Long-Term Intranasal Oxytocin Shown to Be Safe in Adolescent Boys With Autism, With Some Signs of Social Improvement
A pilot study found that long-term intranasal oxytocin was safe and well-tolerated in eight adolescent boys with autism, with six showing improved social communication scores, though overall results were mixed.
Quick Facts
What This Study Found
Six of eight participants showed improved scores on the communication and social interaction domains of the Autism Diagnostic Observation Schedule (ADOS-G). Caregivers of five participants reported positive effects, particularly in the quality of reciprocal communication.
However, standardized behavioral measures (Child Behavior Checklist T-scores and Aberrant Behavior Checklist scores) did not reach statistical significance, though some subcategories showed mild improvement trends. Critically, all eight participants showed excellent compliance and zero side effects across the entire treatment period, with normal blood pressure, blood, and urine results throughout.
Key Numbers
How They Did This
This was a single-armed, open-label pilot study in eight male adolescents with ASD (ages 10-14, IQ range 20-101). Oxytocin was administered intranasally with stepwise dose increases every 2 months (8, 16, then 24 IU per dose). A 1-2 week placebo period was inserted before each dose step. Outcomes were measured using the ADOS-G, Child Behavior Checklist (CBCL), and Aberrant Behavior Checklist (ABC). Safety was monitored via blood pressure, blood tests, and urinalysis.
Why This Research Matters
Most previous oxytocin-autism research used single doses in adults, leaving major questions about whether the treatment is safe for children over extended periods. This study provided the first evidence that long-term nasal oxytocin is safe in adolescents with autism — a critical prerequisite for designing the larger, longer clinical trials needed to determine whether oxytocin can meaningfully help young people with social communication difficulties.
The Bigger Picture
This pilot study addressed a critical gap in oxytocin-autism research by demonstrating safety in a pediatric population over an extended treatment period. It represents an early step in the journey toward determining whether this natural neuropeptide could become a standard treatment for social difficulties in autism — one of the most sought-after therapeutic goals in developmental neuroscience.
What This Study Doesn't Tell Us
This was a very small (n=8), open-label study with no control group, making it impossible to separate oxytocin's effects from placebo responses or natural development. The wide IQ range (20-101) adds heterogeneity. Behavioral rating scales did not reach statistical significance. The authors themselves acknowledge the results are too preliminary for definitive conclusions about efficacy.
Questions This Raises
- ?Would a larger, double-blind, placebo-controlled trial confirm the social communication improvements seen in this pilot study?
- ?Is there an optimal dose of intranasal oxytocin for adolescents with autism, and does the stepwise dosing approach used here offer advantages?
- ?Do certain subgroups of children with autism (e.g., those with specific oxytocin system biomarkers) respond better to oxytocin treatment?
Trust & Context
- Key Stat:
- 6 of 8 boys improved on social communication measures In the first long-term safety study of nasal oxytocin in adolescents with autism, most participants showed improved ADOS-G scores with zero side effects over the treatment period.
- Evidence Grade:
- This is a small, open-label pilot study with no control group — the lowest tier of clinical evidence. While it provides important safety data, the lack of blinding and small sample size mean the efficacy signals should be interpreted with extreme caution. It primarily serves as justification for larger controlled trials.
- Study Age:
- Published in 2013, this was an early and pioneering study in pediatric oxytocin-autism research. Since then, several larger trials have been conducted in both children and adults, with mixed results on efficacy but generally consistent safety findings.
- Original Title:
- Long-term administration of intranasal oxytocin is a safe and promising therapy for early adolescent boys with autism spectrum disorders.
- Published In:
- Journal of child and adolescent psychopharmacology, 23(2), 123-7 (2013)
- Authors:
- Tachibana, Masaya, Kagitani-Shimono, Kuriko, Mohri, Ikuko, Yamamoto, Tomoka, Sanefuji, Wakako, Nakamura, Ayumi, Oishi, Masako, Kimura, Tadashi, Onaka, Tatsushi, Ozono, Keiichi, Taniike, Masako
- Database ID:
- RPEP-02292
Evidence Hierarchy
Frequently Asked Questions
Is intranasal oxytocin approved for treating autism?
No, intranasal oxytocin is not approved for treating autism spectrum disorder. This pilot study was one of the first to test its long-term safety in adolescents with ASD. While some participants showed improvement, the study was too small to prove efficacy. Larger clinical trials are needed before oxytocin could be considered for approval as an autism treatment.
Were there any side effects from long-term oxytocin use in these children?
No. All eight participants showed excellent compliance and zero side effects throughout the entire treatment period. Blood pressure, blood tests, and urine samples remained normal. This safety finding was the study's most important contribution, as it was the first to demonstrate long-term nasal oxytocin safety in children with autism.
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Cite This Study
https://rethinkpeptides.com/research/RPEP-02292APA
Tachibana, Masaya; Kagitani-Shimono, Kuriko; Mohri, Ikuko; Yamamoto, Tomoka; Sanefuji, Wakako; Nakamura, Ayumi; Oishi, Masako; Kimura, Tadashi; Onaka, Tatsushi; Ozono, Keiichi; Taniike, Masako. (2013). Long-term administration of intranasal oxytocin is a safe and promising therapy for early adolescent boys with autism spectrum disorders.. Journal of child and adolescent psychopharmacology, 23(2), 123-7. https://doi.org/10.1089/cap.2012.0048
MLA
Tachibana, Masaya, et al. "Long-term administration of intranasal oxytocin is a safe and promising therapy for early adolescent boys with autism spectrum disorders.." Journal of child and adolescent psychopharmacology, 2013. https://doi.org/10.1089/cap.2012.0048
RethinkPeptides
RethinkPeptides Research Database. "Long-term administration of intranasal oxytocin is a safe an..." RPEP-02292. Retrieved from https://rethinkpeptides.com/research/tachibana-2013-longterm-administration-of-intranasal
Access the Original Study
Study data sourced from PubMed, a service of the U.S. National Library of Medicine, National Institutes of Health.
This study breakdown was produced by the RethinkPeptides research team. We analyze and report published research findings without making health recommendations. All interpretations are based solely on the published abstract and study data.