Autism study identifies brain gate as therapy target

Researchers have identified hyperactivity in the reticular thalamic nucleus as a driver of autism-like behaviors in mice.

Experimental Drugs Reverse Autism Symptoms

Stanford Medicine researchers studied autism-like behaviors in a mouse model lacking the Cntnap2 gene. They found the reticular thalamic nucleus RTN, a brain gate between the thalamus and cortex, to be overactive during sensory input and social interaction, and this hyperactivity was linked to seizures, repetitive actions, and reduced social interaction. Using two methods to calm RTN activity, a seizure drug called Z944 and chemogenetic tools (DREADD), researchers reversed these deficits in the mice.

The researchers say the results point to a shared mechanism behind autism and epilepsy and open a path for therapies that target brain circuits. The work in Science Advances will need to be tested in humans to assess safety and effectiveness before any clinical use. Still, the finding marks a notable step toward understanding how the brain gates sensory information and how that gating goes awry in autism.

This study shifts attention from single genes to how brain wiring shapes behavior. It highlights a gatekeeper region that could be manipulated with drugs or neuromodulation. If people are later able to translate this to humans, it could lead to targeted therapies rather than broad behavioral interventions.

But there is caution. Results in mice rarely predict human outcomes. Safety, ethics, and the risk of hype around a cure require careful communication. The research also underscores why many people with autism experience epilepsy and how comorbidity could inform treatment choices.

Highlights

Future studies will test whether these results translate to humans.