Rayati Mohammad, Mansouri Vahid, Ahmadbeigi Naser
Gene Therapy Research Center, Digestive Diseases Research Institute, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran.
Heliyon. 2024 Feb 24;10(5):e27087. doi: 10.1016/j.heliyon.2024.e27087. eCollection 2024 Mar 15.
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.
多形性胶质母细胞瘤（GBM）是最致命的癌症之一，预后较差。自最初发现并进行医学描述后的过去一个世纪里，针对这种疾病的有效治疗方法进展有限。尽管付出了诸多努力，但仅有少数几种药物获批用于其治疗。然而，这些治疗方法在总生存率和无进展生存率方面并未取得实质性改善。原因之一在于其独特特征，如异质性以及由于血脑屏障和肿瘤-血屏障这两个巨大障碍导致的药物递送困难。在过去几年中，治疗方法取得了重大进展，催生了有前景的新型和先进疗法。靶向特异性疗法，如单克隆抗体（mAb）和小分子，就是两个重要例子；然而，它们并未使GBM患者的生存率得到显著提高。基因治疗作为一种相对新兴的先进方法，有望成为癌症尤其是GBM的潜在治疗手段。由于其独特性质，它有潜力解决先前治疗以及诸如mAb等更新的先进疗法的局限性。本综述旨在阐明GBM治疗中基因治疗的现状和进展，同时展示其未来前景。
