ADAR1-IN-1 is a potent ADAR1 inhibitor. It significantly suppresses the proliferation of DU-145 cells (IC50 = 1.11 μM), inhibits colony formation, migration, and invasion, and induces cell cycle arrest and apoptosis. ADAR1-IN-1 effectively and safely impedes tumor growth and can be utilized in prostate cancer (PCa) research.

ADAR1-IN-1 (Compound C12) effectively inhibits the growth and colony formation of DU-145 cells at a concentration of 1 μM, with the most significant suppression observed at 3 μM over 1-10 days. Within 24 hours, ADAR1-IN-1 (1-3 μM) dose-dependently inhibits the migration and invasion of DU-145 cells and induces cell cycle arrest at the G0/G1 phase, along with apoptosis. At 48 hours, it reduces the expression of N-cadherin and vimentin while increasing p53 expression. Furthermore, after 72 hours, ADAR1-IN-1 significantly inhibits the proliferation of androgen receptor-dependent VCaP cells, castration-resistant 22Rv1 cells, and AR-negative PC-3 cells, with IC₅₀ values of 1.76 μM, 1.79 μM, and 0.72 μM, respectively.

ADAR1-IN-1 (Compound C12) administered intraperitoneally at doses of 15-30 mg/kg twice daily for 19 days, demonstrates significant antitumor efficacy and good safety in mouse models with DU-145 and 22Rv1 tumor xenografts.