Cancer and Blood Disease Institute, Children's Hospital Los Angeles, Los Angeles, CA 90027, USA
Dis Model Mech. 2021 Feb 22;14(2):dmm048930. doi: 10.1242/dmm.048930.
In the treatment of children and adolescents with cancer, multimodal approaches combining surgery, chemotherapy and radiation can cure most patients, but may cause lifelong health problems in survivors. Current therapies only modestly reflect increased knowledge about the molecular mechanisms of these cancers. Advances in next-generation sequencing have provided unprecedented cataloging of genetic aberrations in tumors, but understanding how these genetic changes drive cellular transformation, and how they can be effectively targeted, will require multidisciplinary collaboration and preclinical models that are truly representative of the environment. Here, I discuss some of the key challenges in pediatric cancer from my perspective as a physician-scientist, and touch on some promising new approaches that have the potential to transform our understanding of these diseases.
在儿童和青少年癌症的治疗中，结合手术、化疗和放疗的多模式方法可以治愈大多数患者，但可能会给幸存者带来终身健康问题。目前的治疗方法仅适度反映了对这些癌症分子机制的更多了解。下一代测序的进步为肿瘤中的遗传异常提供了前所未有的编目，但要了解这些遗传变化如何驱动细胞转化，以及如何有效地靶向这些变化，需要多学科合作和真正代表肿瘤微环境的临床前模型。在这里，我从医生科学家的角度讨论了一些儿童癌症的关键挑战，并探讨了一些有潜力改变我们对这些疾病认识的有前途的新方法。
