Clohessy John G, Pandolfi Pier Paolo
Preclinical Murine Pharmacogenetics Facility and Mouse Hospital, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, United States.
Cancer Research Institute, Beth Israel Deaconess Cancer Center, Department of Medicine and Pathology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, MA, United States.
Front Oncol. 2018 Aug 28;8:340. doi: 10.3389/fonc.2018.00340. eCollection 2018.
Precision medicine holds real promise for the treatment of cancer. Adapting therapeutic strategies so patients receive individualized treatment protocols, will transform how diseases like cancer are managed. Already, molecular profiling technologies have provided unprecedented capacity to characterize tumors, yet the ability to translate this to actionable outcome in the clinic is limited. To enable real time translation of personalized therapeutic approaches to patient care in a co-clinical manner will require the adoption and integration of approaches that facilitate modeling of patient disease. The Mouse Hospital represents an approach that is ideally suited to pre- and co-clinical evaluation of novel therapeutic strategies for clinical care. Patient derived xenograft (PDX) technologies and tumor modeling approaches using genetically engineered mouse models (GEMMs) already have a proven capacity to mimic human tumor responses, and their application can deliver invaluable insights into appropriate clinical approaches for individual patients by mirroring human clinical trials using a Co-Clinical Trial project and Mouse Hospital infrastructure. Additionally, the integration of the Mouse Hospital with other emerging technologies for the application of precision medicines, including organoid technologies, provides a platform that enables medical centers to truly reap the benefits that precision medicine has to offer.
精准医学在癌症治疗方面有着切实的前景。调整治疗策略,让患者接受个性化治疗方案,将改变癌症等疾病的管理方式。目前,分子分析技术已经具备了前所未有的肿瘤表征能力,然而将其转化为临床上可采取行动的结果的能力却很有限。要以联合临床的方式将个性化治疗方法实时转化为患者护理,就需要采用并整合有助于对患者疾病进行建模的方法。小鼠医院代表了一种非常适合对临床护理新治疗策略进行临床前和联合临床评估的方法。患者来源的异种移植(PDX)技术以及使用基因工程小鼠模型(GEMM)的肿瘤建模方法已经被证明有能力模拟人类肿瘤反应,通过利用联合临床试验项目和小鼠医院基础设施来模拟人类临床试验,它们的应用可以为个体患者的适当临床方法提供宝贵的见解。此外,将小鼠医院与包括类器官技术在内的其他新兴精准医学应用技术相结合,提供了一个平台,使医疗中心能够真正收获精准医学所带来的益处。