Issue link: https://viewer.e-digitaledition.com/i/409942
T HROUGH APPROXIMATELY EIGHT years of research that progressed from mouse models to human trials, UF scientists found that a pharmaceutical cocktail made from Thymoglobulin and Neulasta appears to restart the body's ability to produce insulin. "We began looking at the two agents together using slightly different doses than had been used previously," says Michael Haller, MD, Associate Professor of Pediatrics at the UF College of Medicine. "In animal models, we were able to demonstrate clearly that … when you give Thymoglobulin fi rst to basically immune-deplete the mice and then Neulasta to provide immu- nomodulation immediately thereafter, we could see durable reversible rates [of Connecting peptide (C-peptide) retention] in mice of about 85–90 percent." C-peptide levels reflect the body's ability to produce and use insulin. By cont ra st , when ad m in istered a s a monot herapy, Thymoglobulin, a medication used to prevent and treat organ rejection following kidney transplant, provided only as much as a 40 percent reversal. Neulasta — a drug typically used during chemotherapy that promotes the production of white blood cells — demonstrated no reversal rates. Further research into the combination therapy is under- way. However, the recently completed human trials showed that patients with Type 1 diabetes maintained C-peptide levels more than one year following therapy. Two-year results are slated to be released at the end of 2014, according to Dr. Haller. Pursuing the Mechanism Precisely how the combination therapy functions remains unclear. The team hypothesizes that each agent attacks different components of the immune defects linked to Type 1 diabetes. Low doses of Thymoglobulin destroy effector T-cells — which attack beta cells and their antigens — without decimating regulatory T-cells that may protect against effector T-cells. Neulasta catalyzes the production of a number of cells, including regulatory T-cells, dendritic cells and others that help the body bounce back after an immune attack. Approved and Potentially Fast-tracked Because Neulasta and Thymoglobulin are already FDA-approved, researchers aren't slowed by regulatory red tape associated with newly developed drugs. After the second, larger trial closes, it is possible that patients will have rapid access to the therapy. "It's a theoretical advantage, but it's a real one," Dr. Haller says. "You talk to people who have diabetes and want therapies for their kids or themselves now. They don't want to wait 10 years for a drug to be approved." The team hopes to develop an intravenous therapy that would be administered at specifi c intervals and that would signifi cantly reduce or eliminate the need for insulin shots for some patients. ■ TWO DRUGS APPROVED BY THE FDA FOR OTHER USES HAVE A SYNERGISTIC RELATIONSHIP THAT MAY REDUCE THE NEED FOR INSULIN SHOTS AMONG PATIENTS WITH TYPE 1 DIABETES, RESEARCHERS AT THE UNIVERSITY OF FLORIDA (UF) HAVE DISCOVERED. By Michael Ferguson Pharmaceutical Repurposing Yields Potential Diabetes Treatment 1 4 | Greater Boston MD NEWS ■ M D N E W S . CO M