Thoughts About Dr. Faustman's Trials Starting?

You all read it on Bernard’s blog post.

How do you feel about the perspectives of human trials for type 1 diabetes cure having started?

In the meantime, please help digg Bernard’s post.

That is exciting. Too bad I don’t qualify: I had the BCG vaccination as a child and I’m too old also.

I think it is wonderful. I hold a walk for The Iacocca Foundation every year to support Dr. Faustman’s research. I know it may not pan out, but I’m glad she is getting the chance to see it is does.

Here’s the press release if anyone is interested:

Thursday, March 13, 2008

Emily Parker, MGH Public Affairs
(617) 724.6425

MGH Initiates Phase I Diabetes Trial

BOSTON, MA – Scientists at the Massachusetts General Hospital (MGH) have initiated a phase 1 clinical trial to reverse type 1 diabetes. The trial is exploring whether the promising results from the laboratory of Denise Faustman, MD, PhD, can be applied in human diabetes. Faustman’s previous studies have shown that mice with a form of diabetes that closely resembles type 1 diabetes in humans can be cured. In the animal studies, a commonly used vaccine that provides protection against tuberculosis, called Bacillus Calmette-Guerin (BCG) was used effectively to deplete the abnormal immune cells that attack and destroy the insulin producing cells of the pancreas. The first step in the human study, which is currently enrolling volunteers, is to determine whether the same strategy using BCG vaccination can be used to modify the abnormal autoimmune cells that are present in type 1 diabetes, sometimes called “juvenile-onset” diabetes.
“We are pleased to be starting human clinical trials,” said Faustman. “Human trials take time, but we are making the step from curing diabetes in mice to determining whether it will work in men and women with diabetes.”
Type 1 diabetes usually starts during childhood or adolescence and can cause a variety of severe complications including kidney failure, loss of vision, amputations, heart disease, and strokes. It occurs when a person’s immune system attacks and destroys the insulin-producing cells in the pancreas. In the absence of insulin, which is necessary for sugar and other nutrients to enter cells, blood sugar levels rise. The risk for developing complications is closely linked to the elevated blood sugar levels over time. If blood sugar levels are well controlled, the long-term complications can largely be avoided. However, the so-called intensive therapy that is required to maintain near-normal sugar levels requires life-long demands on the patient, including frequent blood sugar monitoring and at least 3 daily injections of insulin or use of an insulin pump, along with restrictive diets. Insulin doses must be adjusted based on blood sugar levels, dietary factors, and anticipated exercise. A cure for diabetes has been highly sought after and has attracted much research interest.
The clinical trial is using the BCG vaccine for several reasons. BCG has been used safely for nearly 80 years as a tuberculosis vaccine. It is now being used in the human trial because it causes a low-grade inflammatory reaction, which in the mouse model of autoimmune diabetes lead to the destruction of the abnormal autoimmune cells.
David M. Nathan, MD, director of the MGH Diabetes Center, who is leading the human study at MGH, provides context: “This is the very first step in what is likely to be a long process in achieving a cure. We first need to determine whether the abnormal autoimmune cells that underlie type 1 diabetes can be knocked out with BCG vaccination, as occurred in the mouse studies.”
The Phase I trial is being supported largely through direct and fundraising support from the Iacocca Foundation, and through support from other donors and the Massachusetts General Hospital. The Iacocca Foundation was founded by Lee Iacocca and his family in 1984 to fund innovative approaches to a potential cure for diabetes. Trial information is available to the public at

About the Iacocca Foundation
The Iacocca Foundation has been a leader in the battle against diabetes for over 20 years. The foundation has granted more than $27 million to innovative and promising research designed to lead to a cure for diabetes and alleviate its complications. The Foundation was established by Lee Iacocca after his wife, Mary, died from complications of type 1 diabetes.

About the Massachusetts General Hospital
Founded in 1811, the MGH is the third oldest general hospital in the United States and the oldest and largest in New England. The 900-bed medical center offers sophisticated diagnostic and therapeutic care in virtually every specialty and subspecialty of medicine and surgery. Each year the MGH admits more than 46,000 inpatients and handles nearly 1.5 million outpatient visits at its main campus and health centers. Its Emergency Department records nearly 80,000 visits annually. The surgical staff performs more than 35,000 operations and the MGH Vincent Obstetrics Service delivers more than 3,500 babies each year. The MGH conducts the largest hospital-based research program in the country, with an annual research budget of approximately $500 million. It is the oldest and largest teaching hospital of Harvard Medical School, where nearly all MGH staff physicians serve on the faculty. The MGH is consistently ranked among the nation’s top hospitals by US News and World Report.

I am really excited about it…only time will tell now.

This is great news. Imagine a means to reverse Type 1 (and possible a low-cost one too)!!!

