In part 1 of this exclusive interview, Dr. Faustman discusses her research and why an old tuberculosis vaccine may lead us to a cure for type 1.
Steve Freed: Dr. Denise Faustman. It’s really exciting being able to talk with you because I’ve been keeping up on your research and it’s really exciting, some of the things that you are doing. It’s just an honor to have you here and to be able to question you about your current research. Maybe you can start off and tell us a little bit about yourself?
Dr. Faustman: It’s a privilege to be here and thank you for inviting me. We do research work at Harvard Medical School at Mass General Hospital that’s about type 1 diabetes. But probably remarkably, things we’ve worked on over the last 20 years are now in clinical trials, real clinical trials with FDA oversight to try to make a change in how we treat and view type 1 diabetes.
Steve Freed: I know you’ve done a lot of research when it comes to diabetes. A lot of things probably didn’t pan out. But now you are working on something that’s completely different and maybe you can kind of give us a general idea of why you’re here, what you’re presenting, and what’s new as far as your research goes? We’re talking about a vaccine, for the first time, [that] is for type 1 diabetes. So it’s kind of exciting if it can come and happen. So maybe you can give us a brief overview?
Dr. Faustman: The question Steve poses is actually really relevant. The question is what didn’t pan out? What didn’t pan out 20 years ago is why we started on this new mission of doing something very different for type 1 diabetes. I was actually recruited to Harvard Medical School to start the first islet transplant program; it was part of my thesis work. So that the East Coast could transplant islets like the Midwest, where I was from. We did transplants, at the Mass General Hospital, of islets. Some of the very early ones. Within 3 or 4 years, although there was great enthusiasm, we learned a very important lesson that people didn’t want to hear, and that was, when we put the islets cells in people with type 1 diabetes, they were getting a kidney transplant the same time. The kidneys survive just fine, but over and over again the islets went bye-bye. They got destroyed and so that was a giant wake-up call to us that even after 15, 20, 30 years of type 1 diabetes the auto-reactive immune response was very brisk, very brutal, and typical immunosuppressant drugs were not going to take it away. So we’ve literally spent the last 20 years working on the nasty problem that has not yet been solved and that is: why is autoimmunity present and how, in people that have long standing autoimmunity, can we take it away and take it away permanently? Because no poor islet transplant has any chance of surviving unless we get rid of this underlying disease process. So this is not a casual project where a lot of people view the lab as ‘eureka! I’ve got the solution.’ This is a 20 year flog to work mostly on human blood and people with type 1 diabetes to find a solution. So the solution we’re working on sounds all too simplistic, but it’s very heavily mechanistic based. It’s to bring forward a 100 year old vaccine called the BCG vaccine. So if you’re a practicing internist in the United States, you probably scratch your head and you go well, I might have heard something about this vaccine. It’s the most commonly used vaccine continuously in the last 100 years. It was developed over 100 years ago for the prevention of tuberculosis, but why we’re using this vaccine is that when you get immunized with the vaccine, whether you’re a type 1 diabetic or a control, it makes your immune response make a cytokine, a hormone called TNF. What we discovered over the last 100 years is in a multitude of autoimmune diseases you have too little TNF. TNF is your friend because TNF molds your immune system. It induces the good T-cells called Tregs and it kills the pathogenic T-cells. So this is about 10 years ago in mice, not that mice are humans right, in these N-stage diabetic mice. We published in 2001 that this was the first way and still remains the first way to permanently cure these mice. So that data was very promising. It was confirmed by global efforts and then the question came: how do we bring it forward into human clinical trials? So the exciting thing right now is that we are actually in phase 2 clinical trials here in Boston in people with long-term type 1 diabetes, trying to use this vaccine to permanently reverse the disease. Although that’s a pretty strong statement to make, it should be acknowledged that there’s now over nine global clinical trials using BCG to reverse other forms of autoimmunity. In the United States, there will soon be trials in a lupus like disease called Sjögren’s at the NIH, also using this vaccine, and there’s very advanced trials in Italy using this vaccine to halt new onset multiple sclerosis. It’s no longer just me, Steve, saying this vaccine holds great promise. It’s now a global effort to try to really thoroughly test the efficacy of this vaccine to lower hemoglobin A1Cs.
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