Stanford researchers reversed autism-like behaviours in mice by targeting a key brain region, offering hope for future therapies.
PALO ALTO: In a groundbreaking study, Stanford Medicine scientists have successfully reversed autism-like symptoms in mice by focusing on a specific brain region known as the reticular thalamic nucleus (RT). The discovery, published in Science Advances, could open the door to new approaches in treating autism spectrum disorder.
Researchers found that overactivity in the RT, which regulates sensory information and communication between brain regions, triggered hallmark autism-related behaviours. These included heightened sensory sensitivity, repetitive motor movements, and increased seizure risk.
Lead investigator Dr. Jeanne Paz and her team tested Z944, an experimental drug originally developed for epilepsy. The medication calmed the RT’s hyperactivity, restoring balanced communication across the brain. Remarkably, the treatment completely reversed autism-like behaviours in mice that had previously been thought irreversible.
“Our findings provide evidence of RT hyperexcitability contributing to ASD-related behaviours and highlight the RT as a promising therapeutic target,” the researchers explained. The study highlights how precise circuit-level interventions could address multiple autism symptoms simultaneously, rather than relying on broad-spectrum approaches.
While the results are promising, the breakthrough remains limited to animal models. No human trials have yet been conducted, and translating these findings into safe, effective therapies will require years of further research and clinical testing. Nonetheless, the study offers rare optimism in the quest to better understand autism’s neurological roots and potential treatment pathways.
