Recently, neuroblastoma PDXs were utilized to target the PI3K pathway (Chanthery et al. 2012; Mohlin et al. 2015; Stewart et al. 2015) and the ALK oncogene (Krytska et al. 2016) for nanofiber-mediated local delivery of the active metabolite of irinotecan (SN-38) (Monterrubio et al. 2016), for delivery of SN-38 nanoparticles conjugated to 3F8 antibodies thereby targeting GD2-expressing cells (Monterrubio et al. 2017) and for concurrent ketoconazole and fenretinide treatment (Lopez-Barcons et al. 2017). This evidence concerns the gene ALK and neuroblastoma.