Backbone Cyclization Turns a Venom Peptide into a Stable and Equipotent Ligand at Both Muscle and Neuronal Nicotinic Receptors

J Med Chem. 2020 Nov 12;63(21):12682-12692. doi: 10.1021/acs.jmedchem.0c00957. Epub 2020 Oct 16.

Abstract

Venom peptides are promising drug leads, but their therapeutic use is often limited by stability and bioavailability issues. In this study, we designed cyclic analogues of α-conotoxin CIA, a potent muscle nicotinic acetylcholine receptor (nAChR) blocker with a significantly lower affinity at the neuronal α3β2 subtype. Remarkably, all analogues retained the low nanomolar activity of native CIA toward muscle-type nAChRs but showed greatly improved resistance to degradation in human serum and, surprisingly, displayed up to 52-fold higher potency for the α3β2 neuronal nAChR subtype (IC50 1.3 nM). Comparison of nuclear magnetic resonance-derived structures revealed some differences that might explain the gain of potency at α3β2 nAChRs. All peptides were highly paralytic when injected into adult zebrafish and bath-applied to zebrafish larvae, suggesting barrier-crossing capabilities and efficient uptake. Finally, these cyclic CIA analogues were shown to be unique pharmacological tools to investigate the contribution of the presynaptic α3β2 nAChR subtype to the train-of-four fade.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Conotoxins / chemistry
  • Cyclization
  • Larva / drug effects
  • Larva / physiology
  • Ligands*
  • Locomotion / drug effects
  • Mice
  • Muscle Contraction / drug effects
  • Muscles / metabolism*
  • Neurons / metabolism*
  • Nicotinic Antagonists / chemistry*
  • Nicotinic Antagonists / metabolism
  • Nicotinic Antagonists / pharmacology
  • Peptides / chemistry*
  • Peptides / metabolism
  • Peptides / pharmacology
  • Protein Binding
  • Protein Structure, Tertiary
  • Receptors, Nicotinic / chemistry
  • Receptors, Nicotinic / metabolism*
  • Venoms / metabolism*
  • Zebrafish / growth & development
  • Zebrafish / physiology

Substances

  • Conotoxins
  • Ligands
  • Nicotinic Antagonists
  • Peptides
  • Receptors, Nicotinic
  • Venoms
  • nicotinic receptor alpha3beta2