The National Institute of Mental Health’s $9-million bet on aggressive autism drug development reflects the dearth of treatment options for the disorder. Will this ‘fast-fail’ approach pay off?

A $9 million grant to the University of California, Los Angeles aims to drive clinical trials for autism that would quickly rule out ineffective compounds.

Two weeks of treatment with a cancer drug called topotecan boosts expression for a year of the gene that’s deficient in Angelman syndrome, according to unpublished mouse research presented 20 March at the New York Academy of Sciences.

Researchers have added two new candidates to the arsenal of compounds that alleviate both the behavioral and molecular hallmarks of fragile X syndrome in mice that model the disorder. A third candidate, minocycline, improves some symptoms in children with the disorder.

A handful of studies point to dietary deficiencies as a contributing factor in some forms of autism, suggesting that supplements — such as carnitine or certain amino acids — may help treat and even prevent the disorder.

Bumetanide, a drug that’s long been used to treat high blood pressure, modestly improves social behaviors in children with mild forms of autism, according to the results of a small trial published in December in Translational Psychiatry.

Two healthcare organizations have pooled their data to create a registry of 20,000 people with autism, a resource that may help speed up clinical trials and other research.

Early intensive therapy may normalize the brain’s response to faces in young children with autism, according to a study published in the November issue of the Journal of the American Academy of Child and Adolescent Psychiatry. The results are part of a randomized, controlled trial of a treatment called the Early Start Denver Model.

Rodent and stem cell models remain challenging for developing psychiatric drugs, says Michael Ehlers, chief scientific officer of neuroscience at Pfizer.

New standards for animal studies, including an emphasis on replicating results and the publication of negative findings, are vital for research progress, says Jacqueline Crawley.