Gene/Viral Treatment Approaches for Malignant Brain Cancer

Abstract Gene therapy is a rapidly developing treatment modality that functions on its ability to deliver genes directly to the tumor site in order to yield antitumoral effects and thus prolong patient survival. The delivery of any gene(s) requires a delivery system that either involves the direct delivery of therapeutic gene(s) to the tumor site via viral or nonviral vectors, or through carriers that express the gene(s) in the tumor site via neural stem cells, mesenchymal stem cells, or other carriers. Four basic categories of gene therapy are currently being investigated for the treatment of brain tumors: (i) prodrug activation and suicide gene therapy; (ii) immunomodulatory and cytokine-based gene therapy; (iii) brain tumor hallmark targeting therapy; and (iv) oncolytic virotherapy. Preclinical studies of gene therapy for brain tumors have led to an array of human clinical trials, demonstrated to be remarkably safe and well tolerated with encouraging therapeutic results.