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Malignant rhabdoid tumor (MRT) and atypical teratoid/rhabdoid tumor (AT/RT) are rare but devastating childhood cancers. Most children diagnosed with MRT and AT/RT are under the age of two, and most will die from their disease despite intensive treatment interventions. New insights into what causes these cancers, and new therapies, are desperately needed. Genetically, MRT and AT/RT are simple cancers, caused by loss of just one gene called SMARCB1. If we are to understand and treat MRT and AT/RT, therefore, we need to understand how loss of SMARCB1 causes cancer.

As the recipient of the Oh Danny Boy I Love You So: The Danny O'Brien Rhabdoid Tumor Research Fund St. Baldrick's Research Grant, Dr. Tansey is working on an innovative molecular mechanism through which loss of SMARCB1 causes MRT and AT/RT. He proposes that these mutations drive cancer by stimulating the activity of a known pro-tumorigenic gene called MYC. Dr. Tansey further proposes that MRT and AT/RT can be effectively treated by drugs that block the actions of MYC, currently being developed by us and others. He is testing this model and exploring its therapeutic implications. Completion of this work has the potential to transform the understanding of how MRT and AT/RT form and how they can be treated. Danny O’Brien was five months old when he was diagnosed with a rare malignant rhabdoid tumor on his liver. Despite treatment to shrink the tumor, he passed away at the tender age of 9 months. Fortunately, he knew nothing but love and affection for all of his short life. This fund honors Danny’s courage and remembers his unconditional love in the midst of his battle with cancer.