Antitumor activity of nifurtimox observed in a patient with neuroblastoma.

BACKGROUND: Chemotherapy-resistant neuroblastoma is a difficult disease to treat with poor survival. OBSERVATIONS: We treated a patient with neuroblastoma who had progressed on conventional chemotherapy. This 5-year-old girl with chemotherapy-resistant neuroblastoma developed Chagas disease at the start of salvage chemotherapy for which she was also started on nifurtimox. The neuroblastoma response to these treatments resulted in clinical remission. In vitro, treatment of a neuroblastoma cell line with nifurtimox resulted in decreased cell viability whereas no effect was seen on an endothelial cell line. CONCLUSIONS: Nifurtimox shows promise as a potential new treatment for neuroblastoma and warrants further testing.