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Molecularly Targeted Agents in the Therapy of Pediatric Brain Tumors

机译：分子靶向剂在儿科脑肿瘤的治疗中

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摘要

Pediatric brain tumors are the leading cause of cancer-related death in children. Recent advances in sequencing techniques, and collaborative efforts to encode the mutational landscape of various tumor subtypes, have resulted in the identification of recurrent mutations that may present as actionable targets in these tumors. A number of molecularly targeted agents are approved or in development for the treatment of various tumor types in adult patients. Similarly, these agents are increasingly being incorporated into pediatric clinical trials, allowing for a targeted approach to treatment. However, due to the genetic heterogeneity of these tumors, focused clinical trials in pediatric patients are challenging and regulatory hurdles may delay access to therapeutic compounds that are in regular use in adult patients. The tumor site-agnostic clinical development of TRK inhibitors for pediatric solid tumors is a current example of how the combination of genetic testing and innovative clinical trial design can accelerate the clinical development of targeted agents for pediatric patients.
机译：小儿脑肿瘤是儿童癌症相关死亡的主要原因。测序技术的最新进展以及编码各种肿瘤亚型的突变景观的协作努力导致鉴定可在这些肿瘤中作为可操作的靶标呈现的复发突变。许多分子靶向剂被批准或开发用于治疗成人患者的各种肿瘤类型。类似地，这些药剂越来越纳入儿科临床试验，允许靶向治疗方法。然而，由于这些肿瘤的遗传异质性，小儿患者的重点临床试验是挑战性，监管障碍可能会延迟进入成年患者的定期用途的治疗化合物。儿科实体瘤的Trk抑制剂的肿瘤部位 - 无症状临床开发是遗传检测和创新临床试验设计的结合如何加速对儿科患者靶向剂的临床发展的目的。

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  • 来源
    《Pediatric drugs》 |2020年第1期|共10页
  • 作者

    Bornhorst Miriam; Hwang Eugene I.;

  • 作者单位

    Childrens Natl Med Ctr Dept Pediat Hematol Oncol Ctr Canc &

    Immunol Res &

    Neurosci Res 111;

    Childrens Natl Med Ctr Dept Pediat Hematol Oncol Ctr Canc &

    Immunol Res &

    Neurosci Res 111;

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  • 原文格式 PDF
  • 正文语种 eng
  • 中图分类 药学;
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