Researchers at the University of Maryland Joslin Diabetes Center are testing whether a novel immunotherapy drug called otelixizumab will help prevent the destruction of insulin-producing cells in people who are newly diagnosed with type 1 diabetes. The center is one of 100 sites in North America and Europe – and the only site in Maryland – to offer the therapy as part of a Phase III clinical trial.
In type 1 diabetes, the body’s immune system attacks the pancreas’ beta cells, and people with the disease need insulin injections to help them process sugar. Patients have about 20 percent of their functioning beta cells left when they are first diagnosed with type 1 diabetes, according to Thomas W. Donner, M.D., lead investigator of the University of Maryland study.
“Preserving these remaining beta cells would be very beneficial to patients. Studies have shown that when type 1 diabetes patients are still making some of their own insulin, their blood sugar levels are much easier to control and they require less insulin,” says Dr. Donner, medical director of the Joslin Diabetes Center at the University of Maryland Medical Center and associate professor of medicine at the University of Maryland School of Medicine. “If this therapy proves to be effective, it could potentially lead to fewer low blood sugar reactions and complications from diabetes in the future.”
The clinical trial, the Durable-Response Therapy Evaluation for Early or New-Onset Type 1 Diabetes, is called DEFEND-1. The study is sponsored by Tolerx, Inc., a Cambridge, Mass., company that is producing the drug in conjunction with GlaxoSmithKline. The study is also being funded by the Juvenile Diabetes Research Foundation.
“The DEFEND-1 study is among the first clinical trials to try to prevent insulin-producing beta cells in the pancreas from being destroyed by the immune system,” Dr. Donner says.
Investigators hope to enroll a total of 240 young adults, age 18 to 35, who have been newly diagnosed with type 1 diabetes. University of Maryland researchers aim to recruit 10 patients.
The participants will be selected at random to receive eight days of otelixizumab infusions within 90 days of being diagnosed with type 1 diabetes. Two out of three people will receive the investigational drug, and the third person will receive a placebo. Neither the physicians nor the patients will know who is getting the drug. All of the participants will receive insulin injections and the usual standard of care for patients with type 1 diabetes. To evaluate the effectiveness of the treatment, researchers will measure C-peptide, a byproduct of the production of insulin in the blood which is a surrogate measure of beta cell function.
Dr. Donner adds, “All patients in the study will receive intensive diabetes management and free blood sugar testing supplies.”
E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs at the University of Maryland and dean of the University of Maryland School of Medicine, says, “Diabetes is a major public health problem affecting more than 170 million people around the world, and researchers at the University of Maryland School of Medicine are committed to finding better treatment options for patients. This study is an example of the innovative clinical research being conducted by our faculty members.”
Otelixizumab is a monoclonal antibody, which is being developed for the treatment of type 1 diabetes and other autoimmune diseases. It targets CD3, a T-lymphocyte receptor involved in normal cell signaling. Data suggest that the antibody works by blocking the function of effector T-cells, which mistakenly attack and destroy insulin-producing beta cells, while stimulating regulatory T-cells, which are believed to protect against effector T-cell damage.
Type 1 diabetes is one of the two major forms of diabetes. Previously known as juvenile diabetes or insulin-dependent diabetes, it accounts for 5 percent to 10 percent of the nearly 24 million people in the United States who have diabetes. Type 2 diabetes is by far the most common form. In type 2 diabetes, the body either doesn’t produce enough insulin or fails to use the insulin that it makes properly.
The University of Maryland Joslin Diabetes Center helps patients with both types of diabetes take charge of their own health and well-being, offering multidisciplinary care and individualized treatment programs. To get more information, people can visit the center’s Web site at http://www.umm.edu/joslindiabetes.
People who are interested in participating in the study should call (410) 328-6470. Additional information about the DEFEND-1 clinical trial is available at http://www.DefendAgainstDiabetes.com.
For patient inquiries, call 1-800-492-5538 or click here to make an appointment.