WEDNESDAY, Feb. 1 (HealthDay News) -- The latest trial of a drug
called GAD-alum to treat type 1 diabetes failed to show any
significant improvement in the common markers of the blood sugar
The researchers, however, said they felt there were possible
reasons for the failure of the current trial, and that specific
groups of people still might benefit from GAD-alum.
"Even if this study failed to reach its primary outcome, there were quite impressive results in certain subgroups, which supports the concept of using auto-antigens in the therapy of recent-onset type 1 diabetes," said study author Dr. Johnny Ludvigsson, a professor in the division of pediatrics, in the department of clinical and experimental medicine at Linkoping University in Sweden.
Results of the study are published in the Feb. 2 issue of the
New England Journal of Medicine. Funding for the study was provided by Diamyd Medical (the drug's maker) and the Swedish Child Diabetes Foundation.
Type 1 diabetes is an autoimmune disease, in which the body's
immune system mistakenly attacks healthy cells. In the case of type
1 diabetes, the immune system attacks the insulin-producing beta
cells in the pancreas. When enough beta cells have been destroyed,
people with type 1 diabetes no longer produce enough insulin to
properly metabolize the carbohydrates in foods. Carbohydrates
become glucose in the body, which is used to fuel the body's cells.
If people with type 1 diabetes don't take insulin (through
injections or an insulin pump), their body's cells can't use the
glucose and blood sugar levels rise to dangerous levels.
There are several autoantibodies (also called autoantigens) that
can cause the destruction of beta cells in type 1 diabetes. One
such autoantibody is called GAD65. Not everyone with type 1
diabetes has GAD65 autoantibodies, but many do. Dr. Jay Skyler,
deputy director of clinical research at the University of Miami's
Diabetes Research Institute, said that about 80 percent of people
with type 1 likely have GAD65 autoantibodies.
GAD-alum was designed to work like a vaccine, hopefully stopping
the autoimmune attack soon after it has begun, to preserve beta
"GAD-alum takes a vaccine kind of approach, and takes the antigen in the autoimmune disease and tries to give it in a way that will vaccinate people so they don't have the destructive autoimmunity," explained Skyler.
Previous research, done by the same group, showed positive
effects from the treatment, especially in those treated less than
six months after being diagnosed with type 1 diabetes, according to
The current study included 334 people between the ages of 10 and
20. All had type 1 diabetes for less than three months, and they
had GAD autoantibodies.
The patients were randomly assigned to one of three treatment
groups: four doses of GAD-alum; two doses of GAD-alum followed by
two doses of placebo; or four doses of placebo.
The most important measure the researchers looked for was levels
of C-peptide. C-peptide is a byproduct of insulin production, so
rising levels of C-peptide indicate increased levels of insulin.
The researchers also measured how much insulin people needed, their
average blood sugar levels over the study period, and how often
they experienced low blood sugar levels.
While GAD-alum appeared safe and was very well tolerated in this
study, it didn't significantly reduce levels of C-peptide, improve
blood sugar levels or lessen the need for insulin, according to the
Ludvigsson said there may be several reasons that the initial
research looked promising, yet this study failed to deliver
positive results. One is that they allowed flu vaccines to be given
at the same time this trial was ongoing, which could have affected
the immune system response somehow, he said. Also, the age of the
patients may alter the results of the trial because younger
patients tend to lose beta cells faster, he noted.
Skyler said the first study showed promise only in a small
subgroup of patients, not in the entire group. "You can't draw firm
conclusions from pilot studies. There are a lot of things that look
quite promising that don't pan out," he said.
But, Skyler added, "I personally don't think we should abandon
GAD; I think we should think about the form it's in, and the route
Ludvigsson said further trials of GAD-alum are already on the
way, using the drug as an intervention for people recently
diagnosed with type 1 diabetes and as a preventive measure for
people who have a high risk of developing type 1 diabetes.
Learn more about type 1 diabetes from the
American Diabetes Association.
Please be aware that this information is provided to supplement the care provided by your physician. It is neither intended nor implied to be a substitute for professional medical advice. CALL YOUR HEALTHCARE PROVIDER IMMEDIATELY IF YOU THINK YOU MAY HAVE A MEDICAL EMERGENCY. Always seek the advice of your physician or other qualified health provider prior to starting any new treatment or with any questions you may have regarding a medical condition.
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