Alex's Lemonade Stand Foundation for Childhood Cancer

Background

The LIN28 family of RNA binding proteins are known drivers of many pediatric cancers. These proteins function by blocking the function of the let-7 family of microRNAs, which exert tumor suppressive effects. To date, despite the panoply of childhood tumors driven by LIN28, efforts to target this protein in pediatric cancer are lacking.

Background

Juvenile myelomonocytic leukemia (JMML) is an aggressive blood cancer of young children, typically affecting those less than 4 years of age. The only current curative treatment is bone marrow transplantation. Yet, despite this very aggressive therapy about 50% of children still ultimately die from their disease. Thus, more effective and less toxic treatments are urgently needed.

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.