100-year-old drug may hold the key to treating autism

Drug developed 100 years ago to treat African sleeping sickness (trypanosomiasis) is being evaluated as a way to help individuals with autism spectrum disorders. According…

In this May 23, 2014 photo, Colleen Jankovich works with her 11-year-old autistic son, Matthew, who is non-verbal and requires 24/7 care, in Omaha, Neb. (AP Photo/Nati Harnik)

Drug developed 100 years ago to treat African sleeping sickness (trypanosomiasis) is being evaluated as a way to help individuals with autism spectrum disorders.

According to researchers, suramin, a treatment for African sleeping sickness (trypanosomiasis) that was synthesized in 1916, has successfully been used in a laboratory trial for the treatment of autism symptoms. Researchers indicate suramin was able to block the nucleotides, such as adenosine triphosphate (ATP), and the receptors linked to autism in the mice, effectively shutting down a process called “cell danger response.”

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This process, as explained to Medical News Today by senior author Dr. Robert K. Naviaux, is what happens when cells experience stress–as they would from factors contributing to autism.

“Twenty percent of the known factors associated with autism are genetic, but most are not,” said Naviaux. “Genes and environmental factors interact. The net result of this interaction is metabolism.”

When cells are threatened they stiffen their membranes and alter their metabolic processes. Unfortunately, prolonged cell danger response can cause permanent damage to cells and can lead to delayed neurodevelopment in children.

“Cells behave like countries at war. When a threat begins, they harden their borders,” he indicated. “They don’t trust their neighbors. But without constant communication with the outside, cells begin to function differently. 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.”

For this reason Naviaux and his team focused on chemical interactions that might reverse the cell danger response, thus leading them to the drug suramin. Suramin blocked ATP signaling pathways, halting the danger response in cells in mice genetically engineered to present with autism spectrum disorders equivalent to someone approximately 30 years of age.

With the cell danger response halted, cells in the mice started to behave normally again and the symptoms of autism disappeared.

The downside, explained the research team, was that the results of the medication intervention were not permanent; the mice remained symptom-free for only 5 weeks; however, the discovery may add another treatment tool to the arsenal for combination therapy for autism.

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“The discovery that a single dose of medicine can fundamentally reset metabolism for weeks means that newer and safer drugs might not need to be given chronically,” said Naviaux. “Obviously correcting abnormalities in a mouse is a long way from a cure in humans, but we think this approach – antipurinergic therapy – is a new and fresh way to think about and address the challenge of autism. Our work doesn’t contradict what others have discovered or done. It’s another perspective.”