A drug that may reverse autism is to be tested on children with the condition, scientists have revealed.
Preliminary results show that the drug, already used to treat sleeping sickness, corrects autism-like symptoms in mice. At the molecular level, it normalises faulty brain connections, cell-to-cell signalling, and metabolic effects thought to underlie the disorder.
The drug, suramin, targets a cell messaging system that produces a metabolic response to stress. According to a new theory, autism is strongly linked to this pathway, known as purinergic signalling.
Scientists in the US found that the drug corrected 17 types of abnormality linked to autism in genetically modified mice, including social behaviour problems.
Autism is a wide ranging condition, mostly seen in boys, that affects a person's ability to socialise and communicate and can have a devastating lifelong impact. Around 600,000 children and adults in the UK are thought to have the disorder.
Professor Robert Naviaux, co-director of the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, said: "Our theory suggests that autism happens because cells get stuck in a defensive metabolic mode and fail to talk to each other normally, which can interfere with brain development and function.
"We used a class of drugs that has been around for almost a century to treat other diseases to block the 'danger' signal in a mouse model, allowing cells to return to normal metabolism and restore cell communication."
He added: "Of course, correcting abnormalities in a mouse is a long way from a cure for humans. But we are encouraged enough to test this approach in a small clinical trial of children with autism spectrum disorder in the coming year.
"This trial is still in the early stages of development. We think this approach, called antipurinergic therapy or APT, offers a fresh and exciting new path that could lead to development of a new class of drugs to treat autism."
The findings are published in the online journal Public Library of Science ONE.
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New drug hope for autism treatment