Isoflavones, bioactive soy compounds, are known to exhibit anticancer activities. The present study investigated the anticancer activities of isoflavones on human retinoblastoma Y79 cells in vitro and in vivo. An MTT cell viability assay showed that the half maximal inhibitory concentration value of isoflavones against human retinoblastoma Y79 cells is 1.23 +/- 0.42 mu mol/l. Flow cytometry analysis indicated that isoflavones blocked G1/S progression. Western blot analysis demonstrated that the mammalian target of rapamycin (mTOR) pathway in Y79 cells was inhibited by isoflavones, with a concomitant decrease in cyclin E1, which accounted for the isoflavone-mediated G1 phase arrest. Isoflavones also inhibited human retinoblastoma growth in vivo; western blot analysis showed inhibition of mTOR and downregulation of cyclin E1 in an isoflavone-treated xenograft mouse model. Together, these results illustrate that isoflavones inhibit retinoblastoma tumour growth in vitro and vivo and that inactivation of the mTOR pathway and downregulation of cyclin E1 is involved in this action. The results of this study suggest that isoflavones could be tested as promising anti-retinoblastoma agent.