A new study has revealed that a well-known drug that was first synthesized in 1916 and used to treat African sleeping sickness can reverse autism-like symptoms in mice and it might help humans eventually.

The findings fit neatly with the idea that autism is caused by a multitude of interconnected factors: twenty percent of the known factors associated with autism are genetic, but most are not. It's wrong to think of genes and the environment as separate and independent factors. Genes and environmental factors interact. The net result of this interaction is metabolism.

Robert K. Naviaux, MD tested the effect of suramin, used to treat trypanosomiasis or African sleeping sickness, a parasitic disease and found that suramin blocked the extracellular signaling pathway used by ATP and other mitokines in a mouse model of autism spectrum disorder (ASD), ending the cell danger response and related inflammation. Cells subsequently began behaving normally and autism-like behaviors and metabolism in the mice were corrected.

However, the biological and behavioral benefits of suramin were not permanent, nor preventive. A single dose remained effective in the mice for about five weeks, and then washed out. Moreover, suramin cannot be taken long-term since it can result in anemia and adrenal gland dysfunction.

Naviaux said that correcting abnormalities in a mouse is a long way from a cure in humans but it is believed that this approach, antipurinergic therapy, is a new and fresh way to address the challenge of autism.

The study is published online in the journal Translational Psychiatry.