XPO1 and acute myeloid leukemia: These inhibitors specifically and reversibly bind to residue Cys528 in the cargo-binding groove of XPO1. Selinexor (KPT-330) is the most advanced oral inhibitor of XPO1. Phase I/II human clinical trials have indicated that selinexor is well-tolerated and has a favorable outcome in patients with either relapsed or refractory acute myeloid leukemia (NCT01607892) and solid tumors (NCT01607905, NCT01896505) [22] (www.clinicaltrials.gov).