Alternative chemical modifications reverse the binding orientation of a pharmacophore scaffold in the active site of macrophage migration inhibitory factor

J Biol Chem. 2007 Aug 10;282(32):23089-95. doi: 10.1074/jbc.M701825200. Epub 2007 May 25.

Abstract

Pharmacophores are chemical scaffolds upon which changes in chemical moieties (R-groups) at specific sites are made to identify a combination of R-groups that increases the therapeutic potency of a small molecule inhibitor while minimizing adverse effects. We developed a pharmacophore based on a carbonyloxime (OXIM) scaffold for macrophage migration inhibitory factor (MIF), a protein involved in the pathology of sepsis, to validate that inhibition of a catalytic site could produce therapeutic benefits. We studied the crystal structures of MIF.OXIM-based inhibitors and found two opposite orientations for binding to the active site that were dependent on the chemical structures of an R-group. One orientation was completely unexpected based on previous studies with hydroxyphenylpyruvate and (S,R)-3-(4-hydroxyphenyl)-4,5-dihydro-5-isoxazole acetic acid methyl ester (ISO-1). We further confirmed that the unexpected binding mode targets MIF in cellular studies by showing that one compound, OXIM-11, abolished the counter-regulatory activity of MIF on anti-inflammatory glucocorticoid action. OXIM-11 treatment of mice, initiated 24 h after the onset of cecal ligation and puncture-induced sepsis, significantly improved survival when compared with vehicle-treated controls, confirming that inhibition of the MIF catalytic site could produce therapeutic effects. The crystal structures of the MIF inhibitor complexes provide insight for further structure-based drug design efforts.

Publication types

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

MeSH terms

  • Anti-Inflammatory Agents / chemistry
  • Binding Sites
  • Carbon / chemistry
  • Chemistry, Pharmaceutical / methods*
  • Crystallography, X-Ray / methods
  • Drug Design
  • Glucocorticoids / metabolism
  • Humans
  • Leukocytes, Mononuclear / cytology
  • Macrophage Migration-Inhibitory Factors / chemistry
  • Macrophage Migration-Inhibitory Factors / metabolism*
  • Models, Chemical
  • NF-kappa B / metabolism
  • Oximes / chemistry*
  • Oximes / pharmacology
  • Protein Binding
  • Recombinant Proteins / chemistry

Substances

  • 4-hydroxybenzaldehyde-O-cyclohexanecarbonyloxime
  • Anti-Inflammatory Agents
  • Glucocorticoids
  • Macrophage Migration-Inhibitory Factors
  • NF-kappa B
  • Oximes
  • Recombinant Proteins
  • Carbon

Associated data

  • PDB/2OOH
  • PDB/2OOW
  • PDB/2OOZ