Results from UF Health pilot study lead to national trial of combination diabetes treatment

Inside the pancreas, beta cells produce insulin, the hormone that helps the body properly metabolize food. However, in patients with Type 1 diabetes, these beta cells are under attack and unable to produce the insulin the body needs, which is why patients with this condition typically must take insulin for the rest of their lives.

However, the results of a new University of Florida pilot study published in the Journal of Clinical Investigation Dec. 15 show that combining two medical treatments preserved beta cell function in patients, allowing them to produce at least some of their own insulin after one year. Patients who produce some of their own insulin often fare better, with fewer complications and better control of blood glucose levels.

Now the international diabetes research network TrialNet, in conjunction with UF researchers, is poised to take the next step in this research by launching the study in a larger, national population of participants newly diagnosed with Type 1 diabetes. The pilot study included participants who had been diagnosed with the disease for four months to two years.

“If the larger trial produces similar results, this type of combination therapy will be a major advance in the treatment of Type 1 diabetes,” said Michael Haller, M.D., an associate professor of pediatrics in the UF College of Medicine and principal investigator of the pilot study and the TrialNet study.

In the pilot study that was completed, Haller treated 17 patients with a cocktail of two drugs. Thymoglobulin, also known as antithymocyte globulin (ATG), was administered to help rid the body of immune system cells that were attacking good beta cells. The other drug, Neulasta, or granulocyte-colony stimulating factor (GCSF), was given to help produce healthy, new immune cells. Eight additional patients received a placebo.

After one year, the participants who received the combination therapy were still producing insulin at baseline levels. Among participants who received a placebo, insulin production was down 40 percent after one year.

“Since the combination therapy used in the pilot study had such positive results in people who had diabetes for four months to two years, we hope the same therapy will show even greater benefit in people with newly diagnosed diabetes,” Haller said.

Up to 3 million Americans have Type 1 diabetes, according to JDRF, formerly known as the Juvenile Diabetes Research Foundation. Although patients can be treated with insulin, complications from the disease and low and high blood glucose levels can still occur, which is one of the reasons why finding new and better treatments is so important.

“What makes these studies so exciting is that they are based on a whole new scientific model that uses a combination of two drugs to reduce the immune response that triggers destruction of insulin-making cells,” said Jay Skyler, M.D., chairman of TrialNet. “Because neither of these drugs was able to accomplish this alone, it challenges the notion that drugs used in combination should first show individual benefit.”

The new ATG-GCSF study in new-onset Type 1 diabetes is open to patients between the ages of 12 and 45 who have been diagnosed with Type 1 diabetes within the past three months. For more information, email study coordinator Miriam Cintron at cintrm@peds.ufl.edu or call 352-273-9264.

The pilot study was funded by The Leona M. and Harry B. Helmsley Charitable Trust and Sanofi.