Structure-Activity Relationship and Molecular Mechanics Reveal the Importance of Ring Entropy in the Biosynthesis and Activity of a Natural Product

J Am Chem Soc. 2017 Feb 22;139(7):2541-2544. doi: 10.1021/jacs.6b10792. Epub 2017 Feb 13.

Abstract

Macrocycles are appealing drug candidates due to their high affinity, specificity, and favorable pharmacological properties. In this study, we explored the effects of chemical modifications to a natural product macrocycle upon its activity, 3D geometry, and conformational entropy. We chose thiocillin as a model system, a thiopeptide in the ribosomally encoded family of natural products that exhibits potent antimicrobial effects against Gram-positive bacteria. Since thiocillin is derived from a genetically encoded peptide scaffold, site-directed mutagenesis allows for rapid generation of analogues. To understand thiocillin's structure-activity relationship, we generated a site-saturation mutagenesis library covering each position along thiocillin's macrocyclic ring. We report the identification of eight unique compounds more potent than wild-type thiocillin, the best having an 8-fold improvement in potency. Computational modeling of thiocillin's macrocyclic structure revealed a striking requirement for a low-entropy macrocycle for activity. The populated ensembles of the active mutants showed a rigid structure with few adoptable conformations while inactive mutants showed a more flexible macrocycle which is unfavorable for binding. This finding highlights the importance of macrocyclization in combination with rigidifying post-translational modifications to achieve high-potency binding.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Biological Products* / chemistry
  • Biological Products* / pharmacology
  • Macrocyclic Compounds / chemistry
  • Molecular Conformation
  • Molecular Dynamics Simulation
  • Peptides / chemistry*
  • Peptides / genetics
  • Peptides / pharmacology
  • Structure-Activity Relationship

Substances

  • Biological Products
  • Macrocyclic Compounds
  • Peptides
  • thiocillin