Grainne McAlonan plans to use brain imaging methods and out-of-scanner measures to assess responses to arbaclofen, a GABAB receptor agonist, in adults with and without autism spectrum disorder.

Studying interactions between older children and adolescents with their adult caregivers is an important indicator of communication skills and are especially important for assessing children with ASD. Mark Clements aims to develop a fully automated audio recording tool for use in assessing communication skills in children with ASD in naturalistic (e.g., home) environments.

Dinstein will identify early neural abnormalities in two- to four-year-old toddlers with autism using whole-night EEG recordings in a hospital sleep lab. Furthermore, he will examine whether toddlers with regressive and classical autism exhibit distinct abnormalities that may indicate differences in their underlying pathology when comparing their brain activity and sleep architecture with that of typically developing toddlers.

An imbalance in excitatory/inhibitory transmission, due to alterations in GABAergic signaling, has been suggested as an underlying cause of autism. These studies argue that the GABAergic system represents a viable therapeutic target for autism. Indeed, the GABA-B receptor agonist R-baclofen has been shown to ameliorate social deficits and repetitive behaviors in two mouse models of autism1. However, baclofen is known to exhibit relatively poor blood-brain-barrier permeability, arguing for the development of drug treatments more accessible to the central nervous system. Yet such drug discovery efforts are complicated by a lack of suitable radiotracers for imaging GABA-B receptors.

Autism spectrum disorder (ASD), affecting 1 in 68 children in the U.S., is a significant unresolved public health concern. The clinical presentations of ASD can be quite broad, and recent evidence points to many different genetic causes. This heterogeneity could lead to a scientific and clinical impasse; each cause of ASD has its own disrupted mechanisms and requires its own unique treatment. However, a more optimistic interpretation, for which there is now accumulating evidence, is that many different primary causes of ASD actually converge on a limited subset of biochemical pathways in nerve cells that mediate cell growth and function. Demonstrating that such a mechanistic convergence exists would be a significant step forward for the field.

Chromosomal microarray analysis (CMA) is a robust technology officially recommended for children with autism spectrum disorders (ASDs). However, CMA genetic testing might raise ethical, legal and social issues such as stigmatization, health insurance concerns and test misconceptions. Therefore, it is essential to understand what might motivate or inhibit test decisions among parents of children with ASD.

Sensitive, standardized, reliable measures of outcomes for clinical trials attempting to change behavior and function in children and adults with autism spectrum disorder (ASD) have been extremely limited to date. Previous clinical trials have often relied on a single measure to quantify change, determined change in one domain or context (i.e., language or solely parent report or assessments only in the clinic), or used an approach measure with limited capability for replication, thus reducing the scope of the research.

How children recognize emotion from facial expressions, understand others’ perspectives, reason through social problems and regulate emotion is critical to their social success. These social cognition skill areas are often affected in children with autism spectrum disorders (ASDs). They are also frequent targets of both behavioral and medical interventions for ASD. There are no reliable, comprehensive, easy-to-use, scalable and standardized assessment tools available to measure responsiveness to treatment in each of these social domains among children with ASD.