Because KML001 induces Akt inactivation and PTEN activation [19] and because Akt activation with PTEN mutation is associated with glioblastoma, in this study, we evaluated the anti-tumor effects of KML001 using PTEN-negative human glioblastoma U87-MG cells in vitro and in vivo, and investigated the possible mechanisms involved in this process. This evidence concerns the gene PTEN and glioblastoma.