Discovery and development of macrocyclic peptide modulators of the cannabinoid 2 receptor

J Biol Chem. 2024 Jun;300(6):107330. doi: 10.1016/j.jbc.2024.107330. Epub 2024 Apr 26.

Abstract

The cannabinoid type 2 receptor (CB2R), a G protein-coupled receptor, is an important regulator of immune cell function and a promising target to treat chronic inflammation and fibrosis. While CB2R is typically targeted by small molecules, including endo-, phyto-, and synthetic cannabinoids, peptides-owing to their size-may offer a different interaction space to facilitate differential interactions with the receptor. Here, we explore plant-derived cyclic cystine-knot peptides as ligands of the CB2R. Cyclotides are known for their exceptional biochemical stability. Recently, they gained attention as G protein-coupled receptor modulators and as templates for designing peptide ligands with improved pharmacokinetic properties over linear peptides. Cyclotide-based ligands for CB2R were profiled based on a peptide-enriched extract library comprising nine plants. Employing pharmacology-guided fractionation and peptidomics, we identified the cyclotide vodo-C1 from sweet violet (Viola odorata) as a full agonist of CB2R with an affinity (Ki) of 1 μM and a potency (EC50) of 8 μM. Leveraging deep learning networks, we verified the structural topology of vodo-C1 and modeled its molecular volume in comparison to the CB2R ligand binding pocket. In a fragment-based approach, we designed and characterized vodo-C1-based bicyclic peptides (vBCL1-4), aiming to reduce size and improve potency. Opposite to vodo-C1, the vBCL peptides lacked the ability to activate the receptor but acted as negative allosteric modulators or neutral antagonists of CB2R. This study introduces a macrocyclic peptide phytocannabinoid, which served as a template for the development of synthetic CB2R peptide modulators. These findings offer opportunities for future peptide-based probe and drug development at cannabinoid receptors.

Keywords: G protein-coupled receptor; allosteric modulator; cannabinoid type 2 receptor; peptide; plant.

MeSH terms

  • Cyclotides / chemistry
  • Cyclotides / pharmacology
  • Drug Discovery
  • HEK293 Cells
  • Humans
  • Ligands
  • Receptor, Cannabinoid, CB2* / agonists
  • Receptor, Cannabinoid, CB2* / chemistry
  • Receptor, Cannabinoid, CB2* / metabolism

Substances

  • Receptor, Cannabinoid, CB2
  • Ligands
  • Cyclotides
  • CNR2 protein, human