Alex's Lemonade Stand Foundation for Childhood Cancer

Neuroblastoma is the most frequent extra-cranial solid tumor in children, accounting for almost 10% of all childhood cancers and close to 15% of childhood cancer deaths. Amplification of the MYCN gene occurs in up to 40% of neuroblastomas, which results in very high levels of MYCN protein and is a marker of very poor prognosis. Current treatment strategies for MYCN amplified tumors are ineffective and in desperate need of new therapeutic avenues. Successful disruption of MYCN function in neuroblastoma would be an exciting therapeutic breakthrough and would save lives.

Germ cell tumors arise from the tissues that make up the reproductive tract, and although rare, cause significant morbidity and mortality in the pediatric age group. This proposal will investigate the role of a gene called Lin-28 in germ cell tumors. Recently, we have discovered that Lin-28 blocks the production of an important class of regulators in the cell called let-7 microRNAs, which are "tumor-suppressors" that protect against cancers of the breast, lung, and colon.