Old Drug May Help Reduce Symptoms Of Autism

First Posted: Jan 15, 2015 11:41 PM EST
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Statisics show that autism spectrum disorders (ASD) affect 1 to 2 percent of children in the United States. With no cure for the problem, various therapies and medications can help many dealing with the behavioral health issue live a normal life.

Now, a recent study has found that a drug could play a significant role in correcting the issue. When treating a genetic form of autism-like symptoms in mice, the drug helped to correct the issue. 

Study author Robert K. Naviaux, MD, PhD, explains the new study's phenomenon, which he refers to as cellular danger response (CDR).

"When cells are exposed to danger in the form of a virus, infection, toxin, or even certain genetic mutations, they react defensively, shutting down ordinary activities and erecting barriers against the perceived threat. One consequence is that communication between cells is reduced, which the scientists say may interfere with brain development and function, leading to ASD."

During the study, researchers treated a Fragile X genetic mouse model with one of the most commonly studied mouse models of ASD via suramin-a drug that's been used for sleeping sickness. The approach, known as antipurinergic therapy or APT, helped to block the CDR signal and allow other cells to restore normal communications, reversing ASD symptoms.

"Our data show that the efficacy of APT cuts across disease models in ASD. Both the environmental and genetic mouse models responded with a complete, or near complete, reversal of ASD symptoms," Naviaux said. "APT seems to be a common denominator in improving social behavior and brain synaptic abnormalities in these ASD models."

The mouse model was started at nine weeks of age with the medicine, which is equal to the same age of an 18-year-old human. Twenty biochemical pathways associated with symptom improvements were identified from a metabolite analysis. Furthermore, the findings also support the hypothesis that disturbance in purinergic signaling - a regulator of cellular functions, and mitochondria (prime regulators of the CDR) - play a significant role in ASD.

Though the drug is some time away from being used in humans, the study and future research will promote new leads in understanding and treating autism.

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