With a high-profile clinical trial recently finished, the drug company Novartis took an unorthodox approach to releasing the results: it divulged them—or at least, a rough summary—in an interview with The New York Times.
The trial, launched in November, was unusual because it was testing a drug for fragile X syndrome, the most common inherited cause of mental retardation. Some considered it unlikely that correcting malfunctioning brain circuits in this way could work, although a handful of researchers have been experimenting with drug treatments in animal modeals. In patients, the drug did make a difference, said Mark Fishman, president of the Novartis Institutes for BioMedical Research in Cambridge, Massachusetts. "Our group feels pretty good about the data," he told Times reporter Gardiner Harris, cautioning that the results haven't been peer reviewed or published. In theory, the therapy might also help in autism because it's designed to counter symptoms in that disease.
Novartis officials declined to comment about why they shared news of the trial this way, or provide any additional information about how participants fared. Fishman noted in the Times that not everyone was helped by the treatment; the phase I trial reportedly included a few dozen patients, and the drug, if it pans out, is still years away from being approved.