New Type 1 Clinical Research Studies Launched

A number of new clinical studies using investigational drugs for type 1 diabetes have been launched by the UCSF Pediatric Diabetes Clinical Research Program under the leadership of Steve Gitelman, MD. Investigational drugs are those that are not approved for use in diabetes by the U.S. Food and Drug Administration (FDA). One novel study involves the immune system’s T cells.

There are different types of T cells, including T cells that unnaturally attack and destroy the insulin-producing beta cells in type 1 diabetes, and T cells called regulatory T cells that help call off the attack. Dr. Gitelman’s goal is to bolster the performance of the regulatory T cells, or “Tregs,” and therefore end the misdirected immune system attack on insulin-producing beta cells. This Treg study is open to adults ages 18 to 35 who are within two years of diagnosis.

A second study involving T cells aims to eliminate the T cells that attack insulin-producing beta cells, while increasing the number of Tregs, thereby resetting the balance of T cells. Dr. Gitelman and his colleagues are combining an antibody treatment anti-thymoctye globulin (brand name Thymoglobulin), which is FDA approved to prevent organ rejection in kidney transplant patients, with granulocyte colony-stimulating (brand name Neulasta), which is FDA approved to boost white blood cell counts in patients receiving chemotherapy. In mouse models of diabetes, this combination appears to be even more effective than Thymoglobulin alone. They hope the same will be true in people. This Thymoglobulin/Neulasta study is open to individuals ages 16 to 45 who are within two years of diagnosis.

For relatives of people with type 1 diabetes who are not yet diagnosed, but who are at risk for type 1 diabetes, there are now promising clinical trials available. Family members should take a simple blood test which screens for the presence of diabetes-related autoantibodies that may appear years before type 1 diabetes develops. This antibody screening is part of a national research program called Type 1 Diabetes TrialNet and is being conducted to help understand more about the development of this disease and to look at ways to delay or prevent it. For those found to be at intermediate risk of developing diabetes (25 percent chance of getting diabetes in the next five years), there is a trial introducing oral insulin . For those found to be at high risk (greater than 50 percent chance of diabetes in the next five years), there is a trial utilizing the anti-CD3 monoclonal antibody.