Approaches to the description and prediction of the binding affinity of small-molecule ligands to macromolecular receptors

Angew Chem Int Ed Engl. 2002 Aug 2;41(15):2644-76. doi: 10.1002/1521-3773(20020802)41:15<2644::AID-ANIE2644>3.0.CO;2-O.

Abstract

The influence of a xenobiotic compound on an organism is usually summarized by the expression biological activity. If a controlled, therapeutically relevant, and regulatory action is observed the compound has potential as a drug, otherwise its toxicity on the biological system is of interest. However, what do we understand by the biological activity? In principle, the overall effect on an organism has to be considered. However, because of the complexity of the interrelated processes involved, as a simplification primarily the "main action" on the organism is taken into consideration. On the molecular level, biological activity corresponds to the binding of a (low-molecular weight) compound to a macromolecular receptor, usually a protein. Enzymatic reactions or signal-transduction cascades are thereby influenced with respect to their function for the organism. We regard this binding as a process under equilibrium conditions; thus, binding can be described as an association or dissociation process. Accordingly, biological activity is expressed as the affinity of both partners for each other, as a thermodynamic equilibrium quantity. How well do we understand these terms and how well are they theoretically predictable today? The holy grail of rational drug design is the prediction of the biological activity of a compound. The processes involving ligand binding are extremely complicated, both ligand and protein are flexible molecules, and the energy inventory between the bound and unbound states must be considered in aqueous solution. How sophisticated and reliable are our experimental approaches to obtaining the necessary insight? The present review summarizes our current understanding of the binding affinity of a small-molecule ligand to a protein. Both theoretical and empirical approaches for predicting binding affinity, starting from the three-dimensional structure of a protein-ligand complex, will be described and compared. Experimental methods, primarily microcalorimetry, will be discussed. As a perspective, our own knowledge-based approach towards affinity prediction and experimental data on factorizing binding contributions to protein-ligand binding will be presented.

Publication types

  • Review

MeSH terms

  • Animals
  • Computer Simulation
  • Drug Design*
  • Enzyme Inhibitors / chemistry*
  • Humans
  • Ligands
  • Macromolecular Substances*
  • Models, Molecular
  • Protein Binding
  • Proteins / antagonists & inhibitors*
  • Structure-Activity Relationship
  • Thermodynamics

Substances

  • Enzyme Inhibitors
  • Ligands
  • Macromolecular Substances
  • Proteins