A drug typically studied for epilepsy may also reverse key symptoms of autism, a new study suggests.
Researchers at Stanford University tested the drug Z944, also known as ulixacaltamide, on mouse models of autism spectrum disorder (ASD), which currently affects roughly one in 31 children in the United States—a significant rise from one in 150 in the early 2000s.
The team found that a single dose of Z944 reversed behaviors associated with autism, including hypersensitivity to light and sound, repetitive movements, social deficits, and a heightened risk of seizures.
The researchers identified the reticular thalamic nucleus (RT), a brain region involved in processing sensory information, as the key target. Z944 suppressed overactivity in this area, effectively halting autistic-like behaviors in the mice.
These findings suggest that the neurological mechanisms underlying both epilepsy and autism may overlap, which could explain why individuals with autism are significantly more likely to develop seizures. Studies indicate autistic people face up to a 30-fold higher risk of epilepsy than the general population, potentially affecting cognition, speech, and social development.
In the study, published Wednesday in Science Advances, mice with mutations in the CNTNAP2 gene—a gene strongly linked to autism—showed overactive neurons in the RT. This overactivity appeared to be driven by excessive currents in T-type calcium channels, which Z944 blocks.
Mice treated with the drug displayed decreased repetitive behaviors, reduced social withdrawal, and lower hyperactivity. However, when the RT was artificially reactivated, the autistic-like behaviors returned, underscoring the region’s role in these symptoms.
While promising, researchers caution that Z944 is still in clinical trials for epilepsy and is not yet available for human use. The team emphasized that further studies are needed to determine whether these findings can translate to effective treatments for people with autism.
“Future research should aim to elucidate how RT-mediated circuit dynamics throughout the brain influence the broader neurobehavioral landscape of ASD, paving the way for circuit-specific, precision interventions,” the study concludes.
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