In the State of the Cure 2014 report, BCG was identified as one of the eight Practical Cure solutions in human clinical trials.
BCG, a project led by Dr. Denise Faustman, Director of the Massachusetts General Hospital Immunology Laboratory, is a tuberculosis drug that Faustman believes will stop the autoimmune attack and allow residual beta cells to replicate. Phase I trials were completed a few years ago with inconclusive results. Phase II trials are currently underway. We look forward to seeing the results. One cautionary note is that the Phase II trials is expected to last 8 years, one of the longest Phase II of any project we have tracked, and will cost $25MM, making it the most expensive Phase II trial of any project we have tracked.
Cara Murphy: How did you and your team get involved in type 1 diabetes research?
Dr. Denise Faustman: During my medical training, I specialized in endocrinology, so I started my career treating a lot of patients with type 1 diabetes. I felt frustrated by the fact that pure clinical care just wasn’t going to have a significant enough impact on patients with type 1 diabetes. 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. It is a nasty disease. I wanted to do more about it, so I began my journey as a researcher. In general, I feel like we are overdue on making significant advances in the type 1 diabetes field. We need to see if there is something we can do beyond making a better insulin—something that really gets at the underlying cause of this disease. This is the goal of my research.
CM: Please describe to us non-scientists what BCG does.
DF: BCG is short for “bacillus Calmette-Guerin.” It is a generic drug that is prepared from a weakened strain of bacteria. It has an impeccable human safety profile and has been administered to more than four billion people since its introduction over 90 years ago. BCG is known worldwide for its use as a vaccine to prevent tuberculosis. It is also administered as an infusion into the bladder to treat bladder cancer. One of the things that BCG does is stimulate the body to make a substance called TNF—tumor necrosis factor—which is a cell signaling protein that helps to regulate the immune system. In our research, we have shown that using the BCG vaccine to temporarily elevate TNF production in the body may be beneficial for people with type 1 diabetes. Specifically, in our Phase I human clinical trial, we demonstrated that BCG vaccination stimulates the body to kill the “bad” T cells—the ones that attack the insulin-producing cells of the pancreas—which appears to allow the pancreas to begin to produce at least some insulin again, even in people who have had type 1 diabetes for a long time. Obviously, we need to collect more data, which we’ll do during our upcoming Phase II trial, but we are very hopeful about the promise of this affordable vaccine that is widely available around the world.
CM: How many years have you been on this journey and what keeps you going?
DF: I have been doing type 1 diabetes research for over 25 years. One of the main things that keeps me going is our patients and all those who are affected by this disease. Even today, many people who are outside of this field or untouched by type 1 diabetes don’t realize that it’s life threatening, or even how intense it is for a patient or caregiver to manage the disease day by day. As a physician and researcher, I believe there is a lot of opportunity to do more for people with type 1 diabetes. The field is ripe for discovery and change.
CM: What are 3 things about your project that you want the type 1 diabetes community to know?
DF: First, with our BCG Human Clinical Trials Program, we are doing some of the first immune intervention trials in the world for people who have had type 1 diabetes for many years. This is unlike most type 1 diabetes trials to date, which have primarily focused on preventing type 1 diabetes from occurring or on halting disease progression in people with new-onset type 1 diabetes.
Second, we are trying to develop an affordable vaccine to reverse type 1 diabetes.
Last, we are now actively recruiting patients for our Phase II study. In this phase of recruitment, we are looking for people age 18 and older who have had type 1 diabetes for less than 20 years. We are asking everyone who is interested to either call us at 617-726-4084 or email us firstname.lastname@example.org, using the subject line “Faustman Lab Research Interest.” For new patients and those that have not donated blood samples at the Faustman lab in over a year, we will schedule a 30-minute screening visit and blood donation at our facility in Boston.
CM: If successful, how will it change the lives of people living with T1D?
DF: Although we won’t know the results until the trials are completed, we do hope that BCG might reduce, prevent or eliminate diabetic complications and make diabetes care easier by reducing the need for insulin.
CM: In what stage of human trials is BCG? When can we expect to learn the next set of results?
DF: We are about to begin to enroll patients into the Phase II trial. We will follow participants in the trial for five years to evaluate the long-term effects of repeat BCG vaccination, which means that it will be some time before we are able to announce results. As some might know, BCG is also being tested in other autoimmune diseases, such as multiple sclerosis. I encourage people to watch for that data as well. I am very excited by all of the new movement around using BCG in autoimmunity.
CM: Are you still recruiting for the trial? If so, how can people get in touch with you?
DF: Yes, as mentioned above, we are now actively recruiting patients for our Phase II study. In this phase of recruitment, we are looking for people age 18 and older who have had type 1 diabetes for less than 20 years. We are asking everyone who is interested to either call us at 617-726-4084 or email us at email@example.com, using the subject line “Faustman Lab Research Interest.” For new patients and those that have not donated blood samples at the Faustman lab in over a year, we will schedule a 30 minute screening visit to donate a blood sample at our facility in Boston.
CM: What are the biggest obstacles your team faces?
DF: Our biggest obstacle is raising money for this research. We are trying to bring a generic drug forward for use in type 1 diabetes, which essentially makes this a non-profit drug development program. That means that we absolutely rely on individual donations and philanthropy to support our program. Right now, we have about $18.4 million of the $25.2 million we need to fully support the Phase II trial. This will cover everything from paying for the regulatory and manufacturing support we need to seeing patients, running equipment, and processing blood samples. What some people don’t realize is that the typical cost of developing a new drug is in the billions. By finding a new use for an existing generic drug, we are following a much, much less expensive model of drug development.
CM: What can the type 1 diabetes community do to support your project?
DF: Please tell others about this research, and contact us if you would like to participate or learn more. Anyone who is interested in making a financial contribution can visit our site, www.faustmanlab.org. By clicking on our “Support” tab, you can find information on how to make a tax-deductible donation. Finally, if you would like to keep up to date with our news, you can email us to sign up for our newsletter (firstname.lastname@example.org), or you can follow us on Facebook (www.facebook.com/FaustmanLab) or on twitter (@FaustmanLab).