Corresponding Author: Kathy Pritchard-Jones, Hugh and Catherine Stevenson Professor of Pediatric Oncology, UCL Institute of Child Health, 30 Guilford Street, London WC1N 1EH, United Kingdom.
Mol Cancer Ther. 2013 Dec;12(12):2619-27. doi: 10.1158/1535-7163.MCT-13-0335. Epub 2013 Nov 20.
The European Network for Cancer Research in Children and Adolescents consortium organized a workshop in Rome, in June 2012, on "Biology-Driven Drug Development Renal Tumors Workshop" to discuss the current knowledge in pediatric renal cancers and to recommend directions for further research. Wilms tumor is the most common renal tumor of childhood and represents a success of pediatric oncology, with cure rates of more than 85% of cases. However, a substantial minority (∼25%) responds poorly to current therapies and requires "high-risk" treatment or relapse. Moreover, the successfully treated majority are vulnerable to the late effects of treatment, with nearly one quarter reporting severe chronic health conditions by 25 years of follow-up. Main purposes of this meeting were to advance our understanding on the molecular drivers in Wilms tumor, their heterogeneity and interdependencies; to provide updates on the clinical-pathologic associations with biomarkers; to identify eligible populations for targeted drugs; and to model opportunities to use preclinical model systems and prioritize targeted agents for early phase clinical trials. At least three different pathways are involved in Wilms tumor; this review represents the outcome of the workshop discussion on the WNT/β-catenin pathway in Wilms tumorigenesis.
欧洲儿童和青少年癌症研究网络于 2012 年 6 月在罗马组织了一次关于“生物学驱动的药物研发肾脏肿瘤研讨会”的研讨会，旨在讨论儿科肾脏癌症的现有知识，并为进一步的研究推荐方向。Wilms 瘤是儿童中最常见的肾脏肿瘤，是儿科肿瘤学的成功范例，其治愈率超过 85%。然而，相当一部分（约 25%）患者对当前的治疗方法反应不佳，需要“高危”治疗或复发。此外，成功治疗的大多数患者易受到治疗的后期影响，近四分之一的患者在 25 年的随访中报告有严重的慢性健康状况。本次会议的主要目的是增进我们对 Wilms 肿瘤中分子驱动因素、其异质性和相互依赖性的理解；提供关于与生物标志物相关的临床病理关联的最新信息；确定适合靶向药物的人群；并建立使用临床前模型系统的机会，并为早期临床试验确定靶向药物的优先级。至少有三种不同的途径参与了 Wilms 肿瘤的发生；这篇综述代表了研讨会关于 Wilms 肿瘤发生中 WNT/β-catenin 途径的讨论结果。
