Western blotting analysis of protein extracts from the tumor tissues revealed that IDH2 inhibition led to a significant downregulation of C-MYC expression in vivo (Fig. 8j, l), consistent with the observation in vitro, where AML cells with IDH2 knockdown or treated with AGI-6780 exhibited a substantial reduction in C-MYC expression (Figs. 6a, b and 8c, g). The gene discussed is IDH2; the disease is acute myeloid leukemia.