Alex's Lemonade Stand Foundation for Childhood Cancer

High Risk neuroblastoma is the most common abdominal tumor in children and accounts for 15% of all pediatric cancer mortality. This is despite arduous intensive toxic chemotherapy treatments with major long-term side effects. Most deaths result from relapsed neuroblastoma, which grows back after initially responding to therapy. Thus, our major goal is to develop novel treatments to prevent neuroblastoma relapse. This will have a major impact on survival for neuroblastoma (currently less than 40% ).

Fatigue is now recognized as the most frequent symptom experienced by children and adolescents with cancer. As we increase our understanding of fatigue in children and adolescents with cancer, it is important to explore new measurements of this symptom. Carnitine is found in nearly all cells of the body and plays a critical role in energy production. Carnitine deficiencies can cause symptoms such as fatigue, muscle pain and weakness in individuals with cancer. The purpose of this study is to explore carnitine as a physiologic marker for fatigue in 60 children and adolescents with cancer.

The first goal of cancer genetics research is to understand why children develop cancer and to identify if specific families or children have an even higher risk of developing cancer. This information impacts the child’s cancer care and determines who else in the family may need increased cancer surveillance to try and detect cancer at its earliest stage. Many cancer susceptibility genes also play a major role in cancer in the general population. Thus, the larger goal of cancer genetics research is to improve our understanding of cancer cells and to identify new targets for treatment.

I received a grant from Alex’s Lemonade Stand Foundation in 2006, when I had promising data that immune cells could be used against pediatric bone cancers. Thanks to the ALSF funding, we were able to continue to work on this project and the results have been very gratifying.