A UCLA Health study found that one dose of rapamycin improved autism-like brain and behavior changes in adult mice within about two hours. The effects were temporary and the research was conducted only in mice.
The study, published in Nature Communications, showed that mild inflammation during pregnancy in mice led to lasting issues in offspring including brain overactivity, sensory sensitivity, repetitive behaviors, and higher seizure risk. Researchers gave adult offspring a single dose of rapamycin, an immune-suppressing drug, which quickly normalized neuron activity, reduced seizure vulnerability, and improved brain network communication.
Dr. Harley Kornblum, senior author and director of the UCLA Intellectual and Developmental Disabilities Research Center, said the rapid functional normalization suggests the adult brain may be more adaptable than assumed. Dr. Janel Le Belle, first author, noted that some autism-related features might be addressed by targeting functional circuitry rather than physical structure.
Dr. Neil Harris, co-senior author, cautioned that rapamycin's benefits did not last and repeated use can be toxic, making it unsuitable as a human treatment. The findings instead point to potential targets like mTOR pathway activity and neuronal balance for future therapies.
