Reported highly effective in combination with TMZ in PTEN-deficient GBM mouse models.Given that the PTEN mutation is found in 36% of GBM patients, this is truly incredible.Also found effective in the treatment of patient-derived xenograft models and MGMT-unmethylated GBM cell lines when given along with RT.Due to the risk of reflux, there is moderate brain penetration with a brain-to-plasma ratio of 50%. The gene discussed is MGMT; the disease is glioblastoma.