In Depth January 14, 2010, 5:00PM EST

The Hunt for an Autism Drug

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Weakley hopes for a drug that will "unlock" her daughter's potential Jeff Hutchens/Reportage by Getty Images

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The company has zeroed in on a rare brain disorder called Fragile X, caused by a mutation on the X chromosome and known to cause a hereditary form of autism. Founded in 2005 by former Massachusetts Institute of Technology neuroscientist Mark F. Bear, Seaside has two drug candidates in clinical trials. Like Jennifer Weakley's drug, Namenda, these compounds zero in on the brain's glutamate system, involved in learning and memory.

"What you need is a company to be successful—to show that the FDA will approve you, and that you can make money—and everybody will jump in," says Seaside CEO Dr. Randy Carpenter. "If [a drug] shows a benefit, we can charge a premium."

Some experimental treatments focus on the social-bonding aspects of the disorder. Oxytocin, which is given to pregnant women to induce labor under the brand name Pitocin, has earned the nickname the "love chemical," because studies in healthy adults have shown that sniffing it results in the formation of strong bonds of trust. Studying the effects of the drug on patients with Asperger's, a syndrome marked by social awkwardness that is on the less severe end of the autism spectrum, psychiatrist Hollander found a reduction in repetitive behaviors and an improved ability to recognize the emotional tone of sentences. Other drugs target the brain's interaction with organs that aren't usually associated with autism, including those of the gastrointestinal tract and the immune system.

One of the most promising treatments in this category is a drug called CM-AT made by a startup called Curemark. Dr. Joan Fallon, the company's founder and CEO, observed that many autistics show a strong preference for foods high in carbohydrates and low in protein. A diagnostic test revealed that some autistic children lack enzymes that digest protein. As a result, these children produce fewer of the essential amino acids that are the building blocks for brain development and neuroreception. Fallon believes this deficiency is linked to the most severe symptoms of autism, and she says an early observational study of CM-AT, an orally ingested powder that delivers protein-digesting protease, showed "significant improvements." Curemark is enrolling patients in phase III clinical trials at 10 to 12 sites—the largest autism trial to date.

Beverly Weakley is counting on Namenda—or some yet-to-be-formulated treatment—to "unlock" Jennifer's potential. She describes how one day, many years ago, she heard Jennifer call out from another room "Mommy, I need you." It was as though a window to her daughter had opened up. She raced to her daughter's side, but by the time she got there, Jen was "gone." Since then, the long silence has been almost unbearable, but Beverly has learned not to dwell on setbacks. She keeps hoping, she says, that the window will reopen.