The Nuances of the Modern Research ParadigmFriday, April 08, 2011
Researcher Denise Faustman, MD, Ph D, wants to find a cure to type 1, but funding is slow in coming.
By: John Parkinson, DiabetesCare.net Clinical Content Coordinator
Dr. Denise Faustman (pictured) understands the practicalities and the subsequent shortcomings of the research paradigm today.
In 2001, her lab reported they had reversed end-stage type 1 diabetes in mice by using Bacillus Calmette-Guerin (BCG) vaccine, an inexpensive generic drug. “The data was better than we ever expected it to be,” says Dr. Faustman of her pre clinical trial work.
Previously, Faustman’s lab as well as some other labs had showed that they could eliminate the abnormal white blood cells in mice and people with type 1 diabetes by temporarily elevating levels of an immune modulator called tumor necrosis factor (TNF). BCG was known to raise TNF levels in humans as it was already on the market and indicated as a tuberculosis vaccination and treatment for bladder cancer. Another appeal, says Dr. Faustman was that the drug already had a long safety record going back several years.
In the ten years since that promising report on animal models, Faustman and her lab just completed phase I human clinical trials last summer. In phase I, the trial showed that low-dose, multiple BCG vaccinations were safe in people with type 1 diabetes.
Today, her lab is in a holding pattern as she awaits sufficient funding to carry out phase II of the trials. Thus far, they have secured $5.5 million of the $8.5 million budget needed.
They have had only one major donor, the Iacocca Foundation, which was developed by the former Chrysler CEO, Lee Iacocca, after his wife died several years ago from type 1 diabetes complications. “This doesn’t fit into the normal circle of life,” acknowledges Dr. Faustman, of her project. “When you go forward with a philanthropic project, you don’t go to the normal players you go to for money. “
It is because of this long, slow process of raising money that Dr. Faustman, who is director of the immunobiology laboratory at the Massachusetts General Hospital and an associate professor of medicine at Harvard Medical School, empathizes with people who are struggling with type 1 diabetes. For those afflicted with this autoimmune disease, it does not appear that medical science is making any inroads into finding a cure, especially when many of these patients have been hearing for years a cure would have been discovered by now. And that underlying frustration can be found in recent diabetes blogs, newsletters, and Dr. Faustman herself.
She also understands the nuances associated with the research. There are no guarantees of success in the end—quite the contrary actually. Scientists could spend years toiling away in research that does not result in a cure or treatment for a disease. Additionally, Dr. Faustman also knows that in order to bring a new drug to market, it would cost hundreds of millions, if not a billion dollars, so there is a reluctance on the part of pharmaceutical companies to invest so much money and end up with nothing to show for its investment.
And with the same ability to understand peoples’ frustrations as well as her peers’ reluctance to go down the road of this research, she can be equally critical of the scientific community overall for the lack of human trials in diabetes type one research. Whereas, there is a lot research done in this area on animal models with subsequent papers written, Dr. Faustman says it does not translate into very many human clinical trials.
Dr. Faustman has been involved with autoimmune research for a number of years. She has been part of some important discoveries including introducing modifying antigens on donor tissues to prevent rejection. This is being studied in human clinical trials for diverse human diseases treatable with cellular transplants. “Some of the strongest support for what we are doing [in diabetes research] comes from data of other autoimmune diseases,” stresses Dr. Faustman.
Weary that the promise has been made before, and understanding the research process, Dr. Faustman still does see the potential for a diabetes cure. “I would like to think it is feasible,” says Dr. Faustman. She believes people outside the scientific community can put more pressure to bear on medical science by demanding more human trials to move the possibility of a cure along. “Make [researchers] more accountable for their data,” says Dr. Faustman.
For now, Dr. Faustman’s work requires her to go back and forth between fundraising and research. “The research is the fun part, acknowledges Dr. Faustman. “Raising money is hard.”
To find out more about Dr. Faustman’s research, go to www.faustmanlab.org.
Originally posted by DiabetesCare.net on April 11, 2011.