09/08/2025
Stanford scientists have reversed autism symptoms in mice – using brain-targeting drugs.
In a groundbreaking study, researchers at Stanford Medicine have identified a specific brain region – the reticular thalamic nucleus – as a key driver of autism-like behaviors in mice. And when they dialed down the activity in that region, the symptoms disappeared.
These weren’t small changes. Mice that had been genetically modified to mimic autism spectrum disorder (ASD) showed classic signs: hypersensitivity to sound and light, social withdrawal, repetitive behaviors, and seizures. But when scientists used experimental drugs to reduce neural hyperactivity in the reticular thalamic nucleus, those behaviors reversed.
The reticular thalamic nucleus works like a sensory gatekeeper, regulating the flow of information between the thalamus and the cortex. In the autism-model mice, it was firing too much – not just during stimulation, but spontaneously, triggering seizures and behavioral disruptions.
The team went further. Using neuromodulation techniques that allow neurons to be switched on or off with designer drugs, they could silence that brain region – and restore normal behavior. And when they artificially boosted activity in the same region in healthy mice, the animals began to show autism-like symptoms.
One of the most surprising parts of the study? The same drugs that worked in this model are already being investigated for epilepsy.
That’s more than a coincidence: around 30% of people with autism also have epilepsy, compared to just 1% of the general population. This shared brain mechanism may explain why the two conditions so often go hand in hand.
While this research is still in its early stages, it points to a powerful new possibility: that some forms of autism may be treatable by targeting specific neural circuits.
Read the study: "Reticular thalamic hyperexcitability drives autism spectrum disorder behaviors in the Cntnap2 model of autism." Science Advances, 20 Aug 2025.
