Making progress in autism drug discovery

Currently, there are still just two medications approved by the U.S. FDA to treat the irritability that is often found in children with autism spectrum disorder (ASD). Furthermore, there are no drugs approved that treat the core symptoms of ASD. And while many drugs have had good outcomes in preclinical animal models, they have been met with very little success in human clinical trials. There are several factors likely contributing to this problem starting with the heterogeneity within the ASD phenotype. It is therefore important that researchers develop their knowledge about the neurobiological basis of ASD as well as possible subgroups within the disorder, based on either specific behavioral profiles or biological pathways. Furthermore, the development of valid biological markers will allow researchers to use more than parent or teacher reports to determine if a treatment is effective. This author believes that there a good chance of finding effective pharmacological treatments for the core symptoms of ASD with this increased knowledge of the biology and etiology.