s long relationship with the lead researcher for the study, Doug Melton, Ph.D. Dr. Melton, himself a parent of two children with T1D, has worked for many years studying embryonic stem cells for a possible cure to the disease. Several of his past studies on beta cell regeneration and beta cell generation from stem cells were performed with significant funding from JDRF.

Betatrophin is the latest in a series of discoveries of natural compounds that seem to trigger the regeneration of beta cells, including transmembrane protein 27, and cdk6.  These proteins, discovered with funding and support from JDRF, as well as betatrophin require additional testing to determine whether they can safely provide benefit to people with T1D. If proven effective, these therapies are only one piece of the puzzle, as the autoimmune process, which is not an issue in type 2 diabetes, will likely need to be addressed simultaneously for any therapy to be effective in T1D.

Moving forward, JDRF will continue to watch with interest as betatrophin is studied to learn more about it. The discovery of the hormone has led to a number of questions, including betatrophin’s method of action and its effect in people with T1D.

Some of this work has already been started by pharmaceutical companies Evotec and Janssen, who have acquired the rights to develop and market betatrophin. Evotec has stated that it believes it could have a therapy ready for human testing in three to four years.

JDRF’s vice president, cure therapies, Julia Greenstein, Ph.D. provides her own take: “It’s still too early to tell if there would even be any implications for people with type 1 diabetes. However, the possibility of having a natural hormone that can stimulate beta cell production is certainly interesting.”