Genetic discoveries have invigorated autism research and raised the possibility of finding drug targets based on autism’s underlying pathophysiology, rather than merely treating symptoms.
An experimental group of drugs works wonders in a mouse-model of fragile X syndrome, the most common single-gene cause of autism. The drugs, which calm overactive metabotropic glutamate receptors (mGluRs) in the brain, restore social sniffing behaviour to normal levels, boost learning and memory, and reduce seizures related to the syndrome. Yet despite being the most promising new strategy for treating autism, these drugs are floundering once they reach human subjects.
The obscurity of the causes of autism means that doctors have little to offer the one in 100 people affected worldwide. Intensive behavioural therapies help some, but current medicines are limited to two drugs, risperidone and aripiprazole, used to treat aggression and irritability.
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| An electroencephalogram measures brain connectivity, which, when combined with other measures, may help delineate people with autism into subgroups with their own distinct biology |
Source: SciencePhotoLibrary
