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expert reaction to two clinical trials looking at drugs modulating the hormone vasopressin’s biological pathway in people with autism spectrum disorder

Research published in Science Translational Medicine demonstrates that modulating the hormone vasopressin’s biological pathway enhanced social functioning in adult men and children with autism spectrum disorder.

Dr James Cusack, Director of Science, Autistica, said:

“It’s early days, but it may be possible that vasopressin-based approaches can help some autistic people.  It’s important to remember that in the larger trial the researchers didn’t find this drug was effective on the main measure they chose, so another trial is needed to establish how valid this finding is.  We need more research to understand whether this treatment makes a difference to daily living skills and who benefits most.

“Drug-based treatments are controversial in autism and many autistic people and families are opposed to them and would prefer society to change instead.  Equally other people do want more drug treatments like this and so we need options to accommodate everyone.”

Paper 1: ‘A phase 2 clinical trial of a vasopressin V1a receptor antagonist shows improved adaptive behaviors in men with autism spectrum disorder’ by Federico Bolognani et al. was published in Science Translational Medicine at 19:00 UK time on Wednesday 1 May 2019.

Paper 2: ‘A randomized placebo-controlled pilot trial shows that intranasal vasopressin improves social deficits in children with autism’ by Karen J. Parker et al. was published in Science Translational Medicine at 19:00 UK time on Wednesday 1 May 2019.

Declared interests

Dr James Cusack: “Autistica is engaged in a large-scale European research project called AIMS-2-TRIALS which also includes Roche.”

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