In this work, we establish that, (1) Nilo1-specific targeting of GBM neurospheres depends on their stem-like phenotype, (2) Nilo1 affects the viability of GSCs but not normal stem cells, (3) in a portion of patient-derived GSCs Nilo1 treatment affects cell cycling and triggers differentiation in parallel with p21 induction, and (4) Nilo1 treatment kills a subset of patient-derived GSCs through a Bax-associated apoptotic mechanism. The gene discussed is BAX; the disease is glioblastoma.