Alex's Lemonade Stand Foundation for Childhood Cancer

Ewing sarcoma is an aggressive cancer in children, teenagers, and young adults. Treatment involves intensive chemotherapy, surgery, and/or radiation, including disfiguring surgeries. Many survivors experience long-term side effects. Despite aggressive treatment, the disease is cured in only about 50% of patients, highlighting the urgent need for better, less toxic therapies. One of the biggest challenges in developing new treatments is that we still don’t fully understand what causes Ewing sarcoma at the molecular level.

Rhabdomyosarcoma is the most common soft tissue sarcoma in children and has no targeted treatment options. The most aggressive forms of rhabdomyosarcoma are caused when pieces of two chromosomes break off and fuse together and create an abnormal protein called PAX3-FOXO1. This new protein turns on and off hundreds–if not thousands–of genes, transforming a normal cell into a cancer cell. The exact mechanism of how PAX3-FOXO1 accomplishes this is unknown and insight into this process is hindered by the lack of models.