Structure-guided design of a selective BCL-X(L) inhibitor

Nat Chem Biol. 2013 Jun;9(6):390-7. doi: 10.1038/nchembio.1246. Epub 2013 Apr 21.

Abstract

The prosurvival BCL-2 family protein BCL-X(L) is often overexpressed in solid tumors and renders malignant tumor cells resistant to anticancer therapeutics. Enhancing apoptotic responses by inhibiting BCL-X(L) will most likely have widespread utility in cancer treatment and, instead of inhibiting multiple prosurvival BCL-2 family members, a BCL-X(L)-selective inhibitor would be expected to minimize the toxicity to normal tissues. We describe the use of a high-throughput screen to discover a new series of small molecules targeting BCL-X(L) and their structure-guided development by medicinal chemistry. The optimized compound, WEHI-539 (7), has high affinity (subnanomolar) and selectivity for BCL-X(L) and potently kills cells by selectively antagonizing its prosurvival activity. WEHI-539 will be an invaluable tool for distinguishing the roles of BCL-X(L) from those of its prosurvival relatives, both in normal cells and notably in malignant tumor cells, many of which may prove to rely upon BCL-X(L) for their sustained growth.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology*
  • Apoptosis
  • Benzothiazoles / chemistry
  • Cell Line, Tumor
  • Crystallography, X-Ray
  • Drug Design*
  • Drug Screening Assays, Antitumor
  • Humans
  • Hydrazones / chemistry
  • Kinetics
  • Mice
  • Models, Chemical
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Protein Binding
  • Proto-Oncogene Proteins c-bcl-2 / genetics
  • bcl-X Protein / antagonists & inhibitors*
  • bcl-X Protein / chemistry*

Substances

  • Antineoplastic Agents
  • Benzothiazoles
  • Hydrazones
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Proto-Oncogene Proteins c-bcl-2
  • bcl-X Protein

Associated data

  • PDB/3ZK6
  • PDB/3ZLN
  • PDB/3ZLO
  • PDB/3ZLR
  • PubChem-Substance/162023956
  • PubChem-Substance/162023957
  • PubChem-Substance/162023958
  • PubChem-Substance/162023959
  • PubChem-Substance/162023960
  • PubChem-Substance/162023961
  • PubChem-Substance/162023962
  • PubChem-Substance/162023963