Based upon its ability to penetrate the BBB, to bypass anti-apoptotic mechanism to induce cell death, its activating mode-of-action, and the over-expression of procaspase-3 glioblastoma, PAC-1 has the potential as an orally delivered small molecule with a capacity for both single agent anti-glioblastoma activity, as well as the ability to synergize with TMZ to increase activity and achieve more durable responses in patients. The gene discussed is PACC1; the disease is glioblastoma.