Pharmacological Treatment Given in First Weeks of Life Prevents Symptoms of Autism

March 07, 2022

New Rodent Research Shows a Specific Window to Support Cerebellar Development 

A study released last month shows that timing is critical when treating developmental disorders like autism.  Researchers from the University of Texas Southwestern Medical Center have discovered that blocking an overactive signaling pathway during the first five weeks of life prevents symptoms of autism in developing mice. The current study set out to help develop targeted treatments for autism spectrum disorder (ASD), as currently none exist. The authors recognized that cerebellar pathology is often seen in individuals with ASD. Therefore, they set out to identify specific windows for cerebellar involvement in the development of ASD-relevant behaviors. The team’s goal was to find a pharmacological treatment which, given at the right time, could support cerebellar development and stop ASD in its tracks. They may have achieved this goal. To conduct this research, the team used a genetic mouse model of Tuberous Sclerosis Complex (TSC), a neurodevelopmental disorder associated with high rates of ASD. They took the diseased rodents and administered rapamycin, a drug that inhibits a pathway that is overactive in people and animals with TSC. After four weeks of treatment, starting in the first week of life, the mice displayed normal social behaviors and presented with normal activity in the cerebellar Purkinje neurons. After four weeks without treatment, the improvements still held. The study’s results point towards the possibility that treatment administered at critical periods of development could prevent autism from ever manifesting. TSC is often diagnosed in utero in humans, which would give ample time for doctors to develop a treatment plan for after birth.  


Original Article 

Study Abstract 

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