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A new crop of drugs aim, for the first time, at the core symptoms of this disorder
By Alla Katsnelson
Until now, attempts at treating autism have been limited to drugs that target peripheral symptoms such as anxiety, aggression and repetitive behaviors. But researchers hope that data from a crop of new drugs in development will allow them, for the first time, to treat an underlying mechanism of the condition, potentially helping those with autism to communicate.
The majority of autism cases are idiopathic, meaning that researchers have yet to understand their cause. But some animal studies of autism have pointed to signaling problems in the brain. Targeting those signaling problems, some researchers think, may ameliorate autism symptoms once thought to be intractable. Researchers have gleaned some of this information by studying a handful of diseases caused by single-gene glitches that can result in autism. Such disorders account for about 15 to 20 percent of autism cases, says Geraldine Dawson, scientific director of Autism Speaks. In fragile X, which causes autism in a significant number of cases, the points of contact between neurons contain too much glutamate, a chemical messenger that transmits excitatory signals.
Still, big questions remain. Thus far, researchers have had little success designing drugs that target glutamate without side effects. And should the drugs work, researchers will still need to determine at what age they would be most beneficial, because autism begins early in development. But the results of Seaside's trials and those of similar drugs in the pipeline, Dawson says, “are going to be a huge step to understanding what the path to discovery is going to be.”
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