Autism-like Symptoms Reversed by Sleeping Sickness Drug

Researchers at UC San Diego School of Medicine reported in the online journal ‘Molecular Autism’ that suranim, used for nearly a century as a drug for sleeping sickness, could correct not only environmentally influenced autism-like symptoms in mice, but also genetically-linked symptoms.

The risk of developing autism spectrum disorders (ASD) is increased by many different genetic and environmental factors, and as yet, there remains no firm explanation or permanent cure.

The study’s principal investigator, Robert K Naviaux, explained the mechanism which the drug targets as Cellular Danger Response (CDR). This is the phenomenon by which cells “shut-down” some activities in response to genetic mutations or other threats, severing communication pathways between cells in the brain. This is believed to be a possible basis of ASD.

Suranim was found to block the CDR signal, and therefore re-establish communication links, resulting in the improvement of symptoms involving biochemical pathways similarly reported in ASD in humans, therefore supporting also the hypothesis that CRT and ASD are linked.

This six-month study involved weekly treatment of young adult mice. Although there is a risk of toxicity were it to be used for more than a few months in humans, Navaux commented that a new class of drugs developed from this may be effective even with intermittent treatment, in relieving symptoms of ASD and improving response to traditional therapies.

“Correcting abnormalities in a mouse is a long way from a cure in humans,” warned Naviaux, “but our study adds momentum to discoveries at the crossroads of genetics, metabolism, innate immunity, and the environment for several childhood chronic disorders. These crossroads represent new leads in our efforts to understand the origins of autism and to develop treatments for children and adults with ASD.”