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A pill designed to zero in on abnormal genes that drive specific cancers has produced encouraging early results in children with an uncommon but aggressive type of lymphoma, as well as in children with a rare form of neuroblastoma. A phase 1 clinical trial of the drug crizotinib achieved remissions, with minimal side effects, for 10 of the children participating in a clinical study carried out by the multicenter Children’s Oncology Group (COG). The results were “an exciting proof-of-principle” for the targeted treatment, said the study leader, Yaël P. Mossé, M.D., a pediatric oncologist at The Children’s Hospital of Philadelphia. “We are entering a new era of cancer therapy, in which we use knowledge of basic biology to design very specific drugs that target cancer cells with potentially less side effects on healthy tissue,” said Mossé. “In addition, as we concentrate on targets in molecular pathways, we move away from an exclusive focus on one form of cancer to customizing treatments according to biological activity. Abnormal ALK activity occurs in subtypes of neuroblastoma and subtypes of lymphoma, so identifying ALK activity in individual patients may enable us to provide the most effective care.” To view Multimedia News Release, go to http://www.multivu.com/mnr/50776-childrens-hospital-philadelphia-lymphoma-neuroblastoma-oncology-gene-trial

Researchers have identified the first gene mutation associated with a chronic and often fatal form of neuroblastoma that typically strikes adolescents and young adults. The finding provides the first clue about the genetic basis of the long-recognized but poorly understood link between treatment outcome and age at diagnosis. The study involved 104 infants, children and young adults with advanced neuroblastoma, a cancer of the sympathetic nervous system. Investigators discovered the ATRX gene was mutated only in patients age 5 and older. The alterations occurred most often in patients age 12 and older. These older patients were also more likely than their younger counterparts to have a chronic form of neuroblastoma and die years after their disease is diagnosed. To view Multimedia News Release, go to http://www.multivu.com/mnr/52992-st-jude-pediatric-cancer-genome-project-neuroblastoma-research

Cancer takes more children’s lives than any other disease in the U.S., and nearly 25,000 kids will be diagnosed during the month of September alone. As the largest private funder of childhood cancer research grants, the St. Baldrick’s Foundation is on a mission to take childhood back from cancer by raising critical dollars for lifesaving research and spreading awareness about the harsh realities of childhood cancer. This September, in recognition of Childhood Cancer Awareness Month, St. Baldrick’s will show how the research it funds is accelerating the development of new therapies and cures for kids with cancer everywhere. The Foundation will also share stories of kids affected by cancer – kids like David and Hazel who were both diagnosed with neuroblastoma, but faced two different outcomes. To view the multimedia release go to: https://www.multivu.com/players/English/7916553-st-baldricks-childhood-cancer-awareness-month/