WEDNESDAY, May 1 (HealthDay News) -- An experimental drug for
autism did not improve levels of lethargy and social withdrawal in
children who took it, but it did show some other benefits, a new
Children on arbaclofen did improve on an overall measure of
autism severity when compared to kids taking an inactive placebo,
said lead researcher Dr. Jeremy Veenstra-VanderWeele, an associate
professor of psychiatry, pediatrics and pharmacology at Vanderbilt
He is to present the findings Thursday at the International
Meeting for Autism Research (IMFAR) in Spain.
One of 88 children in the United States is now diagnosed with an
autism spectrum disorder, the umbrella term for complex brain
development disorders marked by problems in social interaction and
Veenstra-VanderWeele focused on evaluating the social
improvement with the drug because earlier research had suggested it
could help. However, one of the earlier studies did not compare the
drug to a placebo, but simply measured improvement in those who
took the drug.
In the new study, Veenstra-VanderWeele and his team assigned 150
people with autism, aged 5 to 21, to take the medicine or a
placebo, without knowing which group they were in, for eight weeks.
The participants had been diagnosed with autistic disorder,
Asperger's syndrome or another related condition known as pervasive
In all, 130 finished the study. When no differences were found
in social withdrawal or lethargy between the two groups, the
researchers looked at a scale that measures severity and
improvement of autism with treatment.
Those on the drug improved more on that scale. A child, for
instance, who began the study evaluated as having marked severity
might be described as moderate by the study's end,
"This is the sort of improvement that would motivate us to start a medicine," he said.
The drug is believed to work, Veenstra-VanderWeele said, by
increasing inhibition, improving social functioning and
Right now, Veenstra-VanderWeele said, "there is no medication
that has clear evidence to improve social function in autism."
Those on the drug did report side effects, including suicidal
thoughts reported by one patient on the drug and one on the
placebo. Some patients on the drug became upset more easily; others
The next phase of trials of the drug are in the planning stages,
But more research is needed, said Dr. Andrew Adesman, chief of
developmental and behavioral pediatrics at the Steven and Alexandra
Cohen Children's Medical Center of New York.
Even though the expected benefit did not materialize, Adesman
sees a reason to continue to study the medication. "There is
[still] some suggestion of benefit from the medicine," Adesman
said. "It just didn't quite show up where they expected."
The drug may offer benefit to some children with autism, Adesman
said. "But it's unclear which children may be the best
The trial received funding from the drug's maker, Seaside
Therapeutics. The medication is not currently approved by the U.S.
Food and Drug Administration.
The data and conclusions of research presented at medical
meetings should be viewed as preliminary until published in a
To learn more about
autism, visit the U.S. Centers for Disease Control and Prevention.
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.
Copyright © EBSCO Publishing. All rights reserved.