Drugs currently under clinical investigation for relapsed/refractory neuroblastoma patients include the mTOR inhibitor, rapamycin (NCT01467986), and the ALK inhibitors, crizotinib (NCT00939770, NCT02559778, NCT01606878, NCT02034981) and LDK378 (NCT01742286). The gene discussed is ALK; the disease is neuroblastoma.