Utilizing preclinical models to develop targeted therapies for rare central nervous system cancers.
Utilizing preclinical models to develop targeted therapies for rare central nervous system cancers.
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DOI：
10.1093/neuonc/noab183
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发表时间：
2021-11-02
期刊：
影响因子：
15.9
通讯作者：
Holland EC
中科院分区：
文献类型：
--
作者：
Arakaki AKS;Szulzewsky F;Gilbert MR;Gujral TS;Holland EC
患有罕见中枢神经系统（CNS）肿瘤的患者通常预后不良，治疗选择有限。从历史上看，这些癌症由于患者数量少而难以研究。最近的技术进步已经确定了这些罕见癌症的分子驱动因素，我们现在可以用它们来生成这些疾病的代表性临床前模型。在这篇综述中，我们概述了不同模型的优点和缺点，强调了各种体外和离体模型的效用，用于靶点发现和机制研究，以及多种体内模型用于治疗验证。我们还强调了最近的文献室管膜瘤，组蛋白突变的高级别胶质瘤和非典型畸胎瘤样横纹肌样肿瘤的临床前模型生成和筛选方法，所有这些都是最近建立的遗传或表观遗传驱动的罕见CNS癌症。这些临床前模型对于推进目前依赖常规治疗的这些罕见CNS癌症的靶向治疗至关重要。
Patients with rare central nervous system (CNS) tumors typically have a poor prognosis and limited therapeutic options. Historically, these cancers have been difficult to study due to small number of patients. Recent technological advances have identified molecular drivers of some of these rare cancers which we can now use to generate representative preclinical models of these diseases. In this review, we outline the advantages and disadvantages of different models, emphasizing the utility of various in vitro and ex vivo models for target discovery and mechanistic inquiry and multiple in vivo models for therapeutic validation. We also highlight recent literature on preclinical model generation and screening approaches for ependymomas, histone mutated high-grade gliomas, and atypical teratoid rhabdoid tumors, all of which are rare CNS cancers that have recently established genetic or epigenetic drivers. These preclinical models are critical to advancing targeted therapeutics for these rare CNS cancers that currently rely on conventional treatments.
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