Type 1 diabetes, the autoimmune form that usually strikes young people, is big and getting bigger: As many as 3 million Americans may have it, and their numbers are growing quickly and mysteriously.
So any promise of potential progress is a big deal, and it was splashy news back in 2012 when Dr. Denise Faustman, director of the Immunobiology Laboratory at the Massachusetts General Hospital, reported positive results for a test of a tuberculosis vaccine called BCG — Bacillus Calmette-Guerin — in a tiny trial of three longtime diabetes patients. Of particular appeal: BCG has been around for nearly a century, has been shown to be safe and, long since generic, is also cheap.
Now, Faustman has just announced the launch of a far bigger human clinical trial, aiming for 150 patients from ages 18 to 65. Though BCG is known as a vaccine, the trial will use it not to prevent diabetes but to try to reverse the disease — at least partially — in patients who already have it.
Dr. Faustman’s research, funded mainly by the Iacocca Foundation, has been considered somewhat controversial, so the results of the new trial — which is expected to last five years and is a “Phase II” trial to assess how effective the treatment is — could lay that debate to rest.
Our conversation, lightly edited:
So how do you see the headline here? And should it begin ‘This is not a cure’? What’s your main message to the public?
I think the main point of what we’re doing is: These are the first trials trying to intervene immunologically in people with long-standing autoimmunity and trying to reverse the disease, but doing it with a cheap, inexpensive, hundred-year-old drug.
But how would you calibrate the promise, or hope, of how well this might work for people?
It’s interesting because these trials, although we were kind of the first in the world to start in Phase I, they’ve caught on, on a global basis — using repeat BCG in diverse autoimmune diseases, such as multiple sclerosis, such as Sjögren’s disease. So, although we were kind of lonely to begin with, suggesting this hundred-year-old drug might have major therapeutic impacts, we’re not so lonely anymore. In fact, in Europe, there are Phase III trials going on in multiple sclerosis.
So, we’re seeing clinical effects in patient populations where people thought it wasn’t possible to reverse or partially reverse a disease. So we’re pretty hopeful that this inexpensive way might make a significant dent on the clinical course of a disease and do it at incredible cost savings to the public.
So, does that go as far as possible ‘cure’? Continue reading