A drug commonly investigated for epilepsy may reverse core symptoms of autism, according to a new study conducted on mice.
Researchers studied mouse models of autism spectrum disorder (ASD), a condition affecting one in 31 U.S. children and increasingly diagnosed since the early 2000s. The team administered Z944 (ulixacaltamide), a drug primarily explored as a potential epilepsy treatment, and observed striking results.
A single dose of Z944 reversed multiple autism-related behaviors in the mice, including heightened sensitivity to light and sound, repetitive movements, social deficits, and seizure susceptibility.
The drug achieved this by targeting the reticular thalamic nucleus (RT), a brain region responsible for processing sensory information. Suppressing overactivity in this area appeared to halt autistic behaviors.
The findings suggest a potential overlap between brain mechanisms underlying autism and epilepsy, which may explain why individuals with ASD are significantly more prone to seizures. Research indicates autistic individuals may be up to 30 times more likely to develop epilepsy than the general population, which can exacerbate cognitive and social challenges over time.
The study, led by Stanford University researchers and published in Science Advances, used genetically modified mice carrying mutations in the CNTNAP2 gene, strongly linked to autism.
These mice exhibited hyperactive neurons in the RT, likely driven by abnormal currents in T-type calcium channels. Z944, a T-type calcium channel antagonist, effectively blocked these currents, calming autistic behaviors and reducing hyperactivity.
When the researchers artificially increased activity in the RT, autistic behaviors returned, reinforcing the role of this brain region in symptom expression.
While the study highlights the therapeutic potential of Z944 for both autism and epilepsy, the drug is still in clinical trials for epilepsy, and its effects in humans remain untested. Researchers emphasized the need for further studies to explore how targeted interventions in RT circuits could inform precision treatments for ASD.
With autism diagnoses on the rise in the U.S.—from one in 150 children in the early 2000s to one in 31 today—these findings could pave the way for novel approaches to managing the condition.
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