Antitumor activity of nifurtimox observed in a patient with neuroblastoma

J Pediatr Hematol Oncol. 2006 Oct;28(10):693-5. doi: 10.1097/01.mph.0000212994.56812.f2.

Abstract

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.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Cell Survival / drug effects
  • Chagas Disease / complications
  • Chagas Disease / diagnosis
  • Chagas Disease / drug therapy*
  • Child, Preschool
  • Cyclophosphamide / administration & dosage
  • Disease Progression
  • Dose-Response Relationship, Drug
  • Drug Screening Assays, Antitumor
  • Female
  • Humans
  • Neuroblastoma / complications
  • Neuroblastoma / diagnosis
  • Neuroblastoma / drug therapy*
  • Nifurtimox / administration & dosage
  • Nifurtimox / pharmacology
  • Nifurtimox / therapeutic use*
  • Recurrence
  • Remission Induction
  • Tomography, X-Ray Computed / methods
  • Topotecan / administration & dosage
  • Treatment Outcome

Substances

  • Topotecan
  • Cyclophosphamide
  • Nifurtimox