I will be the first to admit. I freaked out a little bit when Dr. Denise Faustman took the time to answer my questions I sent to her regarding her research. I was absolutely thrilled! Now, in my 11 years as a journalist, I've interviewed a few notable people - Nine Inch Nails, Seether, Foghat and Tom Daschle (when he was Senate Majority Leader) - but I did not get that thrilling rush in any of those.
Without further ado, the Q&A with Faustman. The questions are answered and gives me more reason to hope!
1) Please explain your study.
Faustman (F): Our study is a placebo-controlled Phase II human clinical trial. It will test whether giving patients with established (not newly diagnosed) type 1 diabetes repeat BCG vaccinations will have a beneficial effect on their disease. We specifically want to see if there is any improvement in the ultimate end point for diabetes, a change in HBA1c. As you know we had some restoration of insulin secretion among those that receive the BCG vaccine in Phase I so now is the time to do a larger trial and also look for very important endpoints like changes in HbA1Cs.
2) What made you begin this study?
F: There is a long history here. I started my career treating a lot of patients with type 1 diabetes and was frustrated by the fact that, even with insulin, many of them were going to have trouble controlling their blood sugar, and all of them faced the risk of developing diabetes-related complications in the long term. That prompted me to begin my journey as a researcher, where I was involved in some early islet transplantation research. The problem there was that once we transplanted the islet cells into a patient with type 1 diabetes, the new cells would get attacked because of the ongoing autoimmune disease. This led me to start researching ways to get at the underlying cause of this disease--and then to the mouse trials where we reversed established type 1 diabetes in mice using a two pronged approach to kill the bad T cells to stop the autoimmune attack and then "re-educate" the immune system so it would not resume the attack on the insulin-secreting cells of the pancreas. The treatment we used to kill the bad T cells and halt the autoimmune attack is the equivalent of BCG, which is in clinical trials today.
3) Is this a cure? Why or why not?
F: In mice with advanced type 1 diabetes, we used two approaches to permanently eliminate their disease - no insulin, regenerated pancreas, no treatment necessary the rest of their lives. The first approach killed the existing bad T cells that attack and destroy the insulin secreting cells of the pancreas, while the second approach "re-educated" the immune system so that new “bad” T cells could not be generated. The BCG clinical trial is a translation of that first approach in humans. It does not permanently address the autoimmune attack, and would likely require repeat dosing, which we are exploring in our trial. However, based on the data we have, repeat doses of the BCG vaccine may be able to continue to arrest the immune attack on the insulin secreting cells enough for the pancreas to begin regenerating and secreting at least some insulin. In our research, we have learned that this is likely to be significant in helping to prevent long–term diabetic complications and long term normalize blood sugars.
4) How do you compare your study to other studies that claim to either be close to a cure or have cured?
F: There really aren't any cures for type 1 diabetes today, not in the way that most people think of cures. What I can say is different about our study is that we are testing an inexpensive generic drug, and that we are focused on people who have what we would call advanced or established type 1 diabetes. These are not people with new-onset disease, but people who have been told for many years that their pancreas was "destroyed" - that it could no longer secrete any insulin. We now know that for many people with advanced type 1 diabetes, this simply isn't true, and the BCG vaccine might be a way to get their pancreases to produce even more insulin and help protect them from the complications of this disease. We are also different in that this would be a fairly "easy" therapy for type 1 diabetics, since it involves only periodic vaccination with a drug that is safe and inexpensive, rather than a treatment that would require immunosuppressants, daily insulin, daily blood sugars, cell transplants or expensive drugs or devices. This is the first development of an immune-intervention in people with long standing disease to attempt to reverse the hyperglycemia.
5) How far away are you from clinical trials or have you already started?
F: We are currently enrolling patients into our Phase II trial. Our Phase I trial was completed a few years ago and showed that repeat vaccination with BCG could at least temporarily restore some level of insulin production in patients who had been living with type 1 diabetes for an average of 15 years. Since completing our Phase I trials, we have been raising the money and working with the FDA and our own clinical trial teams to plan and launch the Phase II trial. Since this is a non-profit clinical trial program (because BCG is a generic drug, off-patent), rather than one with a pharmaceutical sponsor, raising the money to support the trials can be a very slow process!
6) What are the criteria for participants? How long will the trials last?
F: The current Phase II trial will last for 5 years. For the first group of patients we are enrolling, the basic eligibility criteria are that potential participants must be age 18 or older and have had type 1 diabetes for fewer than 20 years. Other specific criteria are listed on the clinicaltrials.gov site: http://clinicaltrials.gov/ct2/show/NCT02081326. We hope to announce at a later time that we will be enrolling people with even longer durations of type 1 diabetes.
7) Do you have any reason to believe your cure or treatment will not become available to the public? Such as being prevented for reaching fruition by pharmaceutical companies or the FDA?
F: Because BCG is a common globally produced generic drug for another indication (tuberculosis prevention), it is highly unlikely to be developed in a for-profit setting. Also if BCG is successful at normalizing blood sugars in long term diabetics it might be viewed by some as competitive to current treatments. For that reason, we are conducting the entire program ourselves at Massachusetts General Hospital, a non-profit setting which gives the highest chance of bringing this drug forward. As is any group working on developing a drug in the US, we are working closely with the FDA to ensure we are designing the trial appropriately and collecting the right data to allow the drug to be approved if the trials demonstrate that it is effective.
8) What do you have to say to Type 1 diabetics who believe no cure will be realized because there is not enough profit?
F: We are working to change that opinion!
Any other Type 1's got that same warm, fuzzy feeling that I do? This is viable research seeking to truly cure Type 1 diabetes by tackling the heart of the problem - the immune system! Faustman answered these questions honestly and with detail. And with a person such as Lee Iacocca providing financial support, I have more hope than ever that this will come to fruition.
Please give your support to this wonderful woman and her research. http://www.faustmanlab.org/