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New trial for autism drug

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A 102-year-old drug used to treat African sleeping sickness is getting new use treating symptoms of autism.

Dr. Robert Naviaux suspected the cause of autism might be metabolic dysfunction, where the energy molecule ATP is "outside" cells. He researched more than two thousand drugs and found one that might help.

That drug was Suramin. Dr. Naviaux tested one dose in a clinical trial of ten boys. Five got the drug.

Dr. Naviaux, Professor of Genetics, UC San Diego School of Medicine says, "Children began to talk sometimes for the first time in sentences in their life." 

Boys who got Suramin had autism severity scores drop from 8.6 to 7, the lowest point on the spectrum. They improved social, language and fine motor skills, and found relief from repetitive motions and fragmented sleep.

Miles McInerney was in the trial but did not receive Suramin; he still wanted to help.

He explains, "I should generally be interested in the ability to possibly find a way that people with worse autism or struggle more with autism than I do, to possibly be able to find a solution that can help them better communicate."

He now uses rowing to reduce the stress related to his autism. For those who did get the drug, Dr. Naviaux says most but not all the effects wore off in eight weeks.

Dr. Naviaux continues, "Some children had learned to tie their shoes for the first time, and other children had learned to zip up a jacket. Those fine motors skills were motor memory that had been retained." 

Miles and his mom are encouraged by the results. Dr. Naviaux says next there will be several phase two trials to determine safety and efficacy for Suramin. He suspects it will be three to five years before phase three trials begin.


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