A five‑week course of the synthetic hormone oxytocin, given as a nasal spray, produced significant improvements in social, emotional and behavioral difficulties in young children with autism, according to research from the University of Sydney published in Molecular Psychiatry.
Led by investigators at the University’s Brain and Mind Centre, this trial provides the first clinical evidence that an intranasal oxytocin regimen can reduce core social interaction deficits in young children with autism. The study is also the first controlled clinical trial to evaluate the efficacy, tolerability and safety of oxytocin nasal spray specifically in a preschool and early‑school‑age group.
Autism spectrum disorder (ASD) comprises a range of neurodevelopmental conditions marked by challenges in social interaction and communication, together with restricted and repetitive behaviors. Current estimates put diagnosed prevalence at roughly one in 68 children, and, while behavioral therapies can improve social and emotional functioning, effective interventions remain limited, resource‑intensive and costly. To date, no medication has been widely accepted to treat the core social deficits of autism.
The randomized, double‑blind, placebo‑controlled crossover trial enrolled 31 children aged three to eight years. Each child received 12 international units (IU) of oxytocin nasal spray twice daily (24 IU per day) for five weeks, followed by a four‑week washout, with the alternate treatment given in the other arm of the crossover. Primary outcomes focused on caregiver‑rated social responsiveness, supported by blind clinician assessments conducted at the Brain and Mind Centre.
Results showed statistically significant improvements in social responsiveness after oxytocin treatment compared with placebo. Parents reported better social engagement at home, and independent clinician ratings documented observable gains in social responsiveness during clinical sessions. The nasal spray was generally well tolerated, with the most commonly reported side effects being increased thirst, more frequent urination and occasional constipation. No serious safety concerns were identified within the parameters of the trial.
Associate Professor Adam Guastella, an autism specialist at the Brain and Mind Centre, noted the study used widely accepted measures for assessing social responsiveness in children with autism and emphasized the concordance between parent reports and blinded clinician observations. Professor Ian Hickie, co‑director of the Brain and Mind Centre and co‑author on the paper, described the findings as an important first step toward developing medical treatments that target social deficits in autism.
The trial builds on a decade of research from the Brain and Mind Centre demonstrating that oxytocin can influence social processes in humans, enhancing eye gaze, emotion recognition and social memory across multiple populations. Recent work from the team has also begun to map oxytocin‑related changes to brain circuitry involved in social behavior. Future research aims to clarify the neural mechanisms by which oxytocin alters social circuitry and to explore whether oxytocin can be used to augment established behavioral, educational or technology‑based social learning interventions.
The research team plans to further develop oxytocin‑based approaches within a multidisciplinary model of care for autism, investigating optimal dosing, treatment duration and how pharmacological support might best complement behavioral therapies to achieve sustained improvements.
Funding: Supported by the National Health and Medical Research Council and the BUPA Health Foundation.
Source: Dan Gaffney – University of Sydney
Image source: Prerana Jangam (public domain). Image is included for illustrative purposes.
Original research citation: “The effect of oxytocin nasal spray on social interaction deficits observed in young children with autism: a randomized clinical crossover trial” by C. J. Yatawara, S. L. Einfeld, I. B. Hickie, T. A. Davenport and A. J. Guastella. Published in Molecular Psychiatry online 27 October 2015. doi:10.1038/mp.2015.162
Abstract
The effect of oxytocin nasal spray on social interaction deficits observed in young children with autism: a randomized clinical crossover trial
Interventions that effectively address core social and behavioral difficulties in autism are limited. We evaluated the efficacy, tolerability and safety of intranasal oxytocin in young children with autism using a double‑blind, randomized, placebo‑controlled crossover design. Thirty‑one children received 12 IU of oxytocin or placebo nasal spray twice daily (24 IU per day) for five weeks, separated by a four‑week washout. Compared with placebo, oxytocin produced significant improvements on the primary outcome of caregiver‑rated social responsiveness. The treatment was well tolerated overall; the most commonly reported adverse events were thirst, increased urination and constipation. These findings support the potential of oxytocin as an early intervention to help improve social interaction deficits in young children with autism.