Autism Reversed in Mice With Century Old Drug

Autism was reversed with a century old drug in adult mice, according to a study recently reported in Translational Psychiatry. A single dose of an antipurinergic agent was used in the study and six-month-old adult mice showed restored social behavior, novelty preference and normal metabolism.

Autism is considered to be a spectrum of disorders that spans a range from minor to severe behavioral and social dysfunctions. It has been reported that about one to two percent of children born in the United States have autism. Many studies have been carried out to determine the genetic, environmental and metabolic factors that produce risk for the development of autism and a clear picture is yet to emerge.

The reported study was carried out at the University of California in San Diego. Dr. R. K. Naviaux from the Departments of Medicine, Pediatrics, and Pathology led the team of research scientists. In the study carried out by Dr. Naviaux and his team, they created a mouse model of autism; that is, they manipulated the mice to exhibit autistic behavioral characteristics. These behavioral characteristics included aversion to novelty and abnormal social behaviors.

Metabolic abnormalities also existed in these mice. Dr. Naviaux’s research does not just look at autism on a behavioral level but looks at the underlying metabolic factors on a cell level that contribute to the development of autism. He has pointed out that genes and the environment interact and the net result of this interaction is metabolism. When cells are “threatened” they produce the “cell danger response” and the metabolic effects of the cell danger response may be a metabolic basis for autism. It is thought that the cell danger response can cause changes in neurological development in children, and this may be what is happening in autism.

The mice in the study were given one dose of the century-old drug in an attempt to reverse autism. The drug is called suramin and was first discovered as a drug that kills organisms that cause African sleeping sickness. This drug is said to be antipurinergic, which means it blocks purinergic receptors in cells. It is thought that activity in these cell receptors is impaired in autism, thereby affecting brain development, the production of new synapses and other neurological factors. The hypothesis of the study was that suramin would possibly protect these receptors from overstimulation. The authors of the study stated that purine activity is a master regulator of behavior and metabolism in the mouse model, and that even a single dose of seramin acutely reversed the metabolic abnormalities of the mice they studied.

A previous study by this team, reported in 2013, showed that weekly injections of suramin reversed autistic traits in six-week-old immature mice. The study reported recently, however, was carried out in adult mice. The results of the recent study indicated that autism may be reversible in adults as well in young developing mice. The adult mice that were exhibiting autistic behaviors changed and began to lose their aversion to novelty (they investigated new areas of a maze) and were more social. The single dose of suramin also corrected metabolic pathways that were disrupted in the adult mice.

While the study is considered to be a success and provided important information about possible mechanisms underlying autism, much more work needs to be done. The researchers have begun a small clinical trial using this drug in humans. This study may be completed by the end of 2014. Since autism is a spectrum of disorders and varies a lot among individuals, it is not likely that one type of treatment will be a remedy for everyone. Showing that autism was reversed in mice with one dose of a century old drug, however, is a significant finding.

By Margaret Lutze

Translational Psychiatry
Medical News Today

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