Using Propranolol to Treat the Core Symptoms of Autism Spectrum Disorder

Posted April 19, 2022

David Beversdorf, M.D., University of Missouri

David Beversdorf, M.D., University of Missouri
Dr. David Beversdorf

Autism spectrum disorder (ASD) is characterized by impairments in social communication and restrictive and repetitive behaviors. Most current pharmacological treatments of ASD focus on symptoms such as agitation, anxiety, obsessive behaviors, and depression; however, there are no drugs proven to address the core components of autism, such as language and social interaction. Propranolol is a drug used to treat hypertension, but is also known for its anti-anxiety effects and used to treat performance and public speaking anxiety. In a small study, Dr. David Beversdorf and his team previously reported a significant beneficial effect from a single dose of propranolol on a structured social interaction task in ASD. With support from a Fiscal Year 2015 (FY15) Autism Research Program Clinical Trial Award, Dr. Beversdorf conducted a large study aimed at investigating the effect of propranolol on youth with ASD and determine whether the team can find specific markers to predict response.

In a double-blind placebo-controlled serial dose clinical trial (NCT02871349), the team tested serial doses of propranolol and examined the effects on social interaction, as well as on language tasks, anxiety, adaptive behaviors, and global function. The subjects included both high-functioning youth and children with autism (ages 7–24). Autism diagnoses were confirmed prior to the beginning of the trial. Participants attended a baseline session where the research team initiated propranolol titration, recorded baseline heart rate variability (HRV), and assessed social reciprocity using the General Social Outcomes Measure (GSOM). The team also used the Clinician Global Clinical Impression-Severity (CGI-S) and the Spence Children’s Anxiety Scale (SCAS) to measure anxiety. The autistic children took propranolol daily for 12 weeks and were assessed for measures of anxiety. At 6 and 12 weeks, the participants followed up via telehealth. The CGI-S, SCAS, and GSOM were assessed again after 12 weeks of treatment. The results of the CGS-S were compared for anxiety between baseline and follow-up and contrasted between the treatment and placebo groups. At 12 weeks, significant improvements in anxiety were observed and were about 2.6 times greater in the treatment group than in the control. As frequently observed in ASD trials, a marked response was also observed in the placebo group; however, the difference in treatment outcomes between the groups reveals a potential for using propranolol for anxiety. There was no overall difference between groups for response to propranolol on social measures. However, for participants aged 7–14, there was a significant relationship between better response to propranolol on conversational reciprocity and lower HRV. This suggests that, in these younger patients, the response to propranolol can be predicted based on markers such as HRV.

Further studies are needed to confirm specificity to the younger population and establish a broader understanding of markers that would predict the best responders to propranolol. Despite this, if propranolol can successfully treat a core component of ASD, this would both address a treatment gap in ASD and represent a new treatment option. This could be particularly important, as it would have an earlier impact in developing children and impact the underserved older population as well. As this drug is also widely available in generic form, it would increase access to care in underserved communities.

Zamzow RM, Ferguson BJ, Stichter JP, Porges EC, Ragsdale AS, Lewis ML, Beversdorf DQ. 2016. Effects of propranolol on conversational reciprocity in autism spectrum disorder: a pilot, double-blind, single-dose psychopharmacological challenge study. Psychopharmacology (Berl). Apr;233(7):1171-8. doi:10.1007/s00213-015-4199-0. Epub Jan 14. PMID:26762378.

Public and Technical Abstracts: Trial of Propranolol in Children and Youth with ASD and Predictors of Response

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Last updated Thursday, May 26, 2022