Gene therapy in glioblastoma multiforme: Can it be a role changer?

• Published in: Heliyon 2024-03, Vol.10 (5), p.e27087-e27087, Article e27087
• Main Authors: Rayati, Mohammad, Mansouri, Vahid, Ahmadbeigi, Naser
• Format: Article
• Language: English
• Subjects: GBM; Gene therapy; Glioblastoma; Immunotherapy; Malignant glioma; Review; Viral vector
• ISSN: 2405-8440; 2405-8440
• DOI: 10.1016/j.heliyon.2024.e27087

Glioblastoma multiforme (GBM) is one of the most lethal cancers with a poor prognosis. Over the past century since its initial discovery and medical description, the development of effective treatments for this condition has seen limited progress. Despite numerous efforts, only a handful of drugs have gained approval for its treatment. However, these treatments have not yielded substantial improvements in both overall survival and progression-free survival rates. One reason for this is its unique features such as heterogeneity and difficulty of drug delivery because of two formidable barriers, namely the blood-brain barrier and the tumor-blood barrier. Over the past few years, significant developments in therapeutic approaches have given rise to promising novel and advanced therapies. Target-specific therapies, such as monoclonal antibodies (mAbs) and small molecules, stand as two important examples; however, they have not yielded a significant improvement in survival among GBM patients. Gene therapy, a relatively nascent advanced approach, holds promise as a potential treatment for cancer, particularly GBM. It possesses the potential to address the limitations of previous treatments and even newer advanced therapies like mAbs, owing to its distinct properties. This review aims to elucidate the current status and advancements in gene therapy for GBM treatment, while also presenting its future prospects.