Promising evidence and remaining issues regarding the clinical application of oxytocin in autism spectrum disorders
Open accessAbstract
Oxytocin is a potential therapeutic for the core symptoms of autism spectrum disorder (ASD), which is currently untreatable with pharmacotherapy. Previous clinical trials of a single dose of oxytocin have consistently reported significantly positive effects on various experimental measures associated with the core symptoms of ASD. These studies used various experimental measures as surrogate endpoints of the trials. However, to date, randomized clinical trials of continual administration of oxytocin have failed to reveal significant positive effects on clinically meaningful endpoints, such as how those with ASD interact during interpersonal interactions. This article reviews both the negative and positive effects of oxytocin on the core symptoms of ASD and their surrogate markers. Some unresolved and critical issues on the development of oxytocin as a new therapeutic have been extracted: optimization of dose, duration of oxytocin treatment, and the development of objective and reliable measurements of clinically meaningful endpoints for the core symptoms of ASD. Furthermore, optimization to the intranasal delivery system and careful consideration of how individuals respond differently to treatments should be addressed in future studies.
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Several clinical studies have investigated intranasal oxytocin as a treatment for ASD, but cannot provide a certain conclusion about its therapeutic potential.
“Several clinical studies have also investigated the potential of intranasal oxytocin as a treatment for socio-cognitive, behavioral, and socio-affective symptoms of ASD. So far, these studies cannot provide a certain conclusion about oxytocin’s therapeutic potential43–47.”
From Randomized clinical trial shows no substantial modulation of empathy-related neural activation by intranasal oxytocin in autism (2021) · cites this paper as [47]
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- Authors
- Yamasue, H.
- Affiliations
- Japan Science and Technology Agency (JP)
- Corresponding author
- Yamasue, Hidenori
- ORCID
- Yamasue, Hidenori
- Venue
- Psychiatry and Clinical Neurosciences
- Publisher
- Wiley
- Published
- 2015-10-16
- Volume
- 70
- Issue
- 2
- Pages
- 89-99
- ISSN
- 1323-1316 (ISSN-L)
- 1440-1819
- OpenAlex
- W1932491949
- Cited by
- 58
- References
- 47
- Citations by year
- 2026: 2 · 2025: 2 · 2024: 2 · 2023: 7 · 2022: 2 · 2021: 8 · 2020: 2 · 2019: 10 · 2018: 5 · 2017: 10 · 2016: 6 · 2015: 2
- FWCI
- 1.20
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Psychiatry and Clinical Neurosciences (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Japan Society for the Promotion of Science
- Japan Science and Technology Agency
- Ministry of Education, Culture, Sports, Science and Technology
- Ministry of Education
- Topics
- Social Sciences › Psychology › Social Psychology › Neuroendocrine regulation and behavior
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Keywords
- oxytocin, autism spectrum disorder, pharmacotherapy, core symptoms, surrogate endpoints, interpersonal interaction, dose optimization, treatment duration, intranasal drug delivery, treatment response variability, clinical trial endpoints
- MeSH terms
- Autism Spectrum Disorder, Administration, Intranasal, Humans, Interpersonal Relations, Oxytocin, Social Behavior, Treatment Outcome
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