Alex's Lemonade Stand Foundation for Childhood Cancer

Background

Acute lymphoblastic leukemia (ALL) is the most common form of childhood leukemia and the leading cause of death in children with cancer. While therapy is often curative, ~15% of children will relapse with recurrent disease and poor outcomes. Why some children develop resistant disease remains unclear.

Background

Approximately a quarter of children with cancer have tumors of the brain and spinal cord. Unfortunately most children with diffuse intrinsic pontine glioma (DIPG), one type of brain tumor, die within two years of diagnosis. Scientists have not been able to find a cure for this tumor but as science advances, we have slowly gained a better understanding of what makes one tumor different from another and what may be the underlying mechanism that triggers this devastating disease.

Background
Current treatments for patients with relapsed sarcomas are ineffective. The best chemotherapy regimens shrink tumors only 40% of the time, without prolonging survival. Effective treatments for these patients are a critical unmet medical need, and new molecularly targeted therapies represent the most promising approach to this difficult problem.

Background

Certain visible changes in the chromosomes and mutations in the genes correlate with good or bad prognosis in leukemia. FLT3 is one of the most frequently mutated genes in pediatric AML and also plays an important role in Infant ALL. In both types of pediatric leukemias the FLT3 protein acts as a gas pedal that is permanently pressed to the floor, telling cells to grow out of control. In order to send this signal, FLT3 needs to bind to an energy molecule called ATP.

Several common childhood leukemias are now considered curable. However, there are features of leukemia cells which continue to make them difficult to treat. One such feature is having an alteration in a gene called MLL. It is not yet fully understood why MLL alterations cause leukemia. In addition, current therapies do not seem as effective against leukemia with MLL alterations as it is in those without. We hope to better understand MLL in a way that will bring about new therapies for the disease. Our own body can destroy cancer cells with the help of tumor suppressor genes.