Alex's Lemonade Stand Foundation for Childhood Cancer

Background

The most common pediatric soft tissue sarcoma, representing 3-5% of all childhood cancers, is Rhabdomyosarcoma (RMS). The most aggressive form, Alveolar RMS (ARMS), is associated with metastasis. Approximately 14% of children with RMS have metastatic disease at the time of initial diagnosis. The outcome for patients with metastatic or recurrent disease remains dismal with standard treatment: an estimated five year survival of <30%.

In many blood cancers, allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents the only treatment with the potential for cure. Graft-versus-host disease (GVHD), however afflict between 30-80% of patients receiving allo-HSCT and remains a major cause of death. The primary cause of GVHD is known to be donor's immune cells, which can recognize recipient's tissues as foreign and attack indiscriminately, often targeting both cancer cells and normal tissues.

The past decades have witnessed major progress in the treatment of childhood cancers. However, the development of more effective therapy is delayed by our limited understanding of how these cancers develop. The p53 gene is mutated in many human cancers, including some of the types with the poorest survival rates in children, such as blood, brain and connective tissue cancers.

Background

Neuroblastoma is the most frequently diagnosed cancer in infancy, comprises more than 7% of cancers in patients younger than 15 years of age, and is responsible for 15% of all childhood cancer deaths. There remains a need for novel treatment strategies to eliminate residual disease resistant to current therapies. We will study if natural killer (NK) cells, an important cell in our immune system, and the immune genes responsible for their behavior play a role in the control and eradication of neuroblastoma.