- Wang, Junjian;
- Zou, June X;
- Xue, Xiaoqian;
- Cai, Demin;
- Zhang, Yan;
- Duan, Zhijian;
- Xiang, Qiuping;
- Yang, Joy C;
- Louie, Maggie C;
- Borowsky, Alexander D;
- Gao, Allen C;
- Evans, Christopher P;
- Lam, Kit S;
- Xu, Jianzhen;
- Kung, Hsing-Jien;
- Evans, Ronald M;
- Xu, Yong;
- Chen, Hong-Wu
The androgen receptor (AR) is overexpressed and hyperactivated in human castration-resistant prostate cancer (CRPC). However, the determinants of AR overexpression in CRPC are poorly defined. Here we show that retinoic acid receptor-related orphan receptor γ (ROR-γ) is overexpressed and amplified in metastatic CRPC tumors, and that ROR-γ drives AR expression in the tumors. ROR-γ recruits nuclear receptor coactivator 1 and 3 (NCOA1 and NCOA3, also known as SRC-1 and SRC-3) to an AR-ROR response element (RORE) to stimulate AR gene transcription. ROR-γ antagonists suppress the expression of both AR and its variant AR-V7 in prostate cancer (PCa) cell lines and tumors. ROR-γ antagonists also markedly diminish genome-wide AR binding, H3K27ac abundance and expression of the AR target gene network. Finally, ROR-γ antagonists suppressed tumor growth in multiple AR-expressing, but not AR-negative, xenograft PCa models, and they effectively sensitized CRPC tumors to enzalutamide, without overt toxicity, in mice. Taken together, these results establish ROR-γ as a key player in CRPC by acting upstream of AR and as a potential therapeutic target for advanced PCa.