Measuring selectivity of oxytocin to enhance response to Pivotal Response Treatment in individuals with autism

  • Awarded: 2017
  • Award Type: Research
  • Award #: 514534

Pamela Ventola seeks to develop more precise treatments for children with autism spectrum disorder (ASD), guided by their distinct brain profiles. Informed by her recently published findings1, Ventola and her colleague, Kevin Pelphrey (George Washington University), will conduct a double-blind, placebo-controlled trial of intranasal oxytocin (OXT) as an enhancer of response to Pivotal Response Treatment (PRT) in young children with ASD. This trial will test the key hypothesis that administration of OXT as a social cognitive neural circuit enhancer will have a more pronounced behavioral effect on children with lower levels of activity in, and functional connectivity among, key social brain regions**.

Ventola and Pelphrey will assess this hypothesis in a 16-week trial of PRT using state-of-the-art brain-imaging and eye-tracking paradigms, as well as key behavioral outcomes. The functional Magnetic Resonance Imaging (fMRI) analysis will include a well-validated, sensitive and reliable paradigm involving coherent biological motion versus scrambled biological motion that Ventola and Pelphrey previously used to identify a biological marker of ASD in the Simons Simplex Collection (SSC) cohort2. Eye tracking will involve a set of novel, highly practical paradigms, developed as part of ongoing SFARI-funded work by Ventola, that are aligned specifically with behavioral outcomes targeted by PRT. Clinical outcomes will be assessed by the SRS-2 Total Score (primary) as well as additional well-validated behavioral scales.

This study will advance personalized medicine in ASD and serve as a proof-of-concept for the use of pharmacological compounds to enhance evidence-based behavioral and cognitive treatments for ASD. The study will also leverage the inferential power of a randomized experiment to elucidate the brain mechanisms underlying social communication deficits in ASD.

** Note: The current project was revised and awarded to Denis Sukhodolsky and Kevin Pelphrey in 2018 (award #592380).


1.Yang Y.J., et al. J. Neurodev. Disord. 9 (2017) PubMed
2.Yang D., et al. Transl. Psychiatry 6, e948 (2016) PubMed
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