Sleeping sickness medication may help lessen ASD symptoms

(HealthDay)—Suramin, a drug first used in the early 1900s to treat sleeping sickness, has shown promise in an early trial as a potential treatment for autism spectrum disorder (ASD), according to research published online May 26 in the Annals of Clinical and Translational Neurology.

Robert Naviaux, M.D., Ph.D., of the Mitochondrial and Metabolic Disease Center at the University of California, San Diego's School of Medicine, and colleagues conducted a study involving 10 boys, aged 5 to 14, with ASD. This was the first human trial to attempt to replicate encouraging results seen in work with mice, the researchers noted. Five of the 10 study patients were given one intravenous infusion of suramin, while the other five were treated with a placebo.

The researchers found that all the boys treated with suramin ended up showing significant—and, in some cases, immediate—improvements, with no serious side effects. The most notable changes involved improved socializing, communication, and playing. Those in the suramin group also showed an increased ability to remain calm and focused, while displaying less repetitive behavior and better coping skills. Suramin also appeared to heighten the ability to benefit from standard treatment, such as speech therapy and behavior therapy. However, all the improvements from the one-dose treatment proved to be temporary, with gains ultimately fading over a matter of weeks.

"The safety and activity of low-dose suramin showed promise as a novel approach to treatment of ASD in this small study," the authors write.

Naviaux has filed a provisional patent application related to antipurinergic therapy for ASD.

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