Is Arbaclofen an Effective Treatment for Autism?

Although not associated with improvement on the study's primary endpoint, arbaclofen improved social skills and global severity in young people with autism spectrum disorder.

Autism spectrum disorder (ASD) frequently occurs in fragile X syndrome, which is associated with increased glutamate-pathway activity in animals (NEJM JW Psychiatry May 2008 and Neuron 2007; 56:955). In a previous study of fragile X patients, investigators examined the effect of lowering hyperexcitability with the gamma-aminobutyric acid B agonist arbaclofen, an experimental drug (NEJM JW Psychiatry Nov 2012 and Sci Transl Med 2012: 4:152). Although arbaclofen did not affect fragile X features, it was associated with improvements in social avoidance, a common symptom of ASD.

To examine the effect of arbaclofen in ASD patients without fragile X, investigators conducted a randomized, double-blind, placebo-controlled trial at 25 sites. The 150 …

Reviewing Author

Barbara Geller, MD

Disclosures

Nothing to disclose

Barbara Geller, MD

Disclosures

Nothing to disclose

Is Arbaclofen an Effective Treatment for Autism?

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Reviewing Author