A Novel Treatment for Children with Anaplastic Wilms’ Tumor

Already funded by CURE & Rally Foundations!

An unmet clinical need exists for children with Wilms’ tumor that present with anaplastic (unfavorable) histology. While traditional chemotherapeutic treatment for histologically favorable Wilms’ tumor has seen great success, many children presenting with anaplastic disease fail to respond to therapies. Anaplastic Wilms’ tumor has relatively little resource development, basic science or translational research compared to other forms of childhood cancer. We propose to study anaplasia in Wilms’ tumor at a preclinical level to discover why anaplastic disease tends to be resistant to standard therapies and to predict a rational drug combination which might be more effective and less toxic for these children. To this end, we will use a combination of functional genomics, drug screening and gene expression data mining to develop a novel targeted therapy combination that is specific to anaplastic Wilms’ tumor. We will then preclinically validate this novel drug combination in vitro, ex ovo, and in vivo in order to provide a strong rationale for moving this therapeutic strategy into the clinical setting.