The Simons Foundation Autism Research Initiative (SFARI) has awarded four grants in response to the 2019 Novel Outcome Measures in Autism Spectrum Disorder (ASD) request for applications (RFA). These grants will support the development and validation of clinical outcome measures aimed at assessing core symptoms of ASD and behavioral changes following treatment.
Evaluation of ASD-related behaviors has traditionally relied on parent- and/or clinician-based reports. These measures, however, are prone to subjective bias and are susceptible to placebo effects. Furthermore, they may lack the sensitivity to detect subtle but clinically meaningful changes in symptom severity. The goal of this award program is to develop and validate measures that can objectively characterize ASD phenotype(s) and reliably distinguish between treatment and placebo effects.
The 2019 RFA follows an earlier RFA (launched in 2015) centered around this same topic, which funded four projects aimed at developing quantitative measures of ASD-related behaviors, including social attention, emotion and language. Those projects advanced the frontiers of measurement science in autism, but intervention research would still benefit from better tools for outcome assessment.
“We need outcome measures that are objective and sensitive to modest change to better assess the efficacy of interventions for autism,” says Wendy Chung, director of clinical research at SFARI and the Kennedy Family Professor of Pediatrics in Medicine at Columbia University.
SFARI will provide $4.5 million over three years as part of the 2019 Novel Outcome Measures in ASD award program.
Paul Wang, deputy director of Clinical Research Associates LLC (an affiliate of the Simons Foundation) adds, “To test intervention approaches for autism, whether those approaches are behavioral or biological, we need outcome measures that are precise, reproducible and clinically meaningful. The measures that are available now either fall short on one of these counts or need further validation. It’s critical that we invest in this type of tool development and validation.”
The four new projects selected for funding are:
Stephen Kanne, Ph.D. (University of Missouri), Antonio Hardan, M.D. (Stanford University) and Micah Mazurek, Ph.D. (University of Virginia)
Multisite validation study of eye-tracking-based measures of autism symptom severity
Finding a tool that can quickly and accurately help triage risk of autism is a critical need in the field and can have a large impact on families and clinicians. Stephen Kanne, Antonio Hardan and Micah Mazurek plan to perform a multisite study to evaluate whether a tool called AutismEYES, which assesses an individual’s eye gaze, can provide a quantitative and objective measure to improve ASD identification and track symptom change over time.
Eva Loth and Robert Leech plan to combine age-appropriate animated cartoons with cutting-edge artificial intelligence techniques to optimize and validate a set of novel tablet tests assessing key social, emotional, motivational and cognitive processes implicated in core features of autism.
Guillermo Sapiro, in collaboration with Geraldine Dawson, seeks to develop and validate automatic, objective and quantitative measurements of social communicative and motor behaviors based on visual attention, affective facial expressions, vocalizations and head movements that can serve as feasible, reliable and sensitive outcome measures in autism clinical trials.
Helen Tager-Flusberg, Ph.D. (Boston University), Connie Kasari, Ph.D. (University of California, Los Angeles), So Hyun (Sophy) Kim, Ph.D. (Weil Cornell Medicine), Catherine Lord, Ph.D. (University of California, Los Angeles) and Shrikanth Narayanan, Ph.D. (University of Southern California)
Evaluating two newly developed treatment outcome measures (BOSCC and ELSA) in the context of an ASD behavioral intervention trial
Impairments in social communication are among the core symptoms of ASD, but at present, there are no validated tools that can be used to evaluate social communication outcomes in clinical trials. Researchers from three sites across the United States will validate two newly developed treatment outcome measures — the Brief Observation of Social Communication Change (BOSCC) and Elicitation of Language Samples for Analysis (ELSA) — over the course of an ASD behavioral intervention (JASPER, Joint Attention Symbolic Play Engagement and Regulation) trial.
The SFARI blog post “The need for objective outcome measures to advance intervention research in autism” discusses these projects and their significance for autism clinical research in further detail.