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MGH initiates phase I diabetes trial
BOSTON - March 13, 2008 - 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
led 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." Trial information
is available to the public at www.faustmanlab.org.
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.
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.
Media Contacts: Emily
Parker, MGH Public Affairs
Physician Referral Service: 1-800-388-4644
Information about Clinical Trials
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