Tracking drug's ability to prevent type 1 diabetes

T1D, formerly called juvenile diabetes, is an autoimmune disease that is predominant in the younger population and has a strong genetic component. The study is seeking participants between the ages of 6 and 45 whose antibody screening results indicate they have a strong likelihood of developing T1D.

"People often think that screening for type 1 diabetes risk factors is unimportant because there is currently nothing that can be done to mitigate their risk level for type 1 diabetes," said William Russell, M.D., Cornelius Vanderbilt Professor of Pediatrics, director of Pediatric Endocrinology and Diabetes at Monroe Carell Jr. Children's Hospital at Vanderbilt and protocol chair for the international trial.

"The ultimate goal of this trial is to find a method to prevent, or at least delay, the development of type 1 diabetes in persons most at risk," Russell said.

Abatacept, under the brand name Orencia, is FDA approved for the treatment of the autoimmune disease rheumatoid arthritis. Abatacept has shown success in interrupting autoimmune attacks on the body's insulin-producing cells in people newly-diagnosed with T1D.

"The autoimmune process that leads to type 1 diabetes can begin long before any symptoms appear," Russell said. "Autoantibodies that target the insulin-producing cells in the pancreas can be present months or even years before a person develops diabetes. Although the presence of these autoantibodies does not signify certainty of developing type 1 diabetes, it can be a very strong indicator and an opportunity to identify people at risk."

Having a blood relative with T1D is why the first participant, Ric Hudgins, joined the trial.

Hudgins says he made a concerted effort to learn all he could about the disease after his 10-year-old son was diagnosed, leading him to become involved with TrialNet and subsequently the abatacept trial.

"I wanted to learn all I could about the disease and I wanted to help in any way to progress potential treatments or preventions for the disease," Hudgins said.

"Although this study is targeted toward people who have a high risk of developing type 1 diabetes, and therefore cannot help my son, hopefully trials like these can help researchers find ways to prevent other children from receiving a type 1 diabetes diagnosis."

Hudgins said that his son, now almost 13, is managing his disease very well and is proud that his family is involved in research trials like this one.

The process of enrolling in T1D prevention trials begins with an antibody screening of a blood relative of someone with T1D, at no cost through the National Institutes of Health-funded TrialNet Pathway to Prevention Study (5U01DK085465-05).

Participants in the prevention trial will receive 14 intravenous infusions of abatacept over a one-year period, returning every six months for tests to determine the efficacy of the drug in limiting immune system damage to the healthy insulin-producing cells.

"This trial focuses on prevention and widening the spectrum of what we can do for people at risk of developing diabetes," Russell said.