OX2R-selective orexin agonism is sufficient to ameliorate cataplexy and sleep/wake fragmentation without inducing drug-seeking behavior in mouse model of narcolepsy

PLoS One. 2022 Jul 22;17(7):e0271901. doi: 10.1371/journal.pone.0271901. eCollection 2022.

Abstract

Acquired loss of hypothalamic orexin (hypocretin)-producing neurons causes the chronic sleep disorder narcolepsy-cataplexy. Orexin replacement therapy using orexin receptor agonists is expected as a mechanistic treatment for narcolepsy. Orexins act on two receptor subtypes, OX1R and OX2R, the latter being more strongly implicated in sleep/wake regulation. However, it has been unclear whether the activation of only OX2R, or both OX1R and OX2R, is required to replace the endogenous orexin functions in the brain. In the present study, we examined whether the selective activation of OX2R is sufficient to rescue the phenotype of cataplexy and sleep/wake fragmentation in orexin knockout mice. Intracerebroventricular [Ala11, D-Leu15]-orexin-B, a peptidic OX2R-selective agonist, selectively activated OX2R-expressing histaminergic neurons in vivo, whereas intracerebroventricular orexin-A, an OX1R/OX2R non-selective agonist, additionally activated OX1R-positive noradrenergic neurons in vivo. Administration of [Ala11, D-Leu15]-orexin-B extended wake time, reduced state transition frequency between wake and NREM sleep, and reduced the number of cataplexy-like episodes, to the same degree as compared with orexin-A. Furthermore, intracerebroventricular orexin-A but not [Ala11, D-Leu15]-orexin-B induced drug-seeking behaviors in a dose-dependent manner in wild-type mice, suggesting that OX2R-selective agonism has a lower propensity for reinforcing/drug-seeking effects. Collectively, these findings provide a proof-of-concept for safer mechanistic treatment of narcolepsy-cataplexy through OX2R-selective agonism.

MeSH terms

  • Animals
  • Cataplexy* / drug therapy
  • Disease Models, Animal
  • Drug-Seeking Behavior
  • Mice
  • Mice, Knockout
  • Narcolepsy* / drug therapy
  • Orexin Receptors / genetics
  • Orexin Receptors / metabolism
  • Orexins / pharmacology
  • Sleep / physiology
  • Wakefulness

Substances

  • Cd200r1 protein, mouse
  • Orexin Receptors
  • Orexins

Grants and funding

This study was funded by a grants from the World Premier International Research Center Initiative from Ministry of Education, Culture, Sports, Science and Technology, the Japan Society for the Promotion of Science (grant number 17H06095), the Core Research for Evolutional Science and Technology (grant number A3A28043), theFunding Program for World-Leading Innovative R&D on Science and Technology (FIRST Program) from JSPS, the Uehara Memorial Foundation, and the Japan Agency for Medical Research and Development (grant number JP21zf0127005), all awarded to MY. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.