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New sleeping sickness drug may help to cure autism

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.

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.

Scientists in the US found that the drug corrected 17 types of abnormality linked to autism in genetically modified mices.

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.

Professor Robert Naviaux, co-director of the Mitochondrial and Metabolic Disease Centre at the University of California at 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 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.

"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.

Suramin is an inhibitor of purinergic signalling that has been used to treat African sleeping sickness since shortly after it was first synthesised in 1916.

The "striking effectiveness" of the drug in mice could pave the way to a "completely new class of anti-inflammatory drugs to treat autism and other disorders", said Prof Naviaux.