Innovative Study Reveals Potential to Reverse Autism Symptoms in Mice Using Epilepsy Medications

Scientists at Stanford Medicine have uncovered new insights into the neurological mechanisms underlying autism spectrum disorder (ASD). Their research highlights that heightened activity in a specific brain region, the reticular thalamic nucleus, may be a key driver of behaviors associated with autism. Utilizing a genetically engineered mouse model that simulates aspects of ASD, researchers observed that hyperactivity in this brain area correlated with increased sensitivity to stimuli, repetitive actions, social interaction deficits, and seizure susceptibility.

The team identified the reticular thalamic nucleus as a crucial gateway for sensory information transfer between the thalamus and cortex. Through detailed neural recordings, they detected abnormal spontaneous activity and hyperexcitability in this region, especially when mice encountered stimuli like light or mild air puffs.

Importantly, the researchers demonstrated that administering drugs known to suppress activity in this brain region could reverse autism-like behaviors. They used the epilepsy drug Z944, which effectively mitigated behavioral deficits in the mouse model. Additionally, employing a cutting-edge technique called DREADD-based neuromodulation, they genetically modified neurons to respond to designer drugs, allowing precise suppression of the overactive reticular thalamic nucleus. This approach not only reversed behavioral issues but also induced similar deficits in normal mice by artificially increasing activity in this area.

These findings emphasize the overlap between the neural circuits involved in autism and epilepsy. Given that epilepsy is more common in individuals with ASD—affecting approximately 30% compared to 1% in the general population—this research may pave the way for novel targeted therapies. The drugs and methods tested in mice could inform future treatments for human patients.

The study, led by senior author John Huguenard, Ph.D., and published in Science Advances, sheds light on the potential for new interventions that target specific brain circuits to treat ASD, especially in cases where epilepsy co-occurs. The research deepens our understanding of how neural circuitry abnormalities contribute to autism and advances the search for effective treatments.

Source: Medical Xpress