TUESDAY, Dec. 11 (HealthDay News) -- A medication typically
prescribed to control high blood pressure that's commonly referred
to as a water pill may ease some of the symptoms of autism,
researchers say.
That's especially true for people who have milder forms of the
disorder, the new research indicates.
"Bumetanide is a promising novel therapeutic agent to treat autism," wrote the study's authors, who were quick to point out that this treatment is not a cure for autism and that larger trials need to be done to determine who would benefit most from this treatment.
Results of the study appear online Dec. 11 in the journal
Translational Psychiatry.
Autism is a developmental disorder that results in communication
problems, social difficulties, repetitive behaviors and restricted
interests, according to background information in the study. The
exact cause of the disorder isn't yet known, though genetic and
environmental factors are believed to play a role.
Scientists have also studied neurotransmitters, suspecting that
these chemical messengers in the brain might not be working as they
should in people with autism. Gamma-aminobutyric acid (GABA) is one
such neurotransmitter, and the medication bumetanide appears to be
able to alter the function of GABA, at least in animals, according
to the researchers.
In this latest study, the researchers gave 60 children who had
been diagnosed with autism or Asperger's syndrome either 1
milligram of bumetanide or an inactive placebo pill daily. All of
the children were between 3 and 11, and the study period lasted
three months.
The severity of autism was determined by videotaped evaluations
done on the first day of the study and the 90th day, by researchers
who didn't know which children had received active treatment and
which had been given a placebo pill.
Using several standardized autism assessment tools, the
researchers found that bumetanide improved autism symptoms. The
effects of the medication were more pronounced in children with
milder autism. In case reports included in the study, some of the
improvements seen included more eye contact, improved non-verbal
communication and better social communication.
Side effects were very mild, though the use of the drug does
require periodic monitoring for potassium levels; one child in the
study had low potassium levels.
Senior study author Yehezkel Ben-Ari, founder of the Institute
of Neurobiology of the Mediterranean Sea in Bohars, France, said
the authors believe that the improvement in symptoms is due to an
effect on GABA caused by the drug.
One expert said the findings look promising.
"This is a very interesting study. The authors have taken a medication that's been on the back shelf for decades and, surprisingly, found a clinically significant benefit when used in children with autism, especially those on the milder end of the spectrum," noted Dr. Andrew Adesman, chief of developmental and behavioral pediatrics at the Steven and Alexandra Cohen Children's Medical Center of New York in New Hyde Park, N.Y.
"Children would need to be on this medication indefinitely, because their improvements deteriorate when the medication is stopped. And, we don't yet know if there are any long-term concerns," he said. "Hopefully, this study will be a catalyst to new research. We need to see additional studies before we can recommend this treatment widely, but since it's already FDA-approved, we should know more in just a few years."
More information
To learn more about
autism, visit the U.S. Department of Health and Human Services.