Secret funding fosters hope for new drugs for autism

C&EN Senior Editor Lisa Jarvis notes that until recent interest from big pharmaceutical companies, a small drug company in Cambridge, Mass. named Seaside Therapeutics was virtually the only company trying to develop drugs for autism and fragile X syndrome.

Diagnoses of autism and related conditions termed autism spectrum disorders have increased dramatically since the 1980s, for reasons not yet fully clear. They affect millions of people worldwide. Fragile X syndrome, the most common known genetic cause of autism, results from mutation in a single gene. Its symptoms range from learning impairment to mental retardation. The disease affects about 1 in 4,000 males and 1 in 6,000 to 8,000 females.

The article describes how Seaside, armed with funding from an anonymous wealthy family and new insights into the basic science behind these disorders, is making progress toward treating these much-neglected diseases. Two of the company's potential drugs show promise in clinical trials as treatments for Fragile X syndrome. One appears to improve the behavior of children with severe social impairments. On the heels of Seaside's encouraging results, big pharmaceutical companies that once showed little interest in tackling these diseases are now trying to develop their own new medications.

ARTICLE FOR IMMEDIATE RELEASE “Tackling Fragile X”

This story is available at http://pubs.acs.org/cen/business/88/8837bus3.html