The Path to a Cure

The research of Dr. Denise Faustman









 
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Dr. Denise Faustman 

Dr. Denise Faustman’s path to a cure for type 1 diabetes has been a long and fascinating one. Dr. Faustman is currently an associate professor at Harvard Medical School and the Director of the Immunobiology Laboratories of Massachusetts General Hospital. She completed her training for a Ph.D. in molecular biology, as well as an M.D., at Washington University School of Medicine in St. Louis. Dr. Faustman’s doctoral research project was with Dr. Paul Lacey, the pioneer in the isolation of islet cells—the insulin secreting cells or beta cells of the pancreas. After completing her residency at Massachusetts General Hospital, Dr. Faustman was recruited by Harvard Medical School to create an islet transplant program. Although she perfected the procedure of isolating the islets, she discovered that unless the autoimmunity that causes diabetes was understood and halted, no amount of new beta cells was going to cure type 1 diabetes. The underlying autoimmune process would continue to destroy them. Dr. Faustman began a methodical investigation into the nature of type 1 diabetes autoimmunity, identifying the immune system’s white blood cells, called T cells, that were involved in attacking the beta cells of the pancreas. She focused on identifying the defects in these faulty T cells and then determined how to exploit these defects to selectively kill them. Eliminating the defective or bad T cells eliminates the cause of type 1 diabetes and, therefore, the disease itself.


During an exciting series of ground-breaking experiments that cured type 1 diabetes in mice, Faustman’s team made the completely unexpected discovery that the beta cells that produce insulin in the pancreas regenerated and started secreting insulin once the disease process that was attacking them was removed. Prior to this breakthrough, it was assumed that the beta cells would have to be replaced by islet transplants, stem cells or some other means. Another important discovery that Dr. Faustman made from years of screening and studying human blood was that the same type of diabetes-causing T cells were present in humans with type 1 diabetes and had the same defect as the bad T cells in mice. Therefore, the same treatment should have the same effect in humans and cure diabetes in people, too.


Dr. Faustman’s next step was to find a drug to eliminate the bad T cells. She focused on identifying a generic drug that was able to induce the body to produce a compound called TNF or tumor necrosis factor. TNF’s function is to kill invading germs and tumor cells. The diabetes-causing T cells have a defect in which TNF selectively kills them. But TNF has no effect on the healthy T cells of the immune system. Dr. Faustman found that a generic drug called BCG induces TNF production. It is inexpensive, safe and readily available all over the world. Dr. Faustman is currently conducting FDA clinical trails in humans that will eventually determine the precise timing and dosing of BCG that is needed to eliminate the bad T cells that cause type 1 diabetes. The FDA requires several more years of study before BCG can be approved to treat type 1 diabetes. Our goal is to help raise the funding for Dr. Faustman to continue these trials, so this treatment will be available to cure type 1 diabetes in people. Similar defects most likely exist in other autoimmune diseases, which could lead to targeted treatments to eliminate defective T cells that cause diseases such as lupus, multiple sclerosis and rheumatoid arthritis. This research has much more far-reaching implications than a cure for type 1 diabetes.


Below, you’ll find several articles, as well as radio and TV interviews with Dr. Faustman. For those who want to delve deeper, a link to scientific publications is provided. For more details about this research and the latest updates on the BCG clinical trials, visit the Faustman Lab Web site.



Learn more


Changing the Face of Medicine   A brief biography of Dr. Faustman


Articles in the Popular Press

A Diabetes Researcher Forges Her Own Path to a Cure  — New York Times

A History of Being Innovative and FunThe Boston Globe

Dr. Denise Faustman Continues Working Toward the CureDiabetes Health

Diabetes: Of Mice and MenNewsweek

After Initial Rejection, Scientists Back Work On Cure for DiabetesWall Street Journal


Interviews

Tracking Developments in Diabetes Research  — NPR Radio Interview 2008  (Post Human Trials) with Dr. Faustman

Moving a Diabetes Cure from Mice to People — NPR Radio Interview 2006 (Pre Human Trials) with Drs. Faustman and Nathan

ReachMD radio interviews — Very interesting series of interviews detailing research. Geared towards M.D.s, so a bit technical.

Recent interview on NECN

Informal video Interview — Dr. Faustman on Diabetes Cure Trial by David & Elizabeth Edelman and Bernard Farrell


Scientific Publications

Selected publications from the Faustman Lab’s website