The University of California (UC) San Diego School of Medicine, US, has reported positive data from a small, randomised Phase I/II clinical trial (SAT1) of the 100-year-old drug ‘suramin’ to treat boys with autism spectrum disorder (ASD).

Originally developed in 1916 for African sleeping sickness (trypanomiasis) and river blindness, suramin inhibits adenosine triphosphate’s (ATP) signalling function.

The results showed measurable, temporary improvement in core symptoms of the disease such as language, social behaviour, restricted or repetitive behaviour and coping skills.

The double-blind, placebo-controlled safety trial evaluated a single, intravenous dose of suramin in ten children aged five to 14 years over six weeks.

The main purpose of the trial was to test the researchers’ underlying theory that cell danger response is a unifying cause for autism. UC San Diego Mitochondrial and Metabolic Disease Centre co-director Naviaux said: “We had four non-verbal children in the study, two six-year-olds and two 14-year-olds.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

“The six-year-old and the 14-year-old who received suramin said the first sentences of their lives about one week after the single suramin infusion. This did not happen in any of the children given the placebo.”

“Even if suramin itself is not the best antipurinergic drug for autism, our studies have helped blaze the trail for the development of new antipurinergic drugs that might be even better.”

The observation that improvements in the children were temporary and faded after several weeks shows the necessity for larger and longer trials with multiple doses of the drug over longer periods.

Naviaux further added: “Even if suramin itself is not the best antipurinergic drug for autism, our studies have helped blaze the trail for the development of new antipurinergic drugs that might be even better.

“Before our work, no one knew that purinergic signalling abnormalities were a part of autism. Now we do, and new drugs can be developed rationally and systematically.”

According to the researchers, no adverse effects were found during SAT1 trial and the drug must be assessed in other larger trials to determine its long-term effects, effective dosage and frequency for various types of patients.


Image: African sleeping sickness is caused by tsetse flies infected by Trypanosoma parasites. Photo: courtesy of UC San Diego Health.