A new drug has been shown to be a potent autism “cure” for the offspring of pregnant mice, illuminating an early intervention method that could help rein in the incident of the neurodevelopmental disorder that currently affects one in 50 U.S. children.

Dr. Yehezkel Ben-Ari, a researcher at Aix-Marseille University in France and senior author of the new study, said in a press release that the new drug works by introducing the compound bumetanide, which in turn lowers the body’s chloride levels. Although the present lack of prenatal screening for autism spectrum disorder (ASD) makes it difficult to administer the medication to at-risk fetuses, the effect observed in mice opens up a new world of possibilities for researchers and drug developers. "This study validates the clinical trials using bumetanide to reduce chloride and restore strong GABAergic inhibition in humans with autism," he said. “This is one of the most promising clinical strategies for ASD currently available.”

The study also sheds new light on the process whereby autism is thought to develop. During birth, a “switch” mediated by oxytocin from the mother quiets the child’s neurons, protecting the brain circuitry during the vulnerable first moment of life. But in autism, this “switch” kicks in, and the child’s neuronal connections take damage. It is believed that higher levels of chloride are responsible for this misfiring.

The study, which is published in the journal Science, shows that the chloride-lowering bumetanide prevents autistic behavior when administered to mice pregnant with mouse models of the disorder. According to Ben-Ari, this boosts confidence in the current theory of development as well as the possibility of a human cure. "The observation that a single treatment of the mother before delivery prevents the expression of … features of autism in offspring illustrates the importance of conditions at delivery and the amazing long-term priming consequences of a wrong start,” he explained.

Autism Cure and Chloride

The current study is the latest in a growing series of responses to the alarming rise in U.S. autism diagnoses. Another example is a 2013 paper from Duke University, which shows that the risk of autism may be heightened in children born to mothers whose labor is induced or whose contractions are strengthened with procedures like hormone treatment. Scientists hope that these efforts will help dispel pseudo-scientific implications of vaccine schedules and bring the world closer to a cure for the disorder that costs the average caregiver tens of thousands each year.

 "I think that an early diagnosis of ASD coupled with a drug such as bumetanide or other regulators acting to reduce aberrant brain activities that perturb neuronal activities are likely future therapies," Ben-Ari concluded.

 

Source: Ben-Ari Y, Tsintsadze T, Khalilov I, et al. Oxytocin-Mediated GABA Inhibition During Delivery Attenuates Autism Pathogenesis in Rodent Offspring. Science. 2014.