Doctor develops a vaccine to cure type 1 Diabetes – Beat0 finds out everything

1. A vaccine for diabetes. This is amazing. Share with us your vision.

We believe we have found a way to stop the destruction of islets by selectively killing the white blood cells that are attacking the pancreas. We are using an existing, safe and inexpensive vaccine called BCG.

2. What inspired you to research on diabetes Dr. Denise Faustman?

I am an immunologist by training and had been working on strategies to help islet transplants. What frustrated me is that transplanted islets suffer the same fate as the original islets. We have to stop the body from attacking the pancreas, and so I began to look for a way to do that.

3. How has been the journey from phase 1 to phase 2 of research on developing the vaccine for diabetes?

Exciting. We went from a small, but very successful, proof of concept Phase I trial to a much larger Phase II trial with 150 patients. It is a huge undertaking supported entirely by philanthropy–a clinical trial for the people by the people!

4. Will this vaccine prevent against Type 1 and Type 2 diabetes or only Type 1 diabetes?

We are testing BCG to treat type 1 diabetics who already have the disease. There are prevention trials underway in other parts of the world, but that is not our study population. We recently learned of BCG work going on in type 2, which is interesting, but very early and, again, that is not our focus with the current studies.

5. What frustrates you in this endeavor?

The pace of doing a clinical trial can be daunting. We are asking tough questions and doing a very long follow up. People want quick answers, but good answers take time.

6. What keeps you going un-frazzled?

The patients that come in every day to donate blood for our research and who seek to enroll in the trial. And the people who sacrifice big and small to help us with our work — things like a handwritten note with a check for $23 describing a lemonade stand fundraiser a child did for our work. All of the stories, the bike rides, the bake sales and just the dedication and energy of the people involved helps us.

7. We learn that many volunteers call you for participating in the trials. This must be encouraging and also indicates the hope diabetics have on the research.

Trial enrollment is very complex and we have set of criteria that the FDA has approved, so we cannot enroll everyone, but we try to give as many people the opportunity to participate in our research as we can. And, yes, we are very encouraged by the interest in the trial.

8. The world of diabetes , 10 years later. How do you vision it?

I cannot speculate on the outcome of our trial, but for type 1 diabetes, I hope we find ways to stop the body from destroying its pancreas (including by potentially using BCG) and help the body regenerate new beta cells. Ten years ago that seemed crazy, but now it is a goal that we can possibly achieve.

9. Your research trials are done on mice. Can success in animal models benefit humans as well?

We did mouse studies over ten years ago. Our Phase I trial was in human patients with long-term type 1 diabetes (mean duration: 15 years). We also have more than a decade of work on samples from human blood. We have not been a mouse lab for a very long time.

10. What next in diabetes research for you?

We are very focused on the Phase II clinical trial to see how BCG helps a large population of type 1 diabetics. We are also learning more about the mechanism of BCG, including how it creates a host relationship with the body and may influence the function of T cells. It is all very exciting. We have a lot of work to do.

Denise Faustman

Denise Faustman, MD, PhD, is Director of the Immunobiology Laboratory at the Massachusetts General Hospital (MGH) and an Associate Professor of Medicine at Harvard Medical School. Her current research focuses on discovering and developing new treatments for type 1 diabetes and other autoimmune diseases, including Crohn's disease, lupus, scleroderma, rheumatoid arthritis, Sjögren's syndrome, and multiple sclerosis. She is currently leading a human clinical trial program testing the efficacy of the BCG vaccine for reversal of long-term type 1 diabetes. Dr. Faustman's type 1 diabetes research has earned her notable awards such as the Oprah Achievement Award for “Top Health Breakthrough by a Female Scientist” (2005), the "Women in Science Award" from the American Medical Women’s Association and Wyeth Pharmaceutical Company for her contributions to autoimmune disease research (2006), and the Goldman Philanthropic Partnerships/Partnership for Cures “George and Judith Goldman Angel Award” for research to find an effective treatment for type 1 diabetes (2011). Her previous research accomplishments include the first scientific description of modifying donor tissue antigens to change their foreignness. This achievement earned her the prestigious National Institutes of Health and National Library of Medicine “Changing the Face of Medicine” Award (2003) as one of 300 American physicians (one of 35 in research) honored for seminal scientific achievements in the United States. Dr. Faustman earned her MD and PhD from Washington University School of Medicine in St. Louis, Missouri, and completed her internship, residency, and fellowships in Internal Medicine and Endocrinology at the Massachusetts General Hospital in Boston, Massachusetts.

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