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Nifurtimox Phase I Clinical Trial

Nifurtimox for Refractory or Relapsed Neuroblastoma

Many significant scientific discoveries remain untranslated.  From a 2-page abstract research paper that described an observed case study of an “accidental” therapeutic benefit for neuroblastoma from treating another condition (Chagas Disease), a Phase I Clinical trial was created in three-months, providing a new therapeutic option for many children.

The Nifurtimox Phase I trial was designed to be as therapeutically effective as possible.  The study is an important clinical step for children battling relapsed or refractory disease by adding a combination of known chemotherapeutic agents (cyclophosphamide and topotecan) to the investigational drug (Nifurtimox) to provide more potential clinical benefit for these children than Nifurtimox alone.