New drug that may help reverse autism is to be tested on children for the first time after successful clinical trials on mice
Drug called suramin is already used to treat sleeping sickness in AfricaScientists in the U.S. have found it corrects autism-like symptoms in miceA small clinical trial on children with the condition will be started this year
06:48 GMT, 14 March 2013
09:29 GMT, 14 March 2013
A drug that may reverse autism is to be tested on children with the condition for the first time, scientists have revealed.
Preliminary results show that the drug called suramin, which is already used to treat sleeping sickness in Africa, 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.
Groundbreaking: Testing of the drug suramin on mice in America has found it can help to correct autism-like symptoms (stock image)
The drug 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 U.S. 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.'
New hope: A small clinical trial on children could be carried out within the year at the Mitochondrial and Metabolic Disease Centre at the University of California in San Diego, pictured
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.
Professor Naviaux and his team believe both genetic and environmental causes of autism can be traced to a sustained cell danger response linked to immunity and inflammation.
'When cells are exposed to classical forms of dangers such as a virus, infection or toxic environmental substance, a defence mechanism is activated,' said the professor.
'This results in changes to metabolism and gene expression (activity) and reduces the communication between neighbouring cells. Simply put, when cells stop talking to each other, children stop talking.'
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 Professor Naviaux.