Protecting Pancreas From Type 1 Damage

Type 1 diabetes progression slowed by experimental medication

/ Author:  / Reviewed by: Robert Carlson, M.D Beth Bolt, RPh

(RxWiki News) With type 1 diabetes, the body does not produce enough insulin to help cells take in blood sugar for energy. An experimental medication may help change that.

Type 1 diabetes is usually diagnosed in children and young adults. It accounts for 5 percent of all diabetes cases. That’s a total of about 3 million Americans, and the number has been climbing, according to the Juvenile Diabetes Association. While there is no cure for the disease, it can be managed, and patients can live full productive lives.

Scientists have recently developed an experimental medication called teplizumab that is showing promise in blocking the advance of type 1 diabetes in its early stages by helping the body maintain insulin production.

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Kevan Herold, MD, a professor of immunobiology and deputy director for translational science at Yale University in New Haven, Connecticut, led this clinical trial of 52 subjects who had new-onset type 1 diabetes.

At diagnosis, Dr. Herold and his team treated patients — most of whom were under the age of 14 — with teplizumab for two weeks and again after one year. Their capacity to make their own insulin to control their blood sugar was compared with a non-treated group of 25.

Patients who benefited still had relatively good control of their blood sugar levels and only a moderate need for insulin injections when the trial began. In the subjects who were taking teplizumab, insulin production levels were maintained for two years in a portion of the responders. They all needed insulin, but at a lower dose than the non-responders.

The researchers noted that this two-year response was longer than most other medications tested against the disease, but teplizumab did not stop or reverse type 1 diabetes.

The authors commented that not all patients benefited from the treatment. Some lost half or more of their ability to produce insulin. This reaction was similar to many in the comparison group who were not receiving the medication.

While there were 18 responders in the group, there were 22 non-responders and 11 patients were taken off teplizumab because of side effects.

The researchers said that the reasons for the different responses are not clear but probably involve differences in metabolism (physical and chemical processes in the body that convert or use energy) and in the severity of their disease at the trial’s start.

“The benefits of treatment among the patients who still had moderately healthy insulin production suggests that the sooner we can detect the prediabetes condition and get this kind of drug onboard, the more people we can protect from the progressive damage caused by an autoimmune attack,” said Jeffrey Bluestone, PhD, a professor and immunologist at University of California-San Francisco, who collaborated in developing the medication.

Dr. Bluestone has concentrated his research efforts on understanding how and why the immune system attacks the body’s own tissues and organs. He has developed medication strategies to eliminate the autoimmune response without producing severe side effects.

Teplizumab is under active investigation for its ability to control autoimmune reactions. Teplizumab uses an antibody that helps prevent T-cells from attacking beta cells of the pancreas, which produce insulin.

Immunotherapies have been used to treat autoimmune diseases, including multiple sclerosis, Crohn’s disease, rheumatoid arthritis and asthma.

Dr. Herold told dailyRx News, “We are trying to launch a phase III trial of teplizumab for new onset type 1 diabetes. Our goal is to get the drug approved. The bottom line is that the drug continues to show efficacy, now in five clinical trials.”

The results of this trial were published online in Diabetes and will appear in the November print edition.

Dr. Bluestone developed teplizumab in collaboration with Ortho Pharmaceuticals in 1987. Dr. Herold has received grant support from MacroGenics, Inc.

Review Date: 
August 15, 2013
Last Updated:
August 30, 2013