What’s in the pipeline? Drugs in development for autism spectrum disorder

Abstract

Autism Spectrum Disorder (ASD) is a complex neurodevelopmental disorder with both core symptoms and associated symptoms (eg, irritability, aggression, and comorbidities) that affect both the individual and the family/systems around them. There have been recent advances in the understanding of the underlying pathophysiology of ASD pertaining to genetics, epigenetics, neurological, hormonal, and environmental factors that contribute to the difficulties found in individuals with ASD. With this improved understanding, there has been a shift in the application of psychopharmacology in ASD and its related disorders. A literature review was conducted to examine research published in the last 5 years between different classes of psychotropic medications and ASD. The broad scope of the existing literature for the use of conventional medications is summarized and novel medications are discussed.

Gonadotropin-releasing hormone agonist against severe aggression in autism

“…At this moment, research on novel medications in patients with ASD does not include the use of GnRH agonists 14. The long-acting GnRH agonist treatment strategy deserves more attention, preferably in a randomised controlled setting.…”

Exploratory analysis of L1 retrotransposons expression in autism

“…Effective medications for the treatment of ASD core symptoms are still lacking. Most of the drug currently in development for ASD is derived from knowledge of genes implicated in monogenic disorders associated with altered neurodevelopmental trajectories and autistic symptoms such as Fragile X, Landau–Kleffner and Rett syndromes [ 76 , 77 ]. As a consequence, the therapeutic approach to ASD subjects has traditionally focused on associated conditions [ 76 ], with poor impact for its core symptoms.…”

De novo Mutations From Whole Exome Sequencing in Neurodevelopmental and Psychiatric Disorders: From Discovery to Application

“…Despite many complications and challenges associated with NDP disorders, increasing implications of de novo event's contribute to the disease etiology, together with downstream functional analyses to explore the disrupted biological process by these de novo events, indicate the advent of the application of WES in potential treatments. Moreover, a large number of ASD drugs currently in the pipeline (Sung et al, 2014) keeps us optimistic about the future. The application of WES in clinical practice has the potential to generate an extraordinarily large dataset for multiple disorders.…”