STI571 as a targeted therapy for CML

Michael E. O'Dwyer, Michael J. Mauro, Brian J. Druker

Research output: Contribution to journalReview articlepeer-review

54 Scopus citations


Chronic myelogenous leukemia (CML) is a clonal hematopoietic stem cell disorder that progresses through distinct phases as the malignant clone progressively loses the capacity for terminal differentiation. It is characterized by the (9;22) translocation and resultant production of the Bcr-Abl tyrosine kinase. Bcr-Abl functions as a constitutively activated tyrosine kinase, and this kinase activity is absolutely required for the transforming function of the Bcr-Abl protein. In preclinical studies, STI571 (Gleevec, imatinib mesylate), a Bcr-Abl tyrosine kinase inhibitor, specifically inhibited the proliferation of Bcr-Abl-expressing cells in vitro and in vivo. STI571 has shown remarkable results in all phases of CML. Although responses are seen in all phases of the disease, durable responses are most common in earlier stage patients. Thus, STI571 has emerged as a paradigm for gene product targeted therapy, offering expanded treatment options for patients with CML.

Original languageEnglish (US)
Pages (from-to)429-438
Number of pages10
JournalCancer Investigation
Issue number3
StatePublished - 2003


  • Bcr-Abl
  • CML
  • Imatinib
  • STI571
  • Tyrosine kinase

ASJC Scopus subject areas

  • Oncology
  • Cancer Research


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