US researchers have launched the first clinical trial to test whether a century-old drug used for African sleeping sickness may help treat children with autism.
The phase 1 clinical trial is the first to test the anti-parasitic drug suramin, as a novel treatment for children with a diagnosis of Autism Spectrum Disorder (ASD), researchers said.
In previous research, Robert K Naviaux at the University of California, San Diego School of Medicine and colleagues found that a single injection of suramin reversed symptoms of ASD in mouse models.
"Autism affects 1 to 2 per cent of children in the United States," said Naviaux, co-director of the Mitochondrial and Metabolic Disease Center at UC San Diego.
"Hundreds of genetic and environmental factors have been shown to increase the risk of ASD, but treatment options for the core symptoms are limited. To date, there are no medications that can cure ASD, and very few that can improve its core symptoms," he said.
To participate in the trial, a child must be a boy between the ages of 4 to 17 years and have been diagnosed with ASD by a psychologist or physician.
"He must be a resident of San Diego County and cannot have a known genetic cause of mutation (such as Fragile X syndrome) or be taking any prescription medications," researchers said.
In the trial, suramin will be given as a single dose through an intravenous line. Half of the participating children will receive suramin while the other half will receive a placebo (saline infusion).
Behavioural and medical tests will be conducted before and after the treatment, and include some blood and urine analyses, researchers said.
Since suramin treatment for autism is unprecedented, Naviaux emphasised it is not known whether suramin will produce any beneficial effect in humans.
He noted that suramin, as currently constituted, cannot be used for more than a few months without a risk of toxicity in humans and that it is not available as an ongoing treatment.
"So even if participants show benefit during the trial, it will not be possible to continue treatment after the study," said Naviaux.
"However, if the study is successful, they may be eligible for the next suramin study," he said.
