Reversing the Symptoms of Autism? A Century-Old Drug May be the Key

Researchers have surprised themselves after discovering that a 100-year-old therapy for African sleeping sickness appears to "reverse" the symptoms of autism in mice. However, the full affects of this drug, or how it impacts human autistics, remain to be seen.

According to a study published in the peer-reviewed journal Translational Psychiatry, researchers were looking for a way to inhibit prolonged purinergic signaling - a potentially harmful cell response that has been theorized to be a driving factor behind the autism symptoms.

This response occurs after a cell perceives a threat. To defend itself, a cell will stiffen its membrane, limiting interaction with the rest of the body and simultaneously changing metabolic processes. However, if this goes on for too long, or never turns off, the body and brain could start to function and even learn very differently.

"Cells behave like countries at war. When a threat begins, they harden their borders. They don't trust their neighbors. But without constant communication with the outside, cells begin to function differently," senior author Robert K. Naviaux said in a statement. "In the case of neurons, it might be by making fewer or too many connections. One way to look at this related to autism is this: when cells stop talking to each other, children stop talking."

The researchers theorized that genetic factors of autism may be making this defensive tactic too sensitive, to the point that it won't turn off.

After investigating ways to turn the process off, the researchers were surprised to find that suramin - a drug synthesized in 1916 to treat trypanosomiasis (African sleeping sickness) - could do just that.

Testing lab mice with autism spectrum disorder (ASD), the researchers found that suramin blocked signaling pathways that would normally trigger and perpetuate a cell danger response.

Amazingly, once the danger response was halted, ASD-like behavior in the mice stopped as well. A single dose was found to last for about five weeks.

However, there are some drawbacks. Turning off a natural defense mechanism is never quite the safest route, but does offer a trade-off that some people may be willing to accept.

"Obviously correcting abnormalities in a mouse is a long way from a cure in humans," Naviaux added, "but we think this approach is a new and fresh way to think about and address the challenge of autism."

The researchers will be launching a Phase 1 trail assessing the treatment in children with ASD later this year.

The study was published in Translational Psychiatry, a Nature publication, on June 17.