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One in 5 children with cancer will not survive. We propose to address gaps in basic and translational research by improving model systems for pediatric diseases. To this end, we have developed a Legacy Gift (research autopsy) program called the Cancer Registry for Familial and Sporadic Tumors (CUREfast) to enable parents of children with cancer to donate tumor tissue to the research community. It may seem unusual to think that innovation can come from leftover surgical tissue or a research autopsy – but we believe it can be field-changing, especially for rare childhood cancers for which cell lines and mouse models do not exist, and for which functional studies have never before been performed.
We have collected Legacy Gifts and advanced disease surgical material from over 500 patients across the country, resulting in 19 patient-derived xenograft (PDX) models created in collaboration with the Jackson Laboratory including four “first-in-world” disease models: epithelioid sarcoma, PAX7:FOXO1+ alveolar rhabdomyosarcoma, and parameningeal rhabdomyosarcoma.
These autopsies still represent a small sample of the children lost to cancer every year, and thus a tragically lost opportunity for the research community that faces limited availability of pediatric models. To improve pediatric cancer model systems, we propose to study the genetics, make a cell culture and make a PDX mouse model for each child’s cancer in honor of their life – and to the benefit of future children.