Mouse T1D Prevention Study Highlights Approach to Accelerate Translational Research

JDRF has a plan for creating a world without type 1 diabetes (T1D) that focuses on driving promising therapies through the R&D pipeline and into the hands of individuals with T1D. A critical step in progress along the pipeline is known as the translational research phase. The goal of translational research is to move research discoveries or concepts to the clinical stage of testing to determine if they have the desired therapeutic effects in people with T1D. In other words, translational research is a critical link between great ideas and real products in bringing future T1D treatments and cures to market that will benefit individuals with T1D.

An approach to accelerating translational research is to repurpose drugs or agents being developed for other diseases, especially drugs that have been approved to be used in the clinic for other indications. An example of such is a recent study, partially funded by JDRF that discovered a novel way to prevent T1D in mice using a drug already approved to treat a type of cancer in people. In the study, published in the Proceedings of the National Academy of Sciences, the researchers tested this drug (and another experimental drug in the same class) being used to treat lymphoma that inhibits an enzyme called lysine deacetylase. They found that very low doses of the drug (100 times lower than used in cancer treatment) blocked harmful inflammation that plays a role in destroying insulin-producing cells in the pancreas of mice with T1D. Ultimately, the drug prevented the mice from developing T1D. According to lead researcher Dan Ploug Christensen, “Our research shows that very low doses of anticancer drugs used to treat lymphoma can reset the immune response to not attack the insulin-producing cells.”

While these findings shed light on a possible new way of stopping T1D in its tracks, they also highlight the need to quickly evaluate this finding in people to see if these findings in mice can be translated to humans. The researchers in this study stress that the doses of the cancer drug they used have been proven safe in children and may prove useful in preventing T1D in people at risk for developing the disease. Only through translational research that lays the groundwork for broader clinical trials in people with T1D will we learn if this is the case.

JDRF will continue to explore efforts with the study’s investigators as they seek to move this research forward through the pipeline. Additionally, JDRF has recently announced a number of new initiatives to accelerate translational research, including the launch of T1D Innovations, a request for proposals specifically asking for plans to test drugs already approved for other  indications in T1D, and a new partnership with the Pfizer’s Centers for Therapeutic Innovation (CTI) to help move other promising therapies more quickly through the pipeline.