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正在开发的髓母细胞瘤药物:现有先导物、临床试验和缺陷。

Medulloblastoma drugs in development: Current leads, trials and drawbacks.

机构信息

Department of Pharmaceutical Sciences, University of Connecticut, 69 North Eagleville Rd, Unit 3092, Storrs, CT, 06029-3092, United States.

Department of Pharmaceutical Sciences, University of Connecticut, 69 North Eagleville Rd, Unit 3092, Storrs, CT, 06029-3092, United States.

出版信息

Eur J Med Chem. 2021 Apr 5;215:113268. doi: 10.1016/j.ejmech.2021.113268. Epub 2021 Feb 8.

Abstract

Medulloblastoma (MB) is the most common malignant brain tumor in children. Current treatment for MB includes surgical resection, radiotherapy and chemotherapy. Despite significant progress in its management, a portion of children relapse and tumor recurrence carries a poor prognosis. Based on their molecular and clinical characteristics, MB patients are clinically classified into four groups: Wnt, Hh, Group 3, and Group 4. With our increased understanding of relevant molecular pathways disrupted in MB, the development of targeted therapies for MB has also increased. Targeted drugs have shown unique privileges over traditional cytotoxic therapies in balancing efficacy and toxicity, with many of them approved and widely used clinically. The aim of this review is to present the recent progress on targeted chemotherapies for the treatment of all classes of MB.

摘要

髓母细胞瘤(MB)是儿童中最常见的恶性脑肿瘤。目前 MB 的治疗包括手术切除、放疗和化疗。尽管在其治疗方面取得了显著进展,但部分儿童会复发,肿瘤复发预后不良。根据其分子和临床特征,MB 患者在临床上分为四组:Wnt、Hh、Group 3 和 Group 4。随着我们对 MB 中相关分子途径的了解不断增加,MB 的靶向治疗也在不断发展。与传统的细胞毒性疗法相比,靶向药物在平衡疗效和毒性方面具有独特的优势,其中许多药物已被批准并广泛应用于临床。本综述旨在介绍目前针对所有类型 MB 的靶向化疗的最新进展。

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