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.