Teplizumab may prevent, reverse Type 1 diabetes


Doctors are studying ways to cure the disease before it happens and even get rid of the disease in people who already have it. A clinical trial is being held at Vanderbilt University testing the drug Teplizumab.

"It's been well studied in individuals who have been newly diagnosed with diabetes. What if we take someone who is at high risk to develop diabetes, can we actually prevent it?" said Dr. William Russell, director of pediatric endocrinology and diabetes at Vanderbilt.

The drug battles an immune system protein called CD3. The goal is to find out if it can also stop the destruction of the insulin-producing cells in the pancreas before diabetes occurs.

Meanwhile, researchers at the University of Colorado are curing it in animals. By isolating specific T cells that attack the pancreas, they developed a drug that can stop diabetes from developing, and even reverse it in mice that already have it.

Vanderbilt researchers plan to follow patients for up to four years. Doctors say their ultimate goal for the trial is to enroll 150 people in the U.S.

For more information on the trial, visit www.diabetestrialnet.org.

More information:

Background: Diabetes is a group of diseases marked by high levels of blood glucose resulting from flaws in insulin production, insulin action or both. It can lead to serious complications and premature death, but steps can be taken to control the disease and lower the risk of complications. (Source: Center for Disease Control and Prevention)

Type 1: Type 1 diabetes develops when the body's immune system destroys beta cells in the pancreas, the only cells in the body that make the hormone insulin that regulates blood glucose. It most often strikes children and young adults, though disease onset can occur at any age. In adults, Type 1 diabetes accounts for approximately 5 percent of all diagnosed cases of diabetes. Risk factors may be autoimmune, genetic, or environmental. There is no known way to prevent Type 1 diabetes. Several clinical trials for preventing Type 1 diabetes are currently in progress or are being planned.

Genes vs. environment: With the growing worldwide epidemic of diabetes, a key question is to what extent is diabetes in our genes and to what extent is it due to changes in our environment? About 40 percent of people in the U.S. carry one or more of risky genes, but only about one out of 100 in this category will actually develop the disease. This reflects the fact that there are likely one or more environmental factors that trigger diabetes in these genetically susceptible individuals. What exactly these environmental factors are remains unknown.

"It's a little hard to say that sixty five percent is genes and thirty five percent is environment. If people live to be 150, that sixty five percent number might turn into eighty five percent or ninety percent," said Dr. William Russell, from Vanderbilt University. "The genes set the stage for that but there are probably environmental triggers that actually initiate the process."

Stopping it: Teplizumab has been created to change the function of the T lymphocytes that mediate the destruction of the insulin-producing beta cells of the islets of the pancreas. Teplizumab binds to an epitope of the CD3-epsilon chain expressed on mature T cells and by doing so, may modulate the immunologic response that is a key component of the disease. If Teplizumab is effective and has the ability to preserve or protect beta cells of the pancreas, patients may require less injected insulin and their blood glucose levels may be easier to control. Teplizumab represents a key shift in the management of autoimmune disease that focuses on the introduction of tolerance rather than broad spectrum immunosuppression.

Type 1 Diabetes TrialNet is an international network of researchers who are exploring ways to prevent, delay and reverse the progression of type 1 diabetes. TrialNet is conducting clinical trials with researchers from 18 Clinical Centers in the United States, Canada, Finland, United Kingdom, Italy, Germany, Australia and New Zealand. Studies are available for people newly diagnosed with type 1 diabetes, as well as for relatives of people with type 1 diabetes who are at greater risk of developing the disease.

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