• Neuroblastoma cell lines, especially MYCN-expanded cell lines, are sensitive to this inhibitor. The inhibitor has a significant inhibitory effect on the growth activity of tumor cells, induces apoptosis and mediates G1 phase block.• Induction of p53 pathway activation and apoptosis in a mouse model of p53 wild-type tumor xenografts.• Combinations with other inhibitors significantly inhibit cell viability. The gene discussed is MYCN; the disease is neuroblastoma.