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

IF 3.4 2区 医学 Q2 CLINICAL NEUROLOGY Expert Review of Neurotherapeutics Pub Date : 2024-09-01 Epub Date: 2024-08-01 DOI:10.1080/14737175.2024.2376847
Matthew J Goldman, Alexandra M Baskin, Martyn A Sharpe, David S Baskin
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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.

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高级别胶质瘤基因治疗的进展:临床证据综述。
简介高级别胶质瘤(HGG)是最致命、最难治疗的癌症之一。尽管开展了大量研究工作,但自 2000 年代初以来,治疗效果一直没有重大突破。抗胶质瘤基因疗法已在临床前研究中显示出前景,并正在许多临床试验中进行研究:本手稿回顾了目前探索基因疗法治疗 HGG 的临床试验情况。利用 clinicaltrials.gov 提供的信息,对过去 5 年(2018-2023 年)内启动的所有试验以及其他重要试验进行了编目和综述。本综述讨论了试验细节、创新方法和同时进行的药物干预。综述还深入探讨了所用基因疗法的亚型、长期趋势和未来方向:试验处于早期阶段(I 期或 II 期),已发表的结果中有临床疗效报告。在基因疗法中或同时利用免疫疗法的协同效应正在成为未来有望取得突破的途径。不同试验在给药途径、载体选择、药物组合和干预时机等方面存在很大差异。在未来的试验中,对新诊断的 HGG 尽早进行干预并避免使用皮质类固醇可能会提高疗效。正在进行的试验结果表明,未来的HGG治疗前景广阔。
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来源期刊
Expert Review of Neurotherapeutics
Expert Review of Neurotherapeutics Medicine-Neurology (clinical)
CiteScore
7.00
自引率
2.30%
发文量
61
审稿时长
4-8 weeks
期刊介绍: Expert Review of Neurotherapeutics (ISSN 1473-7175) provides expert reviews on the use of drugs and medicines in clinical neurology and neuropsychiatry. Coverage includes disease management, new medicines and drugs in neurology, therapeutic indications, diagnostics, medical treatment guidelines and neurological diseases such as stroke, epilepsy, Alzheimer''s and Parkinson''s. Comprehensive coverage in each review is complemented by the unique Expert Review format and includes the following sections: Expert Opinion - a personal view of the data presented in the article, a discussion on the developments that are likely to be important in the future, and the avenues of research likely to become exciting as further studies yield more detailed results Article Highlights – an executive summary of the author’s most critical points
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