Validation of a non-oncogene encoded vulnerability to Exportin 1 inhibition in pediatric renal tumors

We identified XPO1 as an aberrantly activated, non-oncogene encoded targetable vulnerability in pediatric renal tumors (Wilms and malignant rhabdoid tumor [MRT]) using OncoTarget, a Master Regular-based precision cancer medicine tool. We demonstrate significant anti-tumor activity of selinexor and eltanexor, two selective XPO1 inhibitors, in patient derived xenograft mice models of Wilms and MRT, and report on a case of successful treatment of a pediatric cancer patient. RNASeq profiles of MRT cell lines and PLATESeq profiles of selinexor (or DMSO control) perturbation in multiple MRT cell lines.