Preclinical Models of Pediatric Brain Tumors-Forging Ahead.
Preclinical Models of Pediatric Brain Tumors-Forging Ahead.
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DOI：
10.3390/bioengineering5040081
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发表时间：
2018-10-02
期刊：
影响因子：
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通讯作者：
Gopalakrishnan V
中科院分区：
文献类型：
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作者：
Dobson THW;Gopalakrishnan V
大约每10万名0至19岁的儿童中就有5人被诊断患有脑瘤。这些儿童接受的是专为成人设计的药物治疗，这些药物对发育中的大脑有很高的毒性。那些幸存下来的人一生都面临着身体、心理和认知能力有限的高风险。尽管付出了很多努力，但没有一种药物是专门为儿科患者设计的。停滞的政府资助和缺乏对制药行业的经济激励极大地限制了临床前研究和临床适用的儿科脑肿瘤模型的开发。随着更多数据的收集，基于分子异质性的疾病亚组识别增加了设计适用于预测性药物筛选的特定模型的需求。为了克服这些挑战，临床前方法需要不断改进。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点和缺点，并根据过去、现在和未来的策略探索潜在的解决方案，以提高其临床相关性。
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.