Experimental models reveal possible treatment for alveolar rhabdomyosarcoma

Alveolar rhabdomyosarcoma, a solid tumor, is driven by the abnormal fusion of two genes: FOXO1 and either PAX3 or PAX7. Difficulty targeting the resulting fusion proteins combined with a scarcity of models to study the cancer has limited treatment options. In a study published in Cell, Elizabeth Stewart, MD, St. Jude Department of Oncology and researchers from the New York University School of Medicine addressed this problem by combining insights from two independently created models of alveolar rhabdomyosarcoma. An immature muscle cell model that mimicked the disease’s molecular and genetic makeup was used to show that leucine metabolism is a key factor in tumor growth. A patient-derived 2D/3D cell model was then used to test inhibitors targeting leucine metabolism, with promising results.

“Building collaborations such as this with shared expertise allows us to use our model systems to validate each other’s findings.” said co-corresponding author Stewart. “Patients diagnosed with this disease face an uphill battle, but the more we work together, the faster we can get those combinations of therapies to patients that need it the most.”

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