The trials are exciting and many people have been waiting for them to begin for some time now. I don’t mean to rain on the excitement, but after 32 years of hearing that the cure was right around the corner, try to be at least somewhat realistic about them. First, be aware that the mouse experiments for which Dr. Faustman’s results were done using the mouse experiments used Fruends complete adjuvant, while the human clinical trials will test using a vaccine called Bacillus Calmette-Guerin (better known by its acronym BCG). I should note that subsequent trials using mice dosed with BCG do suggest that it has the same impact, but mice are not human beings, and dozens of cures have worked on mice, but so far, none have been successful in humans. That’s why it is a trial!

Having said all of this, I would note that unlike most all other clinical trials, this one is one of the few which include longstanding type 1 diabetes patients included, therefore this is indeed a trial we should look forward to and wish success, but regardless of what happens, it will be several years before we have any results which can be analyzed (for bad or for good), so be patient!

While I always do love to read about new progress in the war against diabetes, I’m still a bit jaded after nearly 18 years of living with the disease. While mice and humans are indeed different but related creatures, the stretch from using different vaccinations to cure the illness in people seems a little bit iffy. So I would probably say that I’m cautiously optimistic (the same reaction I had when the Edmonton Protocol results were announced).

Science has eliminated smallpox from the world. Science has reduced the number of people with polio in a given year from several hundred thousand to perhaps a thousand. Science and industry have made significant improvements to the lives of those with type 1 diabetes. Research is providing data which brings us further everyday to better treatments for us and perhaps a cure and/or a means to prevent type 1…

Any progress is progress whether it leads to a closed door or a new horizon. We all know how hard it is to cope with diabetes so I work on the basis that we all know how great it would be to be diabetes free. I work everyday about the future and what condition my body will be in and my quality of life so if a cure comes Friday or 10 years from now we will be better off for it.
Lets All try to live in Hope.

Pretty exciting–very intriguing. I sent in my request to be involved in the trial. Hopefully they will take me as I turn 46 in 6 months and will be too old to participate. Ugh!


Hope. Just a bit of it.
I can’t participate in the trial since I don’t live in Boston but it was nice to have the trial coordinators respond so quickly. I’m on file for future expansions.

this is awsome news. I have been following Dr Faustmans news for a long time now. Stay healthy folks because the next couple of years are going to be a wild ride. If this works lots of folks will be cured. The ones remaing will be actually fully non repetativly cureable with a working stem cell treatment.

The questions we will be asking ourselves then will be “what do I do now?” I look forward to life being easier. My freinds will be looking forward to me someday forgetting how many carbs are in a bottle of Belgian Ale. Back when I was a kid and dreaming of a cure I always had an image of myself going out for a root beer float once all was well again. These days I see myself just going to bed without checking my sugars first, then walking up late the next day and not running downstairs to do it again and curseing myself for running dry.



I was this close to living in Boston. I am changing Jobs and one of the potential places was looking for someone to do contract work there… It was looking good, but they company just decided to cancel all new positions. 8^(


This is the most promising yet! In case anyone is interested…the Faustman research gets some grants from the JDRF…who gets funded from the drug companies. Obviously drug companies don’t want a cure, they would lose a TON on money…to say the least. Rumor has it that the JDRF is getting pressure from these companies to stop funding her. Give to The Iacocca Foundation!!!

Penny you are not alone!

I registered my 18 mo old son with the lab, I want him at the forefront of this research. I am confidant that the procedure will be approved and who knows maybe he can be included in stage II or III.

Have you noticed how the Juv. Diabetes Foundation website doesn’t even mention the research, the clinical trial, nothing… but it did anounce a partnership with drug manufacturers (?) I do wonder why the insurance industry does not support this research yet, it would be smart business and amazing press.

OK, I’m off my soap box…

One can hope. A cure one can dream, and dreams come true.

Today’s New England Journal of Medicine has released early an editorial by Dr. Faustman and preliminary research on GAD treatment for newly-diagnosed T1.

Immunotherapy on Trial for New-Onset Type 1 Diabetes

GAD Treatment and Insulin Secretion in Recent-Onset Type 1 Diabetes

An interpretation of this information can be found in the following HealthDay news article, picked up by Health Central: New Vaccine May Help Type 1 Diabetics in Future

I am excited about it to an extent i have heard for 28 years the cure will be here soon. I am still hopeful. If this is great but im sure it will take years to get to us patients.

Don’t confuse this editorial of Dr. Faustman’s with reports about her own research.

Also, from what I saw reported about the GAD vaccine study (which is what is under discussion here) long term, it did not improve the children’s ability to produce insulin. Only short term.

I think I might have. Thanks for the clarification.

The results of the GAD vaccine study were that the vaccine slowed the rate of beta-cell decay, but only if it was given within 6 months of initial diagnosis, and the patients/subjects have only been followed for about 18 months. I don’t think the researchers were trying to reverse the damage already present. The operative theory of this research is that if beta-cell decay can be slowed or arrested, then complications related to complete beta-cell destruction could be minimized, delayed, or prevented.