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高级别脑胶质瘤的基因治疗进展:临床证据综述。

Advances in gene therapy for high-grade glioma: a review of the clinical evidence.

机构信息

Department of Neurosurgery, Houston Methodist Hospital, Houston, TX, USA.

Kenneth R. Peak Center for Brain and Pituitary Tumor Treatment and Research, Houston Methodist Hospital, Houston, TX, USA.

出版信息

Expert Rev Neurother. 2024 Sep;24(9):879-895. doi: 10.1080/14737175.2024.2376847. Epub 2024 Aug 1.

Abstract

INTRODUCTION

High-grade glioma (HGG) is one of the most deadly and difficult cancers to treat. Despite intense research efforts, there has not been a significant breakthrough in treatment outcomes since the early 2000's. Anti-glioma gene therapy has demonstrated promise in preclinical studies and is under investigation in numerous clinical trials.

AREAS COVERED

This manuscript reviews the current landscape of clinical trials exploring gene therapy treatment of HGG. Using information from clinicaltrials.gov, all trials initiated within the past 5 years (2018-2023) as well as other important trials were cataloged and reviewed. This review discusses trial details, innovative methodologies, and concurrent pharmacological interventions. The review also delves into the subtypes of gene therapy used, trends over time, and future directions.

EXPERT OPINION

Trials are in the early stages (phase I or II), and there are reports of clinical efficacy in published results. Synergistic effects utilizing immunotherapy within or alongside gene therapy are emerging as a promising avenue for future breakthroughs. Considerable heterogeneity exists across trials concerning administration route, vector selection, drug combinations, and intervention timing. Earlier intervention in newly diagnosed HGG and avoidance of corticosteroids may improve efficacy in future trials. The results from ongoing trials demonstrate promising potential for molding the future landscape of HGG care.

摘要

简介

高级别神经胶质瘤(HGG)是最难治疗且致死率最高的癌症之一。尽管研究工作十分密集,但自 21 世纪初以来,治疗效果并没有取得重大突破。抗神经胶质瘤基因治疗在临床前研究中表现出了潜力,目前正在许多临床试验中进行研究。

涵盖领域

本文综述了目前正在探索基因治疗治疗 HGG 的临床试验现状。使用来自 clinicaltrials.gov 的信息,对过去 5 年内(2018-2023 年)启动的所有试验以及其他重要试验进行了编目和回顾。本综述讨论了试验细节、创新方法和同时进行的药理学干预。该综述还深入探讨了所使用的基因治疗类型、随时间的趋势和未来方向。

专家意见

试验处于早期阶段(I 期或 II 期),并且在已发表的结果中有临床疗效的报告。免疫疗法与基因治疗联合或联合使用,作为未来突破的一个有前途的途径正在出现。关于给药途径、载体选择、药物组合和干预时机,试验之间存在相当大的异质性。在新诊断的 HGG 中尽早干预并避免使用皮质类固醇可能会提高未来试验的疗效。正在进行的试验结果表明,有希望塑造 HGG 治疗的未来格局。

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