Drug Trials May Point the Way to Treating Autism Spectrum Disorders

An article in MIT's Technology Review announces a set of clinical trials that may have a significant impact on treating Fragile X, Rett Syndrome and Tuberous Sclerosis, all inherited genetically-based disorders associated with autism. While these three diagnoses account for a relatively small number of people diagnosed with autism spectrum disorders (ASDs):

We may have our finger on a biochemical pathway that is applicable more generally in autism," said Mark Bear, a neuroscientist at MIT, at the Autism Consortium annual symposium in Boston last month.

...While initial trials will focus on these three specific diseases, Bear and others hope that the treatments will prove more broadly applicable. "The hope is that we'll uncover pathways that are involved in cases of autism with unknown etiology," said Bear. Even though there may be hundreds of genes affected in different cases of autism, he said, "I strongly suspect that there will be a few key pathways, so drugs that regulate that pathway may have a broad impact."

The specific treatments being tested for Rett Syndrome include two growth hormones already approved by the FDA for use with children of small stature. The treatment being tested for Fragile X has been tested successfully in mouse models, but has yet to be approved by the FDA.