Treatment for autism looked tantalizingly close just five years ago, as the first drug studies launched for the disorder Fragile X. Advocates hoped that treatments for the rest of the autism spectrum would soon follow.
Today, that optimism is gone, as drug after promising drug has failed.
Novartis announced last week that two drug trials showed an experimental medication did not improve the conditions of adults and teens with Fragile X, a genetic disorder that can lead to intellectual disability, anxiety, speech delay, seizures, and social challenges. Other trials run by Roche and startup Seaside Therapeutics also failed to show benefits.
“The patient community had been sold on the hope that comes with these clinical trials,” said Robert Ring, the outgoing chief scientific officer of Autism Speaks, an advocacy and research group. “I think we can do a lot better job of spelling out the realistic context in which these things are happening.”
Ring, who formerly headed the autism research unit at drug giant Pfizer, said the results may discourage venture capitalists from pursuing drug development in the field. But he said big drug companies will likely stick with it.
It’ll just take a lot longer, and a lot more work, than advocates and scientists had hoped.