Vasopressin Biology in Autism: From Biomarker to Treatment Target

Free webinar at 1:00 p.m. Eastern time (US), October 8, 2025

This webinar will:

· Describe scientific barriers to progress in developing laboratory-based diagnostic tests and new medications for patients with autism spectrum disorder.

· Determine the relative scientific merits of published findings from animal models of autism spectrum disorder by assessing their face and construct validity to the human disorder.

· Provide detailed scientific information on the biology of social deficits with an emphasis on vasopressin and oxytocin signaling pathways and biologically informed treatment trials in patients with autism spectrum disorder.

Autism spectrum disorder (ASD) is currently diagnosed behaviorally because its pathophysiology remains poorly understood. Consequently, there are no laboratory-based diagnostic tests to detect ASD and no disease-modifying medications that effectively treat its core behavioral features. The capability of rapidly detecting ASD based on neurochemical markers, however, would revolutionize ASD detection, enable more timely behavioral intervention, and provide targets for pharmacological treatment. To address these urgent unmet needs, we developed a translational ASD research platform, spanning studies of naturally low-social rhesus monkeys to children with ASD. Converging evidence from this body of research indicates that the neuropeptide vasopressin plays a critical and conserved role in regulating social abilities, and that brain vasopressin (but not oxytocin) signaling is impaired in low-social monkeys, children with ASD, and newborn infants before the period when ASD first manifests. On the basis of this compelling evidence, we conducted a double-blind, randomized, placebo-controlled pilot trial. We found that intranasal vasopressin treatment is well tolerated and significantly improves social abilities in children with ASD. These findings suggest that a neurochemical marker of impaired social functioning may be present very early in life, before behavioral symptoms emerge, and that the vasopressin signaling pathway may hold diagnostic and therapeutic promise for ASD.

Vasopressin Biology in Autism: From Biomarker to Treatment Target

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About the speaker:

Karen J. Parker, PhD is the inaugural Truong-Tan Broadcom Endowed Professor and Associate Chair of the Department of Psychiatry and Behavioral Sciences at Stanford University, where she leads the Major Laboratories Steering Committee and directs the Social Neurosciences Research Program. She is also an Affiliate Scientist at the California National Primate Research Center and a Fellow of the American College of Neuropsychopharmacology (ACNP). Dr. Parker received her undergraduate and graduate degrees from the University of Michigan. She completed postdoctoral training at Stanford University and joined the Stanford faculty thereafter. The principal goal of her research program is to better understand the biology of social functioning across a range of species, and to translate these fundamental insights to drive diagnostic and treatment advances for patients with social impairments, with a core focus on autism spectrum disorder. Dr. Parker’s research has been supported by the NIH, Simons Foundation, and Department of Defense, published in leading scientific journals (e.g., Science Translational Medicine, PNAS, Molecular Psychiatry), and featured across diverse media outlets (e.g., Huberman Lab podcast, NPR, CBS, New York Times, LA Times, Science, Scientific American). She has attended key opinion leader meetings at the U.S. National Academies and NIH, and held leadership roles on international research and ethics advisory committees for the Society for Neuroscience and ACNP. Dr. Parker currently lives in the San Francisco Bay Area with her husband, three children, and two Australian shepherds.

The post Vasopressin Biology in Autism: From Biomarker to Treatment Target appeared first on Autism Research Institute.

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