Clinical Trials for Children's Brain Cancers

Neuroblastoma is a rare type of cancer that almost always occurs in infants and young children. One in 7,000 kids will be diagnosed with neuroblastoma each year in the U.S. A related rare pediatric cancer is medulloblastoma, which accounts for 15 to 20 percent of all pediatric brain tumors.

Our pediatric oncology research program oversees the Beat Childhood Cancer consortium, which includes 33 hospitals across the United States. Within the consortium, we enrolled 203 children on our clinical trials in 2017. Together we successfully completed a pilot study of children with high-risk Neuroblastoma using precision medicine at diagnosis combined with standard of care and the addition of DFMO as maintenance therapy. This study showed the safety and feasibility of this approach and was approved to move forward in a Phase II clinical trial.

The trials are based on the research from a group of closely collaborating investigators who are developing new therapies for these high-risk children's cancers.