100-Year-Old Drug Suramin Under Investigation As Autism Treatment

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An antimicrobial drug that was developed by researchers at German pharmaceutical company Bayer nearly 100 years ago is being investigated as a possible treatment for autism.

The drug, suramin (Germanin), was created in 1916 for the treatment of a type of a human sleep sickness known as African trypanosomiasis that is caused by a parasite. It has also been used as a remedy for onchocerciasis, more commonly known as river blindness, which is caused by a parasitic worm.

Researchers at the University of California, San Diego School of Medicine have begun a phase I clinical trial that will recruit 20 children who are all male, between the ages of four and 17, and have been diagnosed with autism. The participants cannot have a known genetic cause or mutation, such as Fragile X syndrome, or be taking any prescription medication.

The study — which is the first to test the drug in children — will involve 10 to 12 clinical visits over three to four months for each child. Half the children will receive suramin, intravenously, as a single dose or a placebo of saline. Behavioral and medical tests will be conducted before and after treatment.

In a prior research led by Robert K. Naviaux, MD, PhD, a professor of medicine, pediatrics and pathology at the medical school, and colleagues, a single injection of suramin reversed autism symptoms in mice.

Naviaux theorizes that autism may be the result of unusual cell communication due to  abnormal activation of the cell danger response, which can occur due to cells being damaged by bacteria, viruses, or chemicals such as pollutants.

100-Year-Old Drug Suramin Under Investigation As Autism Treatment
Researchers will investigate whether a single dose of suramin can reverse autism symptoms. Image courtesy of Bayer HealthCare Pharmaceuticals.

Researchers at the University of California, San Diego School of Medicine have launched a clinical trial to investigate the safety and efficacy of an unprecedented drug therapy for autism.

The phase 1 clinical trial, which is recruiting 20 qualifying participants, will evaluate suramin — a century-old drug still used for African sleeping sickness — as a novel treatment for children with a diagnosis of autism spectrum disorder (ASD). Previous published research by Robert K. Naviaux, MD, PhD, professor of medicine, pediatrics and pathology at UC San Diego School of Medicine, and colleagues reported that a single injection of suramin reversed symptoms of ASD in mouse models.

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