Targeting the MLL complex in castration-resistant prostate cancer

Nat Med. 2015 Apr;21(4):344-52. doi: 10.1038/nm.3830. Epub 2015 Mar 30.

Abstract

Resistance to androgen deprivation therapies and increased androgen receptor (AR) activity are major drivers of castration-resistant prostate cancer (CRPC). Although prior work has focused on targeting AR directly, co-activators of AR signaling, which may represent new therapeutic targets, are relatively underexplored. Here we demonstrate that the mixed-lineage leukemia protein (MLL) complex, a well-known driver of MLL fusion-positive leukemia, acts as a co-activator of AR signaling. AR directly interacts with the MLL complex via the menin-MLL subunit. Menin expression is higher in CRPC than in both hormone-naive prostate cancer and benign prostate tissue, and high menin expression correlates with poor overall survival of individuals diagnosed with prostate cancer. Treatment with a small-molecule inhibitor of menin-MLL interaction blocks AR signaling and inhibits the growth of castration-resistant tumors in vivo in mice. Taken together, this work identifies the MLL complex as a crucial co-activator of AR and a potential therapeutic target in advanced prostate cancer.

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

  • Animals
  • Cell Line, Tumor
  • Cell Nucleus / metabolism
  • Cell Proliferation
  • Drug Resistance, Neoplasm*
  • Histone-Lysine N-Methyltransferase / metabolism
  • Humans
  • Male
  • Mice
  • Mice, SCID
  • Myeloid-Lymphoid Leukemia Protein / metabolism*
  • Neoplasm Metastasis
  • Neoplasm Transplantation
  • Prostatic Neoplasms
  • Prostatic Neoplasms, Castration-Resistant / drug therapy
  • Prostatic Neoplasms, Castration-Resistant / metabolism*
  • Proto-Oncogene Proteins / metabolism*
  • Receptors, Androgen / metabolism*
  • Signal Transduction
  • Treatment Outcome

Substances

  • AR protein, human
  • KMT2A protein, human
  • MEN1 protein, human
  • Men1 protein, mouse
  • Proto-Oncogene Proteins
  • Receptors, Androgen
  • Myeloid-Lymphoid Leukemia Protein
  • Histone-Lysine N-Methyltransferase
  • Kmt2a protein, mouse

Associated data

  • GEO/GSE60842