Denise Faustman, MD, PHd
The Faustman Lab at work

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Research by the Faustman Lab

Overview
For over 15 years, the Faustman laboratory has pursued the reversal of established autoimmunity as the prerequisite to successful treatment of type 1 diabetes and other autoimmune diseases. The lab's goal is to investigate treatments that permanently eliminate underlying disease, not just manage symptoms. This approach has led to the discovery of a novel way to permanently reverse type 1 diabetes in mice and is now being tested in human clinical trials.

In addition to bringing forward a potential new type 1 diabetes treatment for humans, another of the lab’s goals is to identify new treatments for other autoimmune diseases such as Crohn's disease, lupus, scleroderma, Sjögren’s syndrome, rheumatoid arthritis, and multiple sclerosis. Significant evidence exists to suggest that these other autoimmune diseases, which have cellular defects similar to those seen in type 1 diabetes, can benefit from the approach the Faustman lab is testing in type 1 diabetes.


Research History
Autoimmune diseases are diseases such as type 1 diabetes, lupus, rheumatoid arthritis, and multiple sclerosis that occur when defective white blood cells mistakenly attack the body’s own healthy tissues. Over the past decade, the Faustman lab’s research has focused on a defect in the white blood cells that cause autoimmune diseases in the mouse and human.

In her research, Dr. Faustman discovered that this cellular defect makes the disease-causing cells sensitive to death in the presence of certain levels of a substance called tumor necrosis factor (TNF), an immune system protein made naturally by the body. Dr. Faustman and colleagues used a brief, non-toxic treatment to induce TNF in mice with advanced diabetes and permanently eliminated their disease. This therapeutic approach not only stopped autoimmunity and restored normal blood sugars in the mice, but also precipitated the regeneration of their insulin-producing cells. These results were published in 2001 in the Journal of Clinical Investigation and in 2003 in Science.

The 2003 Science paper also identified the spleen as a potential new source of adult stem cells that could form new islets in formerly diabetic animals and speed disease reversal and regeneration. However, disease reversal was also seen in animals that did not have live spleen cells introduced. In the human clinical trials currently testing one part of the Faustman lab’s approach to type 1 diabetes reversal, there is no intent of spleen cell transplants for human patients. Dr. Faustman and colleagues hope there is sufficient regeneration and rescue of the islet cells so that transplant is not required.

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