Without philanthropic support, researchers at Mass General may not have been able to enter into clinical trials that have the potential to develop a cure for type 1 diabetes.
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A Research Breakthrough: Hope for Patients with Diabetes
In caring for individuals with diabetes, Massachusetts General Hospital researcher Denise Faustman, MD, PhD, became frustrated that nothing seemed to change. “I would prescribe the newest insulin pump and then follow the patient’s complications over time,” she says. “In order to make bigger dents in the lives of patients, I needed to go back to the lab.”
Since making that decision 20 years ago, Dr. Faustman has asked basic questions about the immune system and the underlying process that causes type 1 diabetes. She determined that, contrary to the prevailing belief, the insulin-producing islet cells found in the pancreas can regenerate. Her landmark study in 2001 established this to be true in mice, and there is evidence that it may be true in humans.
Diabetics everywhere are watching the clinical trial, now underway, that is testing Dr. Faustman’s premise: that the immune system can be stimulated in a way that eliminates only the T cells responsible for type 1 diabetes. David Nathan, MD, director of the Diabetes Center at the Massachusetts General Hospital, is collaborating with Dr. Faustman on the study. Participants receive an injection of Bacillus Calmette-Guerin (BCG), a well-known, safe medication, recently used to treat tuberculosis, which kills off the problematic T cells – the first step in “re-educating” the immune system to allow healthy new islet cells to grow.
“It is a fairly unique clinical trial because it has the noble goal of actually curing disease,” says Dr. Faustman. “No one else is doing this. As a result, we have had patients flying in from Africa and taking buses from northern Canada in order to enroll.
“It’s a new paradigm — the notion that we can regrow things. We’re only at the beginning of understanding this, but I think there will be many more sources of regeneration.”
Dr. Faustman is grateful for support from private philanthropy. “This sort of novel, high-benefit research has been largely supported by private foundations and individuals, including Jackie Fusco and Sue Root,” she notes. “These women are smart, well-informed and driven. Nobody knows more about this disease than the patients and the parents who care for them.”
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