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Calls to Action

Neuronal Nicotonic Receptor Modulation in the Treatment of Autism: A Pilot Trial of Mecamylamine

State/Province Full: 
United States

There is currently no FDA-approved medication for the core symptoms of autism. New directions for therapy need to be based on understanding the central neurobiology of autism. One consistently identified abnormality in autism is a reduction in one of the receptors (nicotinic) for the neurotransmitter acetylcholine. The proposed pilot trial aims to test the response of individuals with autism to agents that target this receptor type, thus changing acetylcholine signaling. There is one nicotinic agent that has been shown to be safe in other childhood-onset disorders, including Tourette's syndrome and attention-deficit/hyperactivity disorder (ADHD). This agent will be pilot-tested in children with autism. Twenty children with autism spectrum disorder will receive the drug or placebo over 13 weeks in a controlled (placebo double-blind) trial. The outcome will be assessed by standard clinical rating scales which will measure a range of behavioral and communicative functions, including attention. Computerized testing will also be used to assess changes in attention objectively. What this means for people with autism: This controlled pilot trial will provide essential evidence required to evaluate the potential of nicotinic-receptor therapy in the treatment of the core symptoms of autism.