Autism is a neurodevelopmental disorder characterized by impaired social interaction and communication. The cause of autism is unknown, but several theories have been proposed. One theory speculates that exposure to stress or other cellular threats can trigger a “cellular danger response” involving purines. If this self-defense mechanism is not regulated properly, the response can remain permanently active, ultimately affecting neuronal development and lead to autism.
Based on this theory, Dr. Robert Naviaux from UCSD reasoned that Suramin, a drug commonly used to treat sleeping sickness, can be repurposed for autism treatment. Suramin acts by blocking purine’s binding to neurons. According to Naviaux, Suramin is capable of halting the negative effects of the active “danger response” on neuronal development and alleviate symptoms of autism. After positive results from mice experiments, researchers began an early stage clinical trial.
The clinical trial consisted of a cohort of 10 patients, and showed extremely promising results. Because the study was so small, it is essential to take the results with a grain of salt. The study also did not address whether participants were receiving behavioral intervention to treat autism, which could bias results. Suramine also has known side-effects including neuropathy, anemia and adrenal insufficiency. Because patients were only exposed to low doses over a short period of time, it’s possible that these negative side-effects could become a problem in future studies. Much more research is needed.
Acknowledgments: Many thanks to Christopher Rutt, MA, a graduate student from the Department of Psychology at Harvard University, for providing expertise and opinions on this subject matter.
Managing Correspondent: Bing Shui
Media Coverage: A drug used to treat sleeping sickness may also help with autism – The Economist
Original Publication: Low-dose suramin in autism spectrum disorder: a small, phase I/II, randomized clinical trial – Annuals of Clinical and Translational Neurology