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Mol Cancer Ther. 2013 Dec;12(12):2619-27. doi: 10.1158/1535-7163.MCT-13-0335. Epub 2013 Nov 20.

Is Wilms tumor a candidate neoplasia for treatment with WNT/β-catenin pathway modulators?--A report from the renal tumors biology-driven drug development workshop.

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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.


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.

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