Targeting selectively oxytocin receptor signalling efficiently improves
social interaction in Fmr1 KO mice
Abstract
Background and Purpose: No drugs targeting the core social features of
autism spectrum disorder (ASD) have been approved. Although clinical
trials with oxytocin (OT) and vasopressin (AVP) have yielded mixed
results, targeting their receptors remains the most promising
pharmacological strategy for addressing social impairments in ASD. This
study aims to identify which receptors and signalling pathways within
this family can sustainably improve social impairments. Experimental
Approach: We used dose-response and kinetic analyses, along with
mathematical modelling, to evaluate OT, AVP, their homologs, and novel
synthetic ligands on G protein coupling, β-arrestins recruitment, and
internalisation of mouse oxytocin (OTR) and vasopressin (V1A, V1B, V2)
receptors in Neuro-2a cells. We tested acute and subchronic
administration of OTR agonists and the novel V1A receptor antagonist,
alongside OT and AVP, for their effects on social interaction in Fmr1 KO
mice, a model exhibiting ASD-like features. Key Results: While OT, AVP
and most compounds were non-selective across the four receptors, the OTR
agonists TGOT or RO6958375 and the V1A antagonist RO6893074 were
selective. TGOT or RO6958375, favouring Gαq signalling, enhanced social
interactions in Fmr1 KO mice while showing minimal effects in wild-type
mice. In contrast, OT, AVP or RO6893074 exhibited limited efficacy in
Fmr1 KO mice. Conclusion and Implications: Selective OTR agonists,
unlike OT and AVP, effectively improved social impairments in Fmr1 KO
mice after acute and subchronic treatment. These findings highlight the
necessity for developing highly selective OTR Gαq-biased agonists to
achieve clinical outcomes in ASD.