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Repurposed Drug Improves ASD Symptoms in Children

By Mark N. Ziats on January 9, 2013


Background: Previous research has indicated that brain signaling using a neurotransmitter known as GABA could be disrupted in children with Autism Spectrum Disorders (ASD).  Brain cells use the molecule chloride to modulate the levels of GABA within the brain, and many drugs used for other diseases work by modulating chloride levels.  A small pilot study previously suggested drugs which modulate chloride levels in the brain may be beneficial to children with autism.


What’s new: In a study recently published in the journal Translational Psychiatry, researchers tested children with autism or Asperger sundrome using the chloride-altering drug bumetanide, which is known to be safe in children when used for other indications.


The study tested the effect of bumetanide in a double-blinded, placebo-controlled trial of sixty children (ages 3-11) with ASD.  The children received either bumetanide or the placebo for 3 months, and were tested for improvement on three separate behavioral rating scales.  The results demonstrated that children on bumetanide were significantly more likely to experience improvements in their symptom severity—as determined by improvements in rating scale scores—than autistic children who received the placebo.  Importantly, the drug was well-tolerated and caused minimal side effects in all children who received it over the course of the 3 months.


Why it’s important: This study provides evidence that adjusting GABA levels in the brain by modulating chloride levels improves the behavioral symptoms of autism.  Because many drugs that act on chloride levels are already known to be safe in children, it is hoped that larger studies can be pursued in order to bring novel treatments targeting this pathways to the clinic.

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