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UC San Diego Health: Researchers Studying Century-Old Drug in Potential New Approach to Autism

Authors Scott LaFee and Heather Buschman, PhD

Small, randomized clinical trial reported measurable, but transient, benefits after single dose of suramin, highlighting novel causative theory and need for more, larger and longer trials.

In a small, randomized Phase I/II clinical trial (SAT1), researchers at University of California San Diego School of Medicine say a 100-year-old drug called suramin, originally developed to treat African sleeping sickness, was safely administered to children with autism spectrum disorder (ASD), who subsequently displayed measurable, but transient, improvement in core symptoms of autism.
Century-Old Drug Tested in Boys with Autism

Suramin is a 100-year-old drug developed to treat African sleeping sickness. It is not approved for any therapeutic use in the U.S. However, a small clinical trial at UC San Diego School of Medicine found that a single intravenous dose of suramin produced measurable, but transient, improvements in five boys with autism spectrum disorder (ASD). More broadly, the trial findings support the “cell danger response theory,” which posits that autism is driven by metabolic dysfunction — and thus treatable. Larger, longer clinical trials are needed to assess suramin as an ASD treatment. In this video we hear from lead researcher Robert Naviaux, MD, PhD, and the mothers of two boys who received suramin in the trial.

ASD encompasses a group of developmental disorders, often characterized by communication and language difficulties, repetitive behaviors and inability to socialize. The Centers for Disease Control and Prevention estimate that ASD occurs in 1 in 68 children, with the condition 4 times more common in boys than girls. ASD has no single known cause, but may involve both genetic problems and environmental factors, such as viral infections, pollutants or complications during pregnancy. One of the aims of the SAT1 study was to test the cell danger hypothesis as a possible unifying theory that contributes to the pathogenesis of ASD.

Writing in the Annals of Clinical and Translational Neurology, first author Robert K. Naviaux, MD, PhD, professor of medicine, pediatrics and pathology at UC San Diego School of Medicine and colleagues describe a novel double-blind, placebo-controlled safety study involving 10 boys, ages 5 to 14 years, all diagnosed with ASD.

Five of the 10 boys received a single, intravenous infusion of suramin, a drug originally developed in 1916 to treat trypanosomiasis (sleeping sickness) and river blindness, both caused by parasites. The other five boys received a placebo. The trial followed earlier testing in a mouse model of autism in which a single dose of suramin temporarily reversed symptoms of the neurological disorder.

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Myalgic Encephalomyelitis / Chronic Fatigue Syndrome (ME / CFS) Post Treatment Lyme Disease Syndrome (PTLDS), Fibromyalgia Leading Research. Delivering Hope.Open Medicine Foundation®

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