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Dr. Denise L. Faustman

Dr. Denise L. Faustman: Groundbreaking Type 1 Research

February 21, 2008

Dr. Denise L. Faustman completed her internship, residency, and fellowships in Internal Medicine and Endocrinology at the Massachusetts General Hospital and became an independent investigator at the MGH and Harvard Medical School in 1987. Currently, she is an Associate Professor of Medicine at Harvard Medical School and Director of the Immunobiology Laboratory at the MGH. She is a member of the AAAS and serves as a frequent member of the Institute of Medicine in Washington, DC. Dr. Faustman has spent the last decade researching the nature of the molecular defect in T-cells that results in the development of autoimmunity. This work led her to discover a novel way to treat diabetic mice, accomplishing for the first time ever, the permanent reversal of established diabetes. Her earlier research accomplishments include the first introduction of the concept of modifying the antigens on donor tissues to prevent their rejection, a scientific accomplishment that is now in human clinical trials for diverse human diseases treatable with cellular transplants. Currently Dr. Faustman is working on strategies for applying this method to treat human disease and to explore the applicability of this process in other autoimmune diseases.

Diabetes1: Can you please introduce yourself and tell us a bit about your background? What are you researching/studying right now?

Dr. Faustman: My name is Denise Faustman, MD, PhD, and I am an Associate Professor of Medicine at Harvard Medical School and director of the Immunobiology Laboratory at Massachusetts General Hospital. In 2001, my lab reversed type 1 diabetes in mice with end-stage disease, and we have been working to translate this into human clinical trials. The first trial (an FDA-approved Phase 1 study) will start this year. It will test a generic drug called bacille Calmette-Guerin (BCG) in patients with type 1 diabetes. We believe that this drug may be able to target and eliminate one type of defective white blood cells in patients with type 1 diabetes that cause disease. As you know, type 1 diabetes is an autoimmune disease, meaning that the body’s white blood cells mistakenly get programmed to attack healthy cells. If we can stop this attack, we are one step closer to reversing this disease. In addition to doing this work on type 1 diabetes, my lab is also investigating therapies for other autoimmune diseases, such as Crohn’s disease and lupus.

Diabetes1: Why did you decide to go into this area of research?

Dr. Faustman: I felt that this type of basic research could be translated to humans faster than basic research such as looking at the worm genome or fruit fly development. That type of research is important to advancing science, but for me, I wanted to be involved in something where the fruits of your basic research could be translated much more immediately into the clinical setting.

Diabetes1: What are some of the most common questions patients ask about this disease, and what would you tell them?

Dr. Faustman: One of the most common questions we hear is: “Why are there so few labs working on therapies to reverse disease for people who already have diabetes? Why has there been so little effort in this area?” My answer to that is that people go where success is. In animal models, there have been many successes curing mice with early stage disease, but little success curing mice with established or late-stage disease. We decided to do high-risk/high-benefit work and were lucky enough to have success curing late-stage mice. Now we will be testing part of our intervention in human trials. Also very few investigators want to perform experiments where the chance of success is low since it may be a loss of their career but great benefit to the public. Unfortunately, positive data is needed to renew grants.

Diabetes1: What is the most exciting thing about the field of diabetes research?

Dr. Faustman: One of the things we have discovered is that the pancreas can regenerate. This might have a therapeutic impact in the future, and it is very exciting.

Diabetes1: Where do you expect us to be in 10 years?

Dr. Faustman I hope that in 10 years we will see that with repeat BCG vaccination, humans with type 1 diabetes will have periods of normal glycemia without having to take insulin injections.

Last updated: 21-Feb-08

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