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. 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.