Drug Treatments for Core Symptoms of Autism Spectrum Disorder: Unmet Needs and Future Directions

Pharmacological treatments for core symptoms of Autism Spectrum Disorder (ASD) are still lacking. The clinical heterogeneity observed in this population (e.g., differences in cognitive functioning or in autism symptom severity) should be taken into account when a new drug is tested. Stratifying this population according to its neurobiological substrate could significantly improve our knowledge regarding the most appropriate pharmacological treatment for individual needs. In this review, we discuss the possible genetic and biological pathways, including the Glutamatergic, GABAergic, and mTOR systems, involved in the pathophysiology of autism, as well as the mechanisms that may be targeted by new drug interventions. Finally, we describe the current progress from the preclinical and clinical studies on some potential therapeutic options for ASD core symptoms.