Does Cerebrospinal Fluid Contain a Biomarker for Autism?

New research suggests that vasopressin might act as a biomarker of ASD.

Posted Jun 15, 2020 | Reviewed by Kaja Perina

Today's post will focus on the neuropeptide vasopressin and its potential role as a biomarker in autism spectrum disorder (ASD). Vasopressin and other neuropeptides have been a common topic on this blog, and I previously wrote about studies in which intranasal vasopressin was administered to individuals with ASD to measure whether symptoms improved over time.

Today we are going to discuss a new study, published in May, 2020 in the journal, Proceedings of the National Academy of Sciences. The study was led by researchers at Stanford University and Washington University in St. Louis. The goal of the study was to measure whether the amount of vasopressin present in cerebrospinal fluid (CSF) in infants was related to the likelihood of those infants going on to have a later diagnosis of ASD. 

Study Methods

Researchers measured how much vasopressin was present in samples of CSF collected from 0-3 month old infants during the course of medical care. Since the samples were collected previously, researchers used medical records to identify which children had gone on to receive a diagnosis of ASD, and which ones had not. The authors identified 11 children who had gone on to have a diagnosis of ASD and 22 children who had not gotten a diagnosis of ASD. The two groups were matched on year of birth, sex, and ethnicity


The results showed the amount of vasopressin in CSF of infants who went on to get a diagnosis of ASD was much lower compared to infants who did not get a later diagnosis of ASD. Furthermore, when the researchers measured whether the amount of vasopressin in CSF could be used to statistically "predict" whether an individual child would go on to have a diagnosis of ASD or not, they found that it could accurately predict later ASD diagnosis with high accuracy. 


What can we take away from this study? As with all research we need to be careful not to over-interpret the findings, especially with a small sample of children. In all science, we need other researchers to replicate these findings with larger groups of children before drawing sweeping conclusions. 

That said, the results are exciting. Broadly speaking, we know that early diagnosis and intervention is effective in improving outcomes for children with ASD. Currently, we are only able to provide behavioral intervention after children begin showing symptoms of ASD. However, symptoms of ASD become clear around 9-12 months at the earliest, and for some children emerge later. It would be hugely beneficial to be able to predict whether a given child was at a particularly high risk of developing ASD so intervention could begin even before symptoms. Research like this is necessary in order to move closer to make these kinds of predictions and to intervene early to improve outcomes. 


Oztan O, Garner JP, Constantino JN, Parker KJ. Neonatal CSF vasopressin concentration predicts later medical record diagnoses of autism spectrum disorder. Proc Natl Acad Sci U S A. 2020;117(19):10609-10613. doi:10.1073/pnas.1919050117