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Sleeping sickness drug cures autism in one dose

This image depicts the transmission electron micrograph of a cell mitochondrion.
This image depicts the transmission electron micrograph of a cell mitochondrion.
Credit: Thomas Deerinck, NCMIR, UC San Diego. Usage Restrictions: Must use photo credit as indicated.

Suramin, a drug developed to treat African sleeping sickness almost a century ago, has been shown to cure autism symptoms in mice in one dose. Dr. Robert K. Naviaux, professor of medicine, pediatrics and pathology at the University of California, San Diego School of Medicine, and colleagues based the discovery on the cellular behavior that is similar in autism and sleeping sickness. The discovery was reported in the June 17, 2014, edition of the journal Translational Psychiatry.

Naviaux and colleagues based their research on the concept that autism has a cellular basis. The contributing factors are genetic, environmental, and several other factors but the main cause of autism is the breakdown of communication between cells due to an immune response that limits the transfer of adenosine triphosphate (ATP) and compounds derived from ATP between cells. ATP is the energy source for all cells in the human body.

The similarity between autism and other diseases that produce an immune response is the key to prevention and reversal of autism according to the researchers. At present there are 19 known receptors for ATP compounds that act to produce communication between various types of cells. Language impairment in autism has been shown to be a manifestation of the lack of activity of one of the compounds derived from ATP. The common immune response that cells produce in the presence of disease or other threats is a thickening of the cell wall. Autism produces thickening of cell walls.

The studies in mice produced dramatic reversal of symptoms and a return to normal function in the brain cells of mice that had autism. The researchers concede that a cure for humans based on the same drug and concepts may be years away. The research does prove for the first time that a multiple factor approach that concentrates on cellular activity as it relates to autism has demonstrated the most dramatic success in finding a cure for autism to date.