Alex's Lemonade Stand Foundation for Childhood Cancer

Childhood cancers are caused by genetic mutations that lead to changes in proteins that are essential for normal human development. The protein MYCN is the cause of many of the most aggressive pediatric cancers, including high-risk neuroblastoma and medulloblastoma. One of the holy grails of pediatric cancer drug development is finding a way to directly target MYCN in patient tumors, but this has been elusive. To address this major unmet need, we have developed an international team of scientists with complementary expertise to attack this problem with innovative new technologies.

Inhibition of Cathepsins to Treat Neuroblastoma Neuroblastoma is a common pediatric cancer that is characterized by abnormal continuous proliferation of neural crest cells late in development. In most infants, this abnormal proliferation spontaneously regresses and clinical management is restricted to surgical removal of large tumors and observation of disease regression. This is particularly striking for stage 4S where even multiple secondary tumors regress.