A small molecule blocking oncogenic protein EWS-FLI1 interaction with RNA helicase A inhibits growth of Ewing's sarcoma

Nat Med. 2009 Jul;15(7):750-6. doi: 10.1038/nm.1983. Epub 2009 Jul 5.

Abstract

Many sarcomas and leukemias carry nonrandom chromosomal translocations encoding tumor-specific mutant fusion transcription factors that are essential to their molecular pathogenesis. Ewing's sarcoma family tumors (ESFTs) contain a characteristic t(11;22) translocation leading to expression of the oncogenic fusion protein EWS-FLI1. EWS-FLI1 is a disordered protein that precludes standard structure-based small-molecule inhibitor design. EWS-FLI1 binding to RNA helicase A (RHA) is important for its oncogenic function. We therefore used surface plasmon resonance screening to identify compounds that bind EWS-FLI1 and might block its interaction with RHA. YK-4-279, a derivative of the lead compound from the screen, blocks RHA binding to EWS-FLI1, induces apoptosis in ESFT cells and reduces the growth of ESFT orthotopic xenografts. These findings provide proof of principle that inhibiting the interaction of mutant cancer-specific transcription factors with the normal cellular binding partners required for their oncogenic activity provides a promising strategy for the development of uniquely effective, tumor-specific anticancer agents.

Publication types

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

MeSH terms

  • Animals
  • Antineoplastic Agents / pharmacology*
  • COS Cells
  • Caspase 3 / metabolism
  • Cells, Cultured
  • Chlorocebus aethiops
  • DEAD-box RNA Helicases / metabolism*
  • Humans
  • Indoles / pharmacology*
  • Mice
  • Neoplasm Proteins / metabolism*
  • Oncogene Proteins, Fusion / antagonists & inhibitors*
  • Proto-Oncogene Protein c-fli-1
  • RNA-Binding Protein EWS
  • Sarcoma, Ewing / drug therapy*
  • Sarcoma, Ewing / pathology
  • Surface Plasmon Resonance
  • Transcription Factors / antagonists & inhibitors*
  • Xenograft Model Antitumor Assays

Substances

  • Antineoplastic Agents
  • EWS-FLI fusion protein
  • Indoles
  • Neoplasm Proteins
  • Oncogene Proteins, Fusion
  • Proto-Oncogene Protein c-fli-1
  • RNA-Binding Protein EWS
  • Transcription Factors
  • YK 4-279
  • Caspase 3
  • DHX9 protein, human
  • DEAD-box RNA Helicases