A randomized placebo-controlled pilot trial shows that intranasal vasopressin improves social deficits in children with autism
Karen J. Parker,Ozge Oztan,Robin A. Libove,Noreen Mohsin,Debra S. Karhson,Raena D. Sumiyoshi,Jacqueline E. Summers,Kyle Hinman,Kara S. Motonaga,Jennifer M. Phillips,Dean S. Carson,Lawrence K. Fung,Joseph P. Garner,Antonio Y. Hardan +13 more
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TL;DR: Preliminary findings suggest that AVP has potential for treating social impairments in children with ASD, and a 4-week intranasal AVP daily treatment was well tolerated with minimal side effects.
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Abstract: The social impairments of autism spectrum disorder (ASD) have a major impact on quality of life, yet there are no medications that effectively treat these core social behavior deficits. Preclinical research suggests that arginine vasopressin (AVP), a neuropeptide involved in promoting mammalian social behaviors, may be a possible treatment for ASD. Using a double-blind, randomized, placebo-controlled, parallel study design, we tested the efficacy and tolerability of a 4-week intranasal AVP daily treatment in 30 children with ASD. AVP-treated participants aged 6 to 9.5 years received the maximum daily target dose of 24 International Units (IU); participants aged 9.6 to 12.9 years received the maximum daily target dose of 32 IU. Intranasal AVP treatment compared to placebo enhanced social abilities as assessed by change from baseline in this phase 2 trial’s primary outcome measure, the Social Responsiveness Scale, 2nd Edition total score (SRS-2 T score; F1,20 = 9.853; P = 0.0052; ηp2 = 33.0%; Cohen’s d = 1.40). AVP treatment also diminished anxiety symptoms and some repetitive behaviors. Most of these findings were more pronounced when we accounted for pretreatment AVP concentrations in blood. AVP was well tolerated with minimal side effects. No AVP-treated participants dropped out of the trial, and there were no differences in the rate of adverse events reported between treatment conditions. Last, no changes from baseline were observed in vital signs, electrocardiogram tracings, height and body weight, or clinical chemistry measurements after 4 weeks of AVP treatment. These preliminary findings suggest that AVP has potential for treating social impairments in children with ASD.
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Citations
Autism spectrum disorder
Catherine Lord,Traolach S. Brugha,Tony Charman,James C. Cusack,Guillaume Dumas,Thomas W. Frazier,Emily J.H. Jones,Rebecca M. Jones,Andrew Pickles,Matthew W. State,Julie Lounds Taylor,Jeremy Veenstra-VanderWeele +11 more
- 16 Jan 2020
TL;DR: This Primer by Lord and colleagues reviews the epidemiology, mechanisms, clinical detection and treatment of autism and identifies the long-term needs of people with autism.
Candidate biomarkers in psychiatric disorders: state of the field
Anissa Abi-Dargham,Scott J. Moeller,Farzana Hasan Ali,Christine DeLorenzo,Katharina Domschke,Guillermo Horga,Aman Jutla,Roman Kotov,Martin P. Paulus,Jose M. Rubio,Gerard Sanacora,Jeremy Veenstra-VanderWeele,John H. Krystal +12 more
TL;DR: A review of the evidence for the most promising biomarkers in the psychiatric neuroscience literature for autism spectrum disorder, schizophrenia, anxiety disorders and post-traumatic stress disorder, major depression and bipolar disorder, and substance use disorders is presented in this article .
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Rescue of oxytocin response and social behaviour in a mouse model of autism
Hanna Hörnberg,Enrique Pérez-Garci,Dietmar Schreiner,Laetitia Hatstatt-Burklé,Fulvio Magara,Stéphane J. Baudouin,Alex Matter,Kassoum Nacro,Eline Pecho-Vrieseling,Peter Scheiffele +9 more
TL;DR: This work identifies a convergence between the genetic autism risk factor Nlgn3 , regulation of translation, and oxytocinergic signalling, which would enable mechanism-based stratification of patient populations to increase the success of therapeutic interventions.
A phase 2 clinical trial of a vasopressin V1a receptor antagonist shows improved adaptive behaviors in men with autism spectrum disorder
Federico Bolognani,Marta del Valle Rubido,Lisa Squassante,Christoph Wandel,Michael Derks,Lorraine Murtagh,Jeff Sevigny,Omar Khwaja,Daniel Umbricht,Paulo Fontoura +9 more
TL;DR: Balovaptan treatment resulted in improved adaptive behaviors in adult men with autism, and dose-dependent and clinically meaningful improvements on the Vineland-II Adaptive Behavior Scales composite score were observed for participants treated with balov aptan 4 or 10 mg compared with placebo.
112
Signalling pathways in autism spectrum disorder: mechanisms and therapeutic implications
TL;DR: In this article , the authors reviewed recent insights into the mechanisms and clinical implications of signal transduction in autism from molecular, cellular, neural circuit, and neurobehavioural aspects.
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Discovery of Highly Selective Brain-Penetrant Vasopressin 1a Antagonists for the Potential Treatment of Autism via a Chemogenomic and Scaffold Hopping Approach
Hasane Ratni,Mark Rogers-Evans,Caterina Bissantz,Christophe Grundschober,Jean-Luc Moreau,Franz Schuler,Holger Fischer,Ruben Alvarez Sanchez,Schnider Patrick +8 more
TL;DR: From a micromolar high throughput screening hit 7, the successful complementary application of a chemogenomic approach and of a scaffold hopping exercise rapidly led to a low single digit nanomolar human vasopressin 1a (hV1a) receptor antagonist suitable for human clinical studies in people with autism.
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Early Predictors of Impaired Social Functioning in Male Rhesus Macaques (Macaca mulatta).
Valentina Sclafani,Valentina Sclafani,Laura A. Del Rosso,Shannon K. Seil,Laura A. Calonder,Jesus E. Madrid,Kyle J. Bone,Elliott H. Sherr,Joseph P. Garner,John P. Capitanio,John P. Capitanio,Karen J. Parker,Karen J. Parker +12 more
TL;DR: Testing whether juvenile LS and HS monkeys differed as infants in their ability to process social information, and whether infant social abilities predicted later social classification, suggests an early capacity to process important social information may account for differences in rhesus monkeys’ motivation and competence to establish and maintain social relationships later in life.
Impermeability of the blood‐cerebrospinal fluid barrier to 1‐deamino‐8‐D‐arginine‐vasopressin (DDAVP) in patients with acquired, communicating hydrocephalus
TL;DR: It is concluded that no measurable amounts of DDAVP can be found in the CSF for up to 2 h after i.v. administration of the peptide, and that for all practical purposes a blood‐CSF barrier to D DAVP exists.
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Plasma vasopressin concentrations positively predict cerebrospinal fluid vasopressin concentrations in human neonates.
Dean S. Carson,Christopher L. Howerton,Joseph P. Garner,Shellie A. Hyde,Catherine L. Clark,Antonio Y. Hardan,Anna A. Penn,Karen J. Parker +7 more
TL;DR: Preliminary support is provided for the use of basal plasma AVP measurement as a proxy for basal brain AVP activity in pediatric populations and the relationship between behavioral measures and AVP concentrations in both central and peripheral compartments in young infants and older children is determined.
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Psychophysiological analysis of the influence of vasopressin on speech in patients with post-stroke aphasias.
TL;DR: It is suggested that the neuropeptide optimizes the activity both in the left and right hemispheres, with primary influence on the right hemisphere.
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