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患者来源的临床前模型对加速神经母细胞瘤患儿个性化医疗实施的前景

The Promise of Patient-Derived Preclinical Models to Accelerate the Implementation of Personalised Medicine for Children with Neuroblastoma.

作者信息

Tucker Elizabeth R, George Sally, Angelini Paola, Bruna Alejandra, Chesler Louis

机构信息

Paediatric Tumour Biology, Division of Clinical Studies, The Institute of Cancer Research, Cotswold Road, London SM2 5NG, UK.

Children and Young People's Unit, The Royal Marsden, Downs Road, Sutton, Surrey SM2 5PT, UK.

出版信息

J Pers Med. 2021 Mar 30;11(4):248. doi: 10.3390/jpm11040248.

Abstract

Patient-derived preclinical models are now a core component of cancer research and have the ability to drastically improve the predictive power of preclinical therapeutic studies. However, their development and maintenance can be challenging, time consuming, and expensive. For neuroblastoma, a developmental malignancy of the neural crest, it is possible to establish patient-derived models as xenografts in mice and zebrafish, and as spheroids and organoids in vitro. These varied approaches have contributed to comprehensive packages of preclinical evidence in support of new therapeutics for neuroblastoma. We discuss here the ethical and technical considerations for the creation of patient-derived models of neuroblastoma and how their use can be optimized for the study of tumour evolution and preclinical therapies. We also discuss how neuroblastoma patient-derived models might become avatars for personalised medicine for children with this devastating disease.

摘要

患者来源的临床前模型如今是癌症研究的核心组成部分,能够极大地提高临床前治疗研究的预测能力。然而,其开发和维护可能具有挑战性、耗时且成本高昂。对于神经母细胞瘤这种神经嵴的发育性恶性肿瘤,可以在小鼠和斑马鱼中建立患者来源的异种移植模型,也可以在体外建立球体和类器官模型。这些不同的方法为支持神经母细胞瘤新疗法的临床前证据综合包做出了贡献。我们在此讨论创建神经母细胞瘤患者来源模型的伦理和技术考量,以及如何优化其使用以研究肿瘤演变和临床前治疗。我们还讨论了神经母细胞瘤患者来源模型如何可能成为患有这种毁灭性疾病的儿童个性化医疗的化身。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4da4/8065808/1d873bbfd036/jpm-11-00248-g001.jpg

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