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Doctors find promise in drug Suramin to treat autism

UC San Diego Health System News May 08, 2018

The CDC estimates that 1 in 68 children in the US are on the autism spectrum. Right now, there are no FDA-approved treatments for the disorder or its symptoms, which can include difficulty with social skills, communication, and repetitive behavior. That’s why results of a small clinical trial in San Diego has grabbed the attention of researchers and families.

Dr. Robert Naviaux suspected the cause of autism might be metabolic dysfunction, where the energy molecule ATP is “outside” cells. He researched more than 2,000 drugs and found one that might help. That drug was Suramin. Dr. Naviaux tested one dose in a clinical trial of 10 boys. Five got the drug.

“Children began to talk sometimes for the first time in sentences in their life,” says Dr. Naviaux.

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.

“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,” says Miles.

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 8 weeks.

“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,” says Dr. Naviaux.

Miles and his mom are encouraged by the results.

Dr. Naviaux says next there will be several phase 2 trials to determine safety and efficacy for Suramin. He suspects it will be 3 to 5 years before phase 3 trials begin.

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