McNerney Kevin O, Teachey David T
Children's Hospital of Philadelphia, Divisions of Hematology and Oncology, Philadelphia, PA 19104, USA.
Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA.
Fac Rev. 2021 Feb 2;10:11. doi: 10.12703/r/10-11. eCollection 2021.
The prognosis for childhood cancer has improved considerably over the past 50 years. This improvement is attributed to well-designed clinical trials which have incorporated chemotherapy, surgery, and radiation. With an increased understanding of cancer biology and genetics, we have entered an era of precision medicine and immunotherapy that provides potential for improved cure rates. However, preclinical evaluation of these therapies is more nuanced, requiring more robust animal models. Evaluation of targeted treatments requires molecularly defined xenograft models that can capture the diversity within pediatric cancer. The development of novel immunotherapies ideally involves the use of animal models that can accurately recapitulate the human immune response. In this review, we provide an overview of xenograft models for childhood cancers, review successful examples of novel therapies translated from xenograft models to the clinic, and describe the modern tools of xenograft biobanks and humanized xenograft models for the study of immunotherapies.
在过去50年里，儿童癌症的预后有了显著改善。这一改善归功于精心设计的临床试验，这些试验结合了化疗、手术和放疗。随着对癌症生物学和遗传学的了解不断增加，我们进入了精准医学和免疫治疗时代，这为提高治愈率提供了潜力。然而，这些疗法的临床前评估更为细致入微，需要更强大的动物模型。对靶向治疗的评估需要分子定义的异种移植模型，这种模型能够捕捉儿童癌症的多样性。新型免疫疗法的开发理想情况下需要使用能够准确重现人类免疫反应的动物模型。在这篇综述中，我们概述了儿童癌症的异种移植模型，回顾了从异种移植模型转化到临床的新型疗法的成功案例，并描述了用于免疫治疗研究的异种移植生物样本库和人源化异种移植模型的现代工具。
