Treatment with generic vaccine kills autoimmune cells, temporarily restoring insulin production
BOSTON – A phase I clinical trial has confirmed that use of a generic vaccine to raise levels of an immune system modulator can cause the death of autoimmune cells targeting the insulin-secreting cells of the pancreas and temporarily restore insulin secretion in human patients with type 1 diabetes. Results of the study – led by Denise Faustman, MD, PhD, director of the Massachusetts General Hospital (MGH) Immunobiology Laboratory – are being published in the open-access journal PLOS ONE, and a larger Phase II trial is currently underway. The paper is available (link will only be active after embargo drops) at http://dx.plos.org/10.1371/journal.pone.0041756.
Faustman's team first reported in 2001 that inducing expression of tumor necrosis factor (TNF), previously shown to destroy insulin-autoreactive T cells, cured type 1 diabetes in mice by permitting pancreatic islets to regenerate. Since high doses of TNF are toxic to humans, the clinical trials use the bacillus Calmette-Guérin (BCG) vaccine, which safely elevates TNF levels. The Iacocca Family Foundation has been the primary supporter of this work.
"We believe we have validated in humans the treatment pathway we originally reported in mice and are seeing early evidence of effectiveness," says Faustman. "Our findings show that this simple, inexpensive vaccine modifies the autoimmunity underlying type 1 diabetes, boosting TNF production and killing the disease-causing T cells, which appears to briefly restore pancreatic beta-cell function. This is not a prevention trial. We are trying to create a regimen that will actually reverse type 1 diabetes in people who are living with the disease. We anticipate that the Phase II trial will give us more direction for turning BCG into a more sustained treatment, including the right dose and the frequency of vaccination needed to sustain a therapeutic response."
Emily,thanks so much for posting this update. I have donated blood to the Faustman lab twice; and will go again in 2013. So excited about this. I may NOT get picked for the Phase II clinical trial, but I am glad to further the effort towards a cure by my blood donations and giving the lab some money, both annually, when I can.. We need to have a telethon, or get other corporate donors.. As old as I am ( 57); I truly believe that even I will see a cure for type one in my lifetime.
I meant future Phase II and Phase III treatment trials are the ones I may not be included in.
Emily - Thanks for the update on Dr. Faustman's work. Wouldn't it be great if the cure for T1 diabetes turned out to be a series of shots using a drug that's long been a generic and demonstrably safe to use? I hope the good doctor's pursuit proves fruitful -- in my lifetime, of course!
To me, this sounds like the real deal. Maybe a cure is just around the corner.