11/11/2010 - Tim Waters has trekked from Atlanta to Nashville every month for two years in hopes of discovering a treatment that would help other children diagnosed with type 1 diabetes.
The lanky, freckled 11-year-old, accompanied by his mother, has missed school, sporting events and friends' parties to come to the Monroe Carell Jr. Children's Hospital at Vanderbilt. It's a sacrifice.
Waters is one of four participants in a first-of-its-kind drug trial for type 1 diabetes, an autoimmune disorder in which the body's immune system destroys insulin-producing cells (beta cells). The study at the Vanderbilt Eskind Children's Diabetes Clinic is part of the national Type 1 Diabetes TrialNet Program.
Diabetes Awareness Month during November serves as a reminder of the work still to be done and the progress already made. Insulin pumps, such as the one Tim wears, help patients manage blood sugar levels. Someone had to test it first.
“The only way to improve current treatments and to find cures for people is to participate in trials,” said Tim's mom, Sheila McFadden Waters, an oncology nurse practitioner in Georgia. “He's getting a standard of care better than any diabetic could ask for. We're hopeful this will have an effect on his pancreas.”
The trial, at the end of phase one, studies the effect of an anti-arthritic medication, Abatacept, on diabetes patients' rogue immune systems.
The next phase in the double-blind study tests participants' insulin production every six months over about two years to see if the drug worked, said Anne Brown, M.S.N., coordinator of the New Onset Type 1 Trials.
“This trial highlights how Vanderbilt draws study participants from some distances, and how dedicated they and their families are to take a day off from work or school once a month for two years to attend their visits,” said Brown. “There's a lot of sacrifice for the whole family. “
Abatacept already has approval to suppress rheumatoid arthritis in children, and researchers hope regular infusions of it can also help type 1 diabetes. The medicine, they hope, will shut down triggers that cause the immune system's attack on insulin-producing cells.
“While the purpose of this study is to determine whether Abatacept can delay or prevent further loss of the remaining beta cells in someone newly diagnosed with diabetes,” said William Russell, M.D., Professor of Pediatrics and the TrialNet Principal Investigator at Vanderbilt, “the ultimate goal is to extend such studies to prevent diabetes from developing in individuals who show early signs of immune dysregulation, but who haven't yet developed diabetes.”
Waters was diagnosed in September 2008, after a 10-day gastrointestinal infection. He lost 18 pounds and was lethargic. A month later, he was enrolled in the Abatacept trial. Since it is a double-blind study, the family does not know if he received a placebo or the drug.
On the family's 29th trip to Children's Hospital last week, Waters fasted for more than 12 hours and had his blood drawn 11 times over a four-hour period. He was allowed one can of a nutritional supplement. His twin sister, Deirdre, was along for support.
Asked if he liked being in the trial, the quiet boy uttered, “It's good,” as he kept his attention on a cartoon.
Deirdre had hopes that the trial would help her brother, the only one of the family’s four children to show any signs or markers of the disease. The Waters family participated in the TrialNet Natural History Study, where family members are screened for diabetes-related antibodies to determine their risk of developing type 1 diabetes.
“I just want him to get help and for it to make him better,” said Deirdre.
Now, Tim and his family wait to see if he had the drug and if it worked.
To learn more about type 1 diabetes research, contact the TrialNet Research Team at (888) 884-8638 or email@example.com or visit www.vanderbiltdiabetesresearch.com.©2014 Vanderbilt University Medical Center