Alex's Lemonade Stand Foundation for Childhood Cancer

The balanced function of blood stem cells ensures normal production of blood cells. Individuals with germline mutations in the RUNX1 gene (RUNX1-FPD), have an increased lifetime risk of developing leukemias. Leukemia is a state of imbalance of stem cells that produce genetically identical abnormal cancerous cells instead of diverse normal blood cells. For leukemias to initiate in RUNX1-FPD, additional genetic abnormalities are required.

Background

Neuroblastoma is among the most common and deadly cancers of childhood. While some improvement has been made in the treatment of high-risk neuroblastoma through the escalation of toxic high-dose chemotherapy and immunotherapy, the overall prognosis for these patients is still poor. Alternative approaches using targeted therapy are critically needed.