Antitumor activity of nifurtimox observed in a patient with neuroblastoma.
BACKGROUND
Chemotherapy-resistant neuroblastoma is a difficult disease to treat with poor survival.


METHODS
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.
Saulnier Sholler
Giselle L
GL
Hasbro Children's Hospital, Lifespan, Providence, RI, USA. Giselle.sholler@uvm.edu
Kalkunte
Satyan
S
Greenlaw
Carla
C
McCarten
Kathleen
K
Forman
Edwin
E
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D045744:Cell Line, Tumor; D049109:Cell Proliferation / Q000187:drug effects; D002470:Cell Survival / Q000187:drug effects; D014355:Chagas Disease / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D002675:Child, Preschool; D003520:Cyclophosphamide / Q000008:administration & dosage; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D004354:Drug Screening Assays, Antitumor; D005260:Female; D006801:Humans; D009447:Neuroblastoma / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D009547:Nifurtimox / Q000008:administration & dosage / Q000494:pharmacology / Q000627:therapeutic use*; D012008:Recurrence; D012074:Remission Induction; D014057:Tomography, X-Ray Computed / Q000379:methods; D019772:Topotecan / Q000008:administration & dosage; D016896:Treatment Outcome
D019772:Topotecan; D003520:Cyclophosphamide; D009547:Nifurtimox