Orexin peptides prevent cataplexy and improve wakefulness in an orexin neuron-ablated model of narcolepsy in mice

Proc Natl Acad Sci U S A. 2004 Mar 30;101(13):4649-54. doi: 10.1073/pnas.0400590101. Epub 2004 Mar 16.

Abstract

Narcolepsy-cataplexy is a neurological disorder associated with the inability to maintain wakefulness and abnormal intrusions of rapid eye movement sleep-related phenomena into wakefulness such as cataplexy. The vast majority of narcoleptic-cataplectic individuals have low or undetectable levels of orexin (hypocretin) neuropeptides in the cerebrospinal fluid, likely due to specific loss of the hypothalamic orexin-producing neurons. Currently available treatments for narcolepsy are only palliative, symptom-oriented pharmacotherapies. Here, we demonstrate rescue of the narcolepsy-cataplexy phenotype of orexin neuron-ablated mice by genetic and pharmacological means. Ectopic expression of a prepro-orexin transgene in the brain completely prevented cataplectic arrests and other abnormalities of rapid eye movement sleep in the absence of endogenous orexin neurons. Central administration of orexin-A acutely suppressed cataplectic behavioral arrests and increased wakefulness for 3 h. These results indicate that orexin neuron-ablated mice retain the ability to respond to orexin neuropeptides and that a temporally regulated and spatially targeted secretion of orexins is not necessary to prevent narcoleptic symptoms. Orexin receptor agonists would be of potential value for treating human narcolepsy.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Animals
  • Carrier Proteins / genetics*
  • Carrier Proteins / therapeutic use*
  • Cataplexy / prevention & control*
  • Catheter Ablation
  • Disease Models, Animal
  • Genetic Therapy
  • Immunohistochemistry
  • Intracellular Signaling Peptides and Proteins*
  • Mice
  • Mice, Transgenic
  • Narcolepsy / genetics*
  • Narcolepsy / therapy*
  • Neurons / pathology
  • Neurons / physiology*
  • Neuropeptides / genetics*
  • Neuropeptides / therapeutic use*
  • Orexins
  • Protein Precursors / genetics
  • Rats
  • Wakefulness / drug effects*

Substances

  • Carrier Proteins
  • HCRT protein, human
  • Intracellular Signaling Peptides and Proteins
  • Neuropeptides
  • Orexins
  • Protein Precursors