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Preclinical Models of Pediatric Brain Tumors—Forging Ahead

机译：小儿脑肿瘤的临床前模型—向前迈进

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摘要

Approximately five out of 100,000 children from 0 to 19 years old are diagnosed with a brain tumor. These children are treated with medication designed for adults that are highly toxic to a developing brain. Those that survive are at high risk for a lifetime of limited physical, psychological, and cognitive abilities. Despite much effort, not one drug exists that was designed specifically for pediatric patients. Stagnant government funding and the lack of economic incentives for the pharmaceutical industry greatly limits preclinical research and the development of clinically applicable pediatric brain tumor models. As more data are collected, the recognition of disease sub-groups based on molecular heterogeneity increases the need for designing specific models suitable for predictive drug screening. To overcome these challenges, preclinical approaches will need continual enhancement. In this review, we examine the advantages and shortcomings of in vitro and in vivo preclinical pediatric brain tumor models and explore potential solutions based on past, present, and future strategies for improving their clinical relevancy.
机译：在100,000名0至19岁的儿童中，大约有五名被诊断出患有脑瘤。这些儿童接受了针对成年人设计的药物治疗，该药物对正在发育的大脑具有高度毒性。那些存活下来的人在一生有限的身体，心理和认知能力中处于高风险。尽管付出了很多努力，但仍没有一种专门为儿科患者设计的药物。政府资金的停滞和制药行业缺乏经济激励措施极大地限制了临床前研究和临床上适用的小儿脑肿瘤模型的开发。随着收集到更多数据，基于分子异质性的疾病亚组识别增加了对设计适用于预测药物筛选的特定模型的需求。为了克服这些挑战，临床前方法将需要不断增强。在这篇综述中，我们研究了体外和体内临床前儿科脑肿瘤模型的优缺点，并根据过去，现在和未来的策略探讨了改善其临床相关性的潜在解决方案。

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