Research consortium looks to identify biomarkers for autism treatments

By Zachary Brennan

- Last updated on GMT

Research consortium looks to identify biomarkers for autism treatments
A new four-year research project involving a consortium of government, non-profit and other large biopharma partners is aiming to create a set of measures that can be used in clinical trials to determine which ASD (autism spectrum disorder) treatments are best for which patients and who will benefit from a particular treatment. 

The researchers said the ultimate goal is to validate a set of tools that will enable clinicians to objectively measure and predict how children with ASD respond to treatment.

The project will receive a total of $28m over the next four years from the NIH (National Institutes of Health) and other sources to test and refine clinical measures of social impairment in ASD in order to better evaluate potential behavioral and drug therapies.

The effort is supported by the Biomarkers Consortium​, a large public-private partnership that aims to accelerate biomedical research progress and includes the CRO (contract research organization) BioClinica, as well as other biopharma heavyweights such as Pfizer, Regeneron, Sanofi and Johnson & Johnson.

James McPartland of Yale School of Medicine, will serve as principal investigator and he and his team will conduct a multi-site study of preschool (3-5 years) and school aged (6-11 years) children, both with and without ASD, over the course of several months.

ASD is a group of neurodevelopmental disorders that affects social interaction and communication skills and can cause restricted and repetitive behaviors. Approximately 1% of children worldwide have an ASD, each with his or her own unique combination of symptoms and levels of impairment.

The heterogeneity in people with an ASD makes it imperative that we find more precisely diagnosed groups of research subjects so that we can objectively evaluate the clinical effects of an intervention​,” said NIMH (National Institute of Mental Health) Director Thomas R. Insel. “This consortium project will develop reliable tools and measures that clinical researchers can use to assess potential treatments​.”


The research team will compare lab-based measures of domains of social impairment to commonly used, standardized clinician and caregiver assessments of social function. Specifically, they will investigate the sensitivity and reliability of these unique measures in terms of how well they indicate changes in a participant’s core social impairment symptoms over time.

The researchers will then evaluate the potential utility of eye tracking responses and measures of brain activity via electroencephalogram (EEG) as biomarkers for future clinical trials. They will investigate how these two noninvasive and relatively inexpensive biomarker measures relate to their recently validated lab-based measures of social function, which will lay the groundwork for ASD researchers to objectively select meaningful subgroups of children and reliably measure the clinical effects of interventions.

In addition to the behavioral measures and biomarker data, this community resource will also include blood samples from subjects and their parents for use in future genetic studies. All data generated in the project will be made available for other researchers to view and analyze through the NIH-funded National Database for Autism Research​ and the NIMH Repository and Genomics Resource​.

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