Despite the accumulating evidence of an important role for the MAPK/ERK pathway in NB tumorigenesis, maintenance, and in particular, chemotherapy resistance/progression, preclinical studies with US Food and Drug Administration (FDA)-approved MEK inhibitors such as trametinib have not demonstrated wide-spread activity outside of RAS mutant NB cell lines, including a lack of activity against ALK mutant and MYCN-amplified NB (Hart et al., 2017; Umapathy et al., 2017). The gene discussed is MYCN; the disease is neuroblastoma.