Alex's Lemonade Stand Foundation for Childhood Cancer

Pediatric cancer remains the second leading cause of death in children. One type of cancer that is particularly difficult to treat is FLT3 mutant acute myeloid leukemia (AML). AML cells that have a FLT3 mutation grow at a rapid rate and develop strategies to escape monitoring by the immune system, including blocking natural killer (NK) cell anti-tumor activity. We also know that many cancer patients have low NK cell numbers and decreased NK cell activity as a result of chemotherapy.

Osteosarcoma is a bone cancer that affects children, adolescents, and young adults. Thanks to modern treatment approaches, nearly 7 out of 10 young people diagnosed with osteosarcoma survive into adulthood.

Lay Summary: Pediatric leukemia remains the second leading cause of cancer death in children. While outcomes for newly diagnosed patients have dramatically improved in the last several decades, response rates for relapsed ALL remain <50%. Chimeric antigen receptor (CAR) T cells are immune cells that are taken out of the body and genetically changed to attack cancer cells. CAR T cells are typically made from the patient’s own immune cells.

Background

Ewing’s sarcoma is an aggressive pediatric bone tumor characterized by the EWS/FLI fusion gene. While advances in multimodal therapy have raised disease-free survival rates above 70% for patients with localized disease, survival rates for in metastatic and refractory cases remain stuck below 30%. We need better therapeutic strategies for these patients. EWS/FLI remains challenging to treat with drugs directly and sequencing of patient tumors failed to identify additional drug-treatable mutations common across patients.