[HTML][HTML] An androgen receptor N-terminal domain antagonist for treating prostate cancer

JK Myung, CA Banuelos, JG Fernandez… - The Journal of …, 2013 - Am Soc Clin Investig
JK Myung, CA Banuelos, JG Fernandez, NR Mawji, J Wang, AH Tien, YC Yang, I Tavakoli…
The Journal of clinical investigation, 2013Am Soc Clin Investig
Hormone therapies for advanced prostate cancer target the androgen receptor (AR) ligand-
binding domain (LBD), but these ultimately fail and the disease progresses to lethal
castration-resistant prostate cancer (CRPC). The mechanisms that drive CRPC are
incompletely understood, but may involve constitutively active AR splice variants that lack
the LBD. The AR N-terminal domain (NTD) is essential for AR activity, but targeting this
domain with small-molecule inhibitors is complicated by its intrinsic disorder. Here we …
Hormone therapies for advanced prostate cancer target the androgen receptor (AR) ligand-binding domain (LBD), but these ultimately fail and the disease progresses to lethal castration-resistant prostate cancer (CRPC). The mechanisms that drive CRPC are incompletely understood, but may involve constitutively active AR splice variants that lack the LBD. The AR N-terminal domain (NTD) is essential for AR activity, but targeting this domain with small-molecule inhibitors is complicated by its intrinsic disorder. Here we investigated EPI-001, a small-molecule antagonist of AR NTD that inhibits protein-protein interactions necessary for AR transcriptional activity. We found that EPI analogs covalently bound the NTD to block transcriptional activity of AR and its splice variants and reduced the growth of CRPC xenografts. These findings suggest that the development of small-molecule inhibitors that bind covalently to intrinsically disordered proteins is a promising strategy for development of specific and effective anticancer agents.
The Journal of Clinical Investigation