From the New York Times:
Novartis, the Swiss pharmaceutical company, disclosed in an interview this week that an experimental drug has brought about substantial improvements in the behaviors of people with Fragile X syndrome in a small clinical trial.
The research involved only a few dozen subjects and has not been published or peer reviewed.
The company refused to reveal many details, citing commercial interests, but a Novartis official cautioned against too much optimism. Dr. Mark C. Fishman, president of the Novartis Institutes for BioMedical Research, said the drug is likely to be years from commercial development and could fail further clinical trials.
If authenticated in further, larger trials, the results could also become a landmark in the field of autism research, since scientists speculated that the drug may help some patients with autism not caused by fragile X, perhaps becoming the first medicine to address autism’s core symptoms.
… “This is perhaps the most promising therapeutic discovery ever for a gene-based behavioral disease,” said Dr. Edward M. Scolnick, former research chief at Merck and now director of the Stanley Center for Psychiatric Research at the Broad Institute at Harvard and the Massachusetts Institute of Technology.