Hopes Dim as Clinical Trials Fail for Biomedical Intervention for Autism

This news story was first published in May 2017 and reports on drug trials from around that time.

The first drug studies in recent years on autism medication treatment focused on Fragile X syndrome, a genetic condition that often occurs alongside autism. These studies included older children and adults rather than young children, because of concerns about unknown side effects of new drugs. Advocates hoped that what was learned could then be applied more widely across the autism spectrum. That optimism faded, as several promising drugs did not deliver.

Recent Trials Show Little Promise:

Novartis announced that two drug trials had not produced the hoped-for results. The experimental medication did not improve the effects of Fragile X. This genetic condition is often associated with intellectual disability, ongoing anxiety, speech delay and seizures.

Social differences, one of the key characteristics of Autism, are also common in Fragile X. Trials run by Roche and, more recently, Seaside Therapeutics had similar outcomes. Neither showed clear benefits, and the adult and teenage participants showed little change.

Note: These negative trials were still useful to the US Food and Drug Administration, helping it better understand how drugs for neurological conditions should be tested.

New Options Going Forward

Robert Ring, then Chief Science Officer at Autism Speaks, stressed the importance of setting realistic expectations about these results. Ring previously led autism research at Pfizer. He suggested that the results were unlikely to encourage investors to fund drug development for autism.

Dr Shafali Jeste, a behavioral child neurologist at the University of California, Los Angeles (UCLA) David Geffen School of Medicine, pointed to several possible reasons for the failed trials. In an opinion piece she co-wrote, published alongside the study, she suggested that mavoglurant, the drug tested, may have been the wrong drug for the purpose, that a different dose or trial length might have shown benefits, or that the drug’s effects in mice simply do not carry over to humans.

Note: Both autism and Fragile X have subjective symptoms that can vary from day to day, so small changes are hard to measure.

What This Means for Families

There is no medication for autism itself, and autism is not something that needs to be cured. Research into medication continues, mainly to help with specific difficulties such as anxiety, sleep problems or seizures that can occur alongside autism or Fragile X. In the meantime, the supports with the strongest evidence focus on communication, understanding a child’s sensory needs, and adjusting the environment at home and school.