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Massachusetts General Hospital

55 Fruit Street
Boston, MA 2114
United States

This project will uncover genetic pathways and FDA approved drugs that have novel anti-tumor activity in a rare, pediatric malignancy of muscle -- embryonal rhabdomyosarcoma (ERMS). Rhabdomyosarcoma effects over 250 patients annually in the United States, of which ERMS is the most common subtype. Our work has already identified a key genetic pathway involved in ERMS tumor growth, the RAS pathway.

Background


The pediatric kidney cancer, Wilms tumor, is the fourth most common type of cancer in children. Wilms tumor seems to arise because of abnormalities in the formation of the kidneys from primitive cells in the embryo. The most studied genes mutated in Wilms tumor include WT1 and beta-catenin, which are essential for controlling the development of normal kidneys and in controlling how cells divide. However, these genes are only mutated in a small percentage of Wilms tumors.

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