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Hormone-based therapies show promise in children and adults with autism

Published on 02/05/19 at 10:14am

A hormone-based therapy might improve social function in people with autism, according to the results of two separate clinical trials.

Treatments based on the hormone vasopressin have shown promise in improving social function in both children and adults with autism, in clinical trials of vasopressin-based therapies.

The first trial tested the effectiveness of a vasopressin nasal spray on children with autism spectrum disorder.

“The parents saw improvement, the clinicians saw improvement, and the children's performance on lab tests also improved with vasopressin compared to placebo” Karen Parker, director of social neuroscience research at Stanford University, who led the study, said.

In contrast the second trial tested, balovaptan, a therapy that activates brain receptors targeted by the hormone, on adults with autism.

Adult men with autism spectrum disorder, who took balovaptan, experienced clinically meaningful improvements in their social behaviours.

In a clinical trial of 223 men with moderate or severe autism, balovaptan was found “to improve the core characteristics of social interaction and communications in adults with ASD,” Dr Paulo Fontoura, senior vice president of neuroscience and rare diseases clinical development at Roche Pharmaceuticals, who led the research, said in a statement.

In explaining the findings, Parker said: “We showed that vasopressin was low in cerebrospinal fluid [among people with autism], and the lower your vasopressin levels, the greater your symptom severity. If we look at where the vasopressin receptors are in the brain, they're in socially relevant areas that have been identified as regulating social functioning.”

Both studies have now proceeded to the next stage of clinical trials. Vasopressin nasal spray is now being tested in a trial of 100 children while balovaptan will be the focus of a Phase 3 trial in adults and a separate Phase 2 trial in children and teenagers.

Louis Goss

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