100-year-old drug gets new use treating symptoms of autism

HOUSTON – The Centers for Disease Control and Prevention estimates that 1 in 68 children in the U.S. 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 have grabbed the attention of researchers and families. 

Dr. Robert K. Naviaux, a professor of genetics at the Departments of Medicine and co-director of the Mitochondrial and Metabolic Disease Center at UC San Diego School of Medicine, 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. Naviaux tested one dose of Suramin 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,” Naviaux said.

Boys who got Suramin had autism severity scores drop from 8.6 to seven, 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 and 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,” McInerney said.

He now uses rowing to reduce the stress related to his autism. For those who did get the drug, Naviaux said  most, but not all the effects wore off in eight 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 motor skills were motor memory that had been retained.” Naviaux said.

McInerney and his mom said they are encouraged by the results.

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.