New findings suggest that children with rhabdomyosarcoma could benefit from tumour genetic testing. Childhood Rhabdomyosarcoma Treatment In children with rhabdomyosarcoma, or RMS, a rare cancer that affects the muscles and other soft tissues, the presence of mutations in several genes, including TP53, MYOD1, and CDKN2A, appears to be associated with a more aggressive form of the disease and a poorer chance of survival. This finding is from the largest-ever international study on RMS, led by scientists at the National Cancer Institute’s (NCI) Center for Cancer Research, part of the National Institutes of Health. The study, published in the Journal of Clinical Oncology on June 24, provides an unprecedented look at data for a large cohort of patients with RMS, offering genetic clues that could lead to more widespread use of tumor genetic testing to predict how individual patients with this childhood cancer will respond to therapy, as well as to the development of targeted treatments for the disease. “These discoveries change what we do with these patients and trigger a lot of really important research into developing new therapies that target these mutations,” said Javed Khan, M.D., of NCI’s Genetics Branch, who led the study. “The standard therapy for RMS is almost a year of chemotherapy, radiation therapy, and surgery. These children get a lot of toxic treatments,” said the study’s first author, Jack Shern, M.D., of NCI’s Pediatric Oncology Branch. “If we could predict who’s going to do well and who’s not, then we can really start to tailor our therapies or eliminate therapies that aren’t going to be effective in a particular patient. And for the children that aren’t going to do well, this allows us to think about new ways to treat them.” What is rhabdomyosarcoma (RMS) RMS is the most common type of soft tissue sarcoma in children. In patients whose cancer has remained localized, meaning that it has not spread, combination chemotherapies have led to a five-year survival rate of 70%-80%. But in patients whose cancer has spread or come back after treatment, the five-year survival rate remains poor at less than 30%, even with…