Optimization of bifunctional pharmacophores for the treatment of neuroblastoma.

DESCRIPTION (provided by applicant): Genetic alterations that maintain cells in a poorly differentiated state are a hallmark of many leukemia and childhood cancers. Among them, neural crest-derived neuroblastoma is the most prevalent cancer in infants andthe most common extracranial solid tumor in children with the majority of patients being 5 years old or younger. Approximately 650 neuroblastoma tumors of the sympathetic nervous system are diagnosed each year in the United States and 10,000 children a year worldwide. Although successfully managed in its milder forms, the 5-year survival rate for patients with high risk neuroblastoma under an aggressive therapeutic regimen is only 40% and is associated with a high morbidity. Safer and more effective treatments are needed to manage this condition. HaRo Pharmaceuticals, Inc. is developing a promising polypharmacological agent that affects two distinct but functionally interconnected cellular pathways, which have been shown to be important for the treatmen