Cladribine: Not just another purine analogue?

Stephen Spurgeon, Margaret Yu, John D. Phillips, Elliot M. Epner

Research output: Contribution to journalReview articlepeer-review

51 Scopus citations


Cladribine was synthesized as a purine analogue drug that inhibited adenosine deaminase. It received FDA approval in the 1980s for treatment of hairy cell leukemia. Given its toxicity towards lymphocytes and its corresponding immunosuppressive effects, it has been studied and found efficacious in a variety of hematologic malignancies and autoimmune conditions, most recently multiple sclerosis. This review highlights pharmacological, toxicological and clinical data for the use of cladribine. It also discusses existing and new mechanisms that may contribute to its unique clinical activity. Emerging data show that in addition to its known purine nucleoside analogue activity, cladribine possesses epigenetic properties, inhibiting S-adenosylhomocysteine hydrolase and DNA methylation. This may contribute to its efficacy and highlights the importance of studying combination therapy with other epigenetic or targeted agents. Clinical trials are underway in a variety of malignant and nonmalignant conditions.

Original languageEnglish (US)
Pages (from-to)1169-1181
Number of pages13
JournalExpert Opinion on Investigational Drugs
Issue number8
StatePublished - Aug 2009


  • B-cell malignancies
  • Cladribine
  • Epigenetics
  • Hypomethylation
  • Multiple sclerosis
  • SAH hydrolase

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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