Oxytocin and autism - A precision medicine framework to unpack mechanisms and evidence

One of the great scientific questions in social neuroscience is how our knowledge of oxytocin, a neuropeptide and hormone with a crucial role in social behaviour, can be translated into meaningful advances to support social development. There is a compelling case for investigating oxytocin as a therapeutic target, from animal studies to converging human evidence from biomarker studies, genetic and epigenetic research, and neuroimaging findings implicating the oxytocinergic system in the social phenotype of Autism Spectrum Disorder (ASD). Despite decades of investigation, intranasal oxytocin has not shown consistent clinical benefits for autistic populations. This review synthesises evidence for oxytocinergic system involvement in ASD, with a focus on clinical trial findings. We argue for progress toward a precision medicine framework integrating biomarker-informed participant stratification, novel trial designs to control for placebo responses, objective outcome measures, and novel approaches to administration, drug development and endogenous stimulation. Together, these innovations offer opportunities to deliver on the potential to bridge existing knowledge of oxytocin's role in social development with its therapeutic potential for ASD.