A drug typically studied for epilepsy may reverse key symptoms of autism, according to a new study in mice. The research, published Wednesday in Science Advances, explored how Z944—also known as ulixacaltamide—affects mouse models of autism spectrum disorder (ASD), which currently affects one in 31 children in the United States.
The researchers found that a single dose of Z944 reversed hallmark signs of autism in the mice, including heightened sensitivity to light and sound, repetitive behaviors, social difficulties, and an increased risk of seizures. The drug targets the reticular thalamic nucleus (RT), a part of the brain responsible for processing sensory information, effectively suppressing overactive neuronal activity in this region.
“Our findings suggest that neural circuits involved in both epilepsy and autism overlap,” the study authors noted, offering a potential explanation for why autistic individuals are up to 30 times more likely to develop epilepsy than the general population.
Autism diagnoses in the U.S. have risen sharply, from one in 150 children in the early 2000s to one in 31 today. While improved diagnosis and awareness are widely cited as contributing factors, some experts, including Health Secretary Robert F. Kennedy Jr., are investigating environmental influences such as pesticides, ultra-processed foods, and toxic metals.
Epilepsy affects approximately three million Americans—roughly one percent of the population—but nearly one in three autistic individuals have the condition. Scientists believe the link may stem from shared genetic mutations and similar abnormal brain activity patterns.
In the study, mice genetically modified with CNTNAP2 mutations, strongly associated with autism, exhibited overactive neurons in the RT due to excess currents in T-type calcium channels. Z944, a T-type calcium channel antagonist, blocked these currents, calming the region and reversing autistic behaviors. However, when the RT was artificially reactivated, the behaviors returned.
The researchers emphasized that while Z944 could potentially treat both autism and epilepsy, the drug is still in clinical trials for epilepsy, and its effects in humans remain untested. “Future research should aim to elucidate how RT-mediated circuit dynamics influence the broader neurobehavioral landscape of ASD, paving the way for circuit-specific, precision interventions,” they wrote.
READ MORE
AbbVie Invests $1.2B in Psychedelic Depression Drug from Gilgamesh
New Molecular Mechanism Could Transform Depression Treatment in Elderly
History of Traumatic Brain Injury Linked to Higher Malignant Brain Tumor Risk
