A Q & A With Denise Faustman, MD

The content for this column is provided by the Iacocca Foundation

Some of the most frequently asked questions about her research

Feb 1, 2005

Denise Faustman, MD, of Massachusetts General Hospital (MGH) and Harvard Medical School, was able to eliminate type 1 diabetes in mice (Science, November 2003). With the help of the Iacocca Foundation, she is working to raise the $11 million needed for the first three years of a program to investigate this potential cure for human type 1 diabetes.

Here she answers some of the most frequently asked questions about her research.

How will you use the $11 million you are raising?

The $11 million covers the first part of our program, which has three components:

1. Additional studies in mice to refine the therapy to be tested in type 1 diabetics

2. Developing and refining a blood test to allow us to evaluate how well the potential therapy works

3. The initial phase 1 human clinical trial

The human clinical trial will test one part of the two-part therapy to specifically eliminate the T cells that incorrectly destroy the islet cells. Each of the two therapy arms will be developed separately. The FDA approval allows one therapy to be tested at a time.

Can you tell us more about this phase 1 trial?

David M. Nathan, MD, at MGH, will lead the trial. It will evaluate the safety, optimal timing for administration and best dosage of a bacillus Calmette-Guerin (BCG) vaccine in type 1 diabetics to see if BCG can help to eliminate the defective cells that attack and kill the insulin-producing cells. This study is approved by both the FDA and by the human studies committee at MGH.

What is BCG? Is it safe?

BCG is a weakened strain of bacteria that has been used for over 80 years as a vaccine to prevent tuberculosis. It is also used in certain cancer treatments. It has been administered to more than 4 billion people and has an established safety profile over a very broad dose range in humans.

We expect the effect of BCG will be similar to that of the agent we used in mice to eliminate the memory T cells. The phase 1 trial using BCG is the first step for finding out if the approach my lab used in mice is applicable in humans.

How do we define a diabetes cure?

A cure for type 1 diabetes is a treatment that permanently halts disease progression after a limited exposure to the therapeutic agent(s), eliminates the underlying cause of disease, and restores normal islet function. A cure does not introduce new disease or defects nor otherwise increase morbidity or mortality. Finally, the treatment must be effective in animals with full-blown disease or long-term disease because results in prediabetic or recently diabetic animals have not been a good predictor of success in humans, and because, for the vast majority of type 1 diabetics who have little or no remaining islet function, a treatment that may delay disease progression represents no cure.

Why don’t you have large government funding for this trial?

Government agencies have specific criteria for the types of trials they fund. Many prefer to fund later-stage trials in which the concepts have been proven and the phase 1 testing and blood assay work are completed. We are hoping that once we can demonstrate that our potential therapy is applicable to humans, we will receive government funding for the phase 2 and phase 3 trials.

How Can I Help?

The cost of the first three years of clinical trials at Massachusetts General Hospital is estimated at $11 million. Contribute to the Join Lee Now campaign and support these efforts to cure type 1 diabetes in humans. Donations may be made over the Internet or by mail.

Internet: To make a donation over the Internet, please visit www.JoinLeeNow.org.

U.S. Mail: To mail your donation, please make checks payable to “Iacocca Foundation.” Send donations to the following address:

The Iacocca Foundation
17 Arlington Street
Boston, MA 02116

Gifts: Give the gift of a donation to support important diabetes research. Donations can be made in a loved one’s name. For more information about gift-giving, call Danielle Briscoe at (212) 255-5340.

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Feb 1, 2005

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