Drug Development in Autism Spectrum Disorder

Two medications, risperidone and aripiprazole, are approved by the US Food and Drug Administration for the treatment of irritability in children age 5 to 17 with DSM-IV autistic disorder. There are no approved medications for social disability or repetitive behavior in autism specifically or autism spectrum disorder (ASD) more broadly. Over the past decade several genes have been identified that dramatically increase the risk of ASD. Identified genes include those causing monogenic disorders (e.g., Fragile X, neurofibromatosis), as well as rare mutations and de novo mutations. These genetic abnormalities may affect the structure and function of neurotransmitter receptors, the intracellular function of neurons, development of neural networks and signaling between neurons among other neurobiological functions. As more is learned about the function of the genes that contribute to the etiology of autism, there is expanding potential for drug development. This scientific panel will present a series of talks outlining the central issues facing drug development in ASD: the role of pilot studies, compound selection, the importance of biomarkers and ethical considerations for studies involving developmentally disabled individuals.
Friday, May 16, 2014: 3:30 PM-5:30 PM
Imperial A (Marriott Marquis Atlanta)