Discovery of potent myeloid cell leukemia 1 (Mcl-1) inhibitors using fragment-based methods and structure-based design

J Med Chem. 2013 Jan 10;56(1):15-30. doi: 10.1021/jm301448p. Epub 2012 Dec 17.

Abstract

Myeloid cell leukemia 1 (Mcl-1), a member of the Bcl-2 family of proteins, is overexpressed and amplified in various cancers and promotes the aberrant survival of tumor cells that otherwise would undergo apoptosis. Here we describe the discovery of potent and selective Mcl-1 inhibitors using fragment-based methods and structure-based design. NMR-based screening of a large fragment library identified two chemically distinct hit series that bind to different sites on Mcl-1. Members of the two fragment classes were merged together to produce lead compounds that bind to Mcl-1 with a dissociation constant of <100 nM with selectivity for Mcl-1 over Bcl-xL and Bcl-2. Structures of merged compounds when complexed to Mcl-1 were obtained by X-ray crystallography and provide detailed information about the molecular recognition of small-molecule ligands binding Mcl-1. The compounds represent starting points for the discovery of clinically useful Mcl-1 inhibitors for the treatment of a wide variety of cancers.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Antineoplastic Agents / chemistry*
  • Crystallography, X-Ray
  • Databases, Factual
  • Drug Design
  • Humans
  • Indoles / chemical synthesis
  • Indoles / chemistry
  • Ligands
  • Magnetic Resonance Spectroscopy
  • Molecular Docking Simulation
  • Molecular Structure
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Protein Binding
  • Proto-Oncogene Proteins c-bcl-2 / antagonists & inhibitors*
  • Proto-Oncogene Proteins c-bcl-2 / chemistry
  • Small Molecule Libraries / chemistry*
  • Structure-Activity Relationship
  • bcl-X Protein / chemistry

Substances

  • Antineoplastic Agents
  • Indoles
  • Ligands
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Proto-Oncogene Proteins c-bcl-2
  • Small Molecule Libraries
  • bcl-X Protein