Alex's Lemonade Stand Foundation for Childhood Cancer

Genetic studies have identified a number of genes that when mutated predispose individuals to blood cancers. In addition, these mutations can promote other health complications throughout their lifetime. For example, children that inherit a mutation in the gene RUNX1 have Familial Platelet Disorder, they live with bleeding problems and, in many cases, autoimmune complications. In addition, affected children have a high incidence of blood cancer during their childhood or later in life. Currently, there are not effective therapies to prevent the development of blood cancer.

Lay Summary: Endemic Burkitt lymphoma (eBL) is the most common childhood cancer in Africa. This cancer is treatable with chemotherapy, but the survival rate for African children is 50%, in contrast to 90% survival for American children. This could be due to two common childhood infections that are considered risk factors for eBL, namely malaria and Epstein­-Barr virus (EBV). A healthy immune system is able to recognize and kill human cells that have become infected or have become cancer cells.

Background

Treatment protocols used in the treatment of pediatric acute myeloid leukemia (AML) are based on chemotherapy drugs, often resulting in side effects due to significant toxicity. In recent years, the emergence of targeted therapy strategies in cancer treatment has enlightened the hope for developing new drugs with higher potency and reduced toxicity in patients.