YOUR HEALTH: Could an old drug be a new treatment for autism?

SAN DIEGO, California – It's a medical condition with a lot of mystery attached to it.

Right now, there are no FDA-approved treatments for the autism disorder or its symptoms.  The disorder can include difficulty with social skills, communication and repetitive behavior.

The CDC estimates autism's prevalence as one in 68 children in the United States: this includes one in 42 boys and one in 189 girls.

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

But now there are results from a very small clinical trial, involving just a few boys in San Diego, that is grabbing the attention of researchers and families.

University of California-San Diego genetics professor 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.

"Children began to talk sometimes for the first time in sentences in their life," recalled Dr. Naviaux.

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.  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," said McInerney.

He now uses rowing to reduce the stress related to his autism.

Dr. Naviaux began by testing Suramin on mouse models.

"We found that Suramin corrected all of the behavioral and learning disabilities if you started in the first month of life," he said.

"We did another study where we looked at animals with autism symptoms that were the age equivalent of a 30-year old that had never been treated before.  And even in that animal the behavioral symptoms and social abnormalities were completely corrected."

For those who did get the drug, Dr. Naviaux says 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," said Dr. Naviaux.   "Those fine motors skills were motor memory that had been retained."

Miles and his mom are encouraged by the results.

Dr. Naviaux says there will be several phase two trials to determine the safety and effectiveness of Suramin.  He believes it will be three to five years before phase three trials begin.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.