After it saved the lives of diabetic mice, a drug used to treat tuberculosis and cancer is now being tested in humans at Massachusetts General Hospital as a possible cure for type 1 diabetes.

Denise Faustman, MD, PhD, director of the MGH Immunobiology Laboratory and associate professor of Medicine at Harvard Medical School, proved that the drug, Bacillus Calmette-Guérin (BCG), cured mice with end-stage diabetes in 2001. She is now leading a Phase I clinical trial in patients that began in February and is expected to take 18 months to complete. BCG is a generic drug with an excellent safety profile in humans. It causes the body to make a natural substance called TNF, which helps regulate the immune system by killing the rogue T-cells that cause diabetes.

BCG has been used safely for nearly 80 years as a tuberculosis vaccine. It is being used in the human trial because it causes a low-grade inflammatory reaction, which in the mouse model of autoimmune diabetes led to the destruction of the abnormal autoimmune cells.

David M. Nathan, MD, director of the MGH Diabetes Center, commented on the study, "This is the very first step in what is likely to be a long process in achieving a cure. We first need to determine whether the abnormal autoimmune cells that underlie type 1 diabetes can be knocked out with BCG vaccination, as occurred in the mouse studies." Trial information is available to the public at www.faustmanlab.org.

Dr. Faustman’s research is unique because most diabetes research focuses on new treatments involving blood glucose monitoring devices. There is almost no emphasis on disease reversal or cure. “Our goal is to reverse established type 1 diabetes, not simply temporarily halt it or treat its symptoms,” says Dr. Faustman. “If we can introduce this inexpensive drug to the market, it will be a tremendous achievement.”

The clinical trial is being supported largely through direct and fundraising support from the Iacocca Foundation and through support from other donors and the Massachusetts General Hospital. The Iacocca Foundation was founded by Lee Iacocca and his family in 1984 to fund innovative approaches to a potential cure for diabetes.

The launch of the clinical trial received press coverage in the U.S. and the U.K. For more information on the MGH Immunobiology Laboratory, go to http://www.faustmanlab.org.