New approaches to drug development in pediatric oncology
New approaches to drug development in pediatric oncology
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DOI：
10.1097/00130404-200507000-00008
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发表时间：
2005-07-01
期刊：
影响因子：
2.2
通讯作者：
Blaney, SM
中科院分区：
文献类型：
--
作者：
Adamson, PC;Blaney, SM
本文回顾了治疗儿童癌症的新药开发范式的变化。尽管在过去的50年里，儿童癌症的治疗和结果有了很大的改善，但在一些癌症中，只取得了有限的进展。需要新的策略来改善预后不良的肿瘤的结果，并将与治疗相关的长期后遗症降至最低。这些战略包括一项针对儿童恶性肿瘤的抗癌药物临床前测试的新的儿科倡议，以及正在进行的儿童肿瘤分子异质性研究，以促进分子靶向药物的评估。此外，继续调查患者间药物暴露和反应变异的遗传基础是优先事项。这些倡议的目标是改善儿童癌症的总体结果，同时将与当前治疗策略相关的短期和长期后遗症降至最低。
This article reviews the changing paradigm of new drug development for childhood cancer. Although there have been dramatic improvements in the treatment and outcome for childhood cancer over the past 50 years, in some cancers, only limited progress has been made. New strategies to improve the outcome for poor-prognosis tumors and to minimize the long-term sequelae associated with therapy are required. Such strategies include a new pediatric-specific initiative for the preclinical testing of anticancer drugs for childhood malignancies, and ongoing research into the molecular heterogeneity of childhood tumors to facilitate the evaluation of molecularly targeted drugs. In addition, continued investigation into the genetic basis underlying interpatient variability in drug exposure and response is a priority. The goal of these initiatives is to improve the overall outcome for childhood cancer while minimizing the short- and long-term sequelae associated with current treatment strategies.