Century-old drug may help children with autism symptoms: study
Published Friday, May 26, 2017 5:36AM EDT
Last Updated Friday, May 26, 2017 2:41PM EDT
A small study found that a 100-year-old drug called suramin, which is used to treat African sleeping sickness, showed promising results for treating autism spectrum disorder (ASD) symptoms in children.
Researchers at the University of California San Diego conducted a randomized double-blind clinical trial using 10 boys with ASD between the ages of five and 14.
Five boys received a single low dose of suramin while the other five got a placebo.
While no improvements were observed in the children who got the placebo, the results showed that the boys who got the drug displayed improvements in language and social behaviour.
“They began to engage in new activities in school playing kickball, singing, engaging with family members,” lead author Robert Naviaux told CTVNews.ca.
According to Naviaux, one 14-year-old boy, who was previously non-verbal, said his first sentence after receiving a dose of the drug.
“[The boy] came into the kitchen while his father was making a snack and said, ‘I want to eat chips,’” Naviaux said.
In a statement the parents’ of the boy said: “We saw our son advance almost three years in development in just six weeks."
Naviaux also said that while children were on the drug the benefit from all their usual therapies and enrichment programs increased dramatically.
The drug, which has previously shown to reverse signs of autism in mice, works by blocking a molecule called Adenosine triphosphate (ATP), which sends signals to the body’s cells warning that it’s under attack.
When the body thinks it’s under attack it sends signals to help the body limit the damage, heal and get things back to normal.
But this process can sometimes get stuck and the body’s cells react as if they’re constantly in danger.
Researchers believe that this may be an underlying cause of some traits of autism.
After searching through the “pharmaceutical encyclopedia” for any drug that would limit ATP, Naviaux found suramin, which was originally developed in 1916 to fight parasites.
By silencing the ATP alarm, suramin allows the body to return to normal and focus on other important processes, explained Naviaux.
“Suramin removed the roadblocks to development,” he said.
But the results were only temporary.
Naviaux told CTVNews.ca that after the initial dose the effects peaked around week three but had mostly faded after week six.
There were no serious side-effects reported but more research is needed to determine if suramin is an effective ASD treatment and safe long-term.
“We are cautiously optimistic that the symptoms of autism are caused by a treatable metabolic syndrome and even children that do have structural changes in the brain can make improvements,” Naviaux said.
Naviaux and his colleagues are now planning on running a second trial that looks at multiple doses over an extended period of time with 40 children.
But even if future trials rule out suramin as a possibility, Naviaux hopes that this trial blazes the trail for new treatments.
“I hope this opens a renaissance in drug development – even a drug that works like [suramin] could offer some hope for improving the future for lives of many kids,” he said.
Dr. Wendy Roberts, medical director at Integrated Services for Autism and Neurodevelopmental Disorders, agrees that though the study was too small to derive any definitive conclusions, but it does offer some hope for a possible future treatment.
“It’s encouraging in that all five of the children who got the suramin drug actually showed some improvement in core symptoms of autism,” she said. “Right now we don’t have a single drug that really targets autism, specifically.
“We tend to borrow drugs from other disorders like anxiety or inattention, so if we could find a drug that is helpful for a significant number of children and young people with autism, that would be great.”
Suramin is not currently approved for the treatment of autism and is not available in Canada.