Discovery and development of clofarabine: A nucleoside analogue for treating cancer

Peter L. Bonate, Larry Arthaud, William R. Cantrell, Katherine Stephenson, John A. Secrist, Steve Weitman

Research output: Contribution to journalReview articlepeer-review

198 Scopus citations


The treatment of acute leukaemias, which are the most common paediatric cancers, has improved considerably in recent decades, with complete response rates approaching ∼90% in some cases. However, there remains a major need for treatments for patients who do not achieve or maintain complete remission, for whom the prognosis is very poor. In this article, we describe the challenges involved in the discovery and development of clofarabine, a second-generation nucleoside analogue that received accelerated approval from the US FDA at the end of 2004 for the treatment of paediatric patients 1-21 years old with relapsed or refractory acute lymphoblastic leukaemia after at least two prior regimens. It is the first such drug to be approved for paediatric leukaemia in more than a decade, and the first to receive approval for paediatric use before adult use.

Original languageEnglish (US)
Pages (from-to)855-863
Number of pages9
JournalNature Reviews Drug Discovery
Issue number10
StatePublished - Oct 2006
Externally publishedYes

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery


Dive into the research topics of 'Discovery and development of clofarabine: A nucleoside analogue for treating cancer'. Together they form a unique fingerprint.

Cite this