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Can mouse models for brain tumors inform treatment in pediatric patients?

机译:小鼠脑肿瘤模型能否为小儿患者提供治疗方法?

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Brain tumors represent the most common solid tumor of childhood. Although the histology of many pediatric brain tumors is similar to that of their adult counterparts, significant differences exist with regard to tumor location and response to therapy. The biological and genetic basis for this difference is poorly understood, as tumor tissue is generally unavailable for such studies. While targeted therapies directed against specific molecules active in cancer represents a new arsenal of agents for treating these tumors, such agents are generally not being developed for pediatric cancer in particular. Therefore, new agents for treatment of pediatric glioma must be obtained from compounds being tested against tumors of comparable histology in adult patients. Compounding this problem, although brain tumors are among the most lethal tumors of childhood, their absolute number is relatively small. As a consequence, trials with new agents must be prioritized based on the likelihood that a particular agent or combination of agents will have efficacy in pediatric cancer. Mouse models for brain tumors may help to identify targeted agents, and combinations of agents, effective against these tumors. Such data can be used to prioritize therapies for clinical trials in children with these tumors.
机译:脑肿瘤是儿童期最常见的实体瘤。尽管许多小儿脑肿瘤的组织学与成人肿瘤相似,但是在肿瘤位置和对治疗的反应方面存在显着差异。人们对这种差异的生物学和遗传基础知之甚少,因为肿瘤组织通常无法用于此类研究。尽管针对在癌症中具有活性的特定分子的靶向疗法代表了一种用于治疗这些肿瘤的新型药物,但这种药物通常并未针对儿童癌症进行开发。因此,必须从针对成年人组织学可比的肿瘤进行测试的化合物中获得治疗小儿神经胶质瘤的新药物。使这个问题更加复杂的是,尽管脑肿瘤是儿童期最致命的肿瘤之一,但其绝对数量却相对较小。因此,必须基于特定药物或多种药物组合在小儿癌症中具有疗效的可能性,对使用新药物的试验进行优先排序。脑肿瘤的小鼠模型可能有助于识别针对这些肿瘤有效的靶向药物和药物组合。此类数据可用于对患有这些肿瘤的儿童进行临床试验的治疗优先次序。

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