Pediatric Oncology and Hematology Department, Translational Research in Child and Adolescent Cancer, Vall d'Hebron Institut de Recerca (VHIR), Barcelona. Universitat Autònoma de Barcelona, Bellaterra, Spain.
Cancer Bioengineering Group, Department of Anatomy and Regenerative Medicine, RCSI University of Medicine and Health Sciences, Dublin, Ireland.
Expert Opin Drug Discov. 2022 Feb;17(2):167-179. doi: 10.1080/17460441.2022.2002297. Epub 2021 Nov 22.
Neuroblastoma is a cancer of the sympathetic nervous system that causes up to 15% of cancer-related deaths among children. Among the ~1,000 newly diagnosed cases per year in Europe, more than half are classified as high-risk, with a 5-year survival rate <50%. Current multimodal treatments have improved survival among these patients, but relapsed and refractory tumors remain a major therapeutic challenge. A number of new methodologies are paving the way for the development of more effective and safer therapies to ultimately improve outcomes for high-risk patients.
The authors provide a critical review on methodological advances aimed at providing new therapeutic opportunities for neuroblastoma patients, including preclinical models of human disease, generation of omics data to discover new therapeutic targets, and artificial intelligence-based technologies to implement personalized treatments.
While survival of childhood cancer has improved over the past decades, progress has been uneven. Still, survival is dismal for some cancers, including high-risk neuroblastoma. Embracing new technologies (e.g. molecular profiling of tumors, 3D models, etc.), international collaborative efforts and the incorporation of new therapies (e.g. RNA-based therapies, epigenetic therapies, immunotherapy) will ultimately lead to more effective and safer therapies for these subgroups of neuroblastoma patients.
神经母细胞瘤是一种交感神经系统癌症，导致儿童癌症相关死亡人数达到 15%。在欧洲每年约 1000 例新诊断病例中，超过一半被归类为高危，5 年生存率<50%。目前的多模式治疗已经提高了这些患者的生存率，但复发和难治性肿瘤仍然是一个主要的治疗挑战。许多新方法为开发更有效和更安全的疗法铺平了道路，最终改善高危患者的预后。
作者对旨在为神经母细胞瘤患者提供新治疗机会的方法学进展进行了批判性评估，包括人类疾病的临床前模型、产生用于发现新治疗靶点的组学数据以及基于人工智能的技术来实施个性化治疗。
尽管在过去几十年中儿童癌症的生存率有所提高，但进展并不均衡。尽管如此，一些癌症的生存率仍然很低，包括高危神经母细胞瘤。接受新技术（例如肿瘤的分子分析、3D 模型等）、国际合作努力和新疗法的纳入（例如基于 RNA 的疗法、表观遗传学疗法、免疫疗法）最终将为这些神经母细胞瘤亚组患者带来更有效和更安全的疗法。
