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Where are we going with CML research?

Leukemia Supplements volume 1, pages S51–S53 (2012)Cite this article

Abstract

The introduction of Abl tyrosine kinase inhibitors (TKI; that is, imatinib, dasatinib and nilotinib) as front-line therapy completely changed the course of chronic myelogenous leukemia (CML) to the point that most of the TKI-responsive newly diagnosed CML patients can be considered ‘clinically’ cured and their progression into blast crisis (BC) a rare event. However, a therapy for those patients who transform is still lacking, and TKIs do not eradicate CML at the stem cell level, therefore leaving a reservoir of cancer stem cells in a dormant stage. Thus, it is not surprising that the focus of CML research has shifted significantly toward the dissection of the mechanisms regulating the survival and self-renewal of TKI-resistant Philadelphia-positive leukemic chronic phase and BC stem cells, with the ultimate goal of developing small molecules capable of selectively killing leukemic but not normal hematopoietic stem cells, thereby achieving a ‘biological’ cure for this disease.

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Authors and Affiliations

  1. Department of Microbiology, Immunology and Medical Genetics, Comprehensive Cancer Center, The Ohio State University, Columbus, OH, USA

    D Perrotti

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Correspondence to D Perrotti.

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Competing interests

DP received consulting fees from Pfizer, grant support from the NIH and Karyopharm Therapeutics and has a patent pending for PP2A-activating drugs for CML.

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This article was published as part of a supplement that was supported by Novartis, MSD Italia, Roche, Celgene, GlaxoSmithKline, Sanofi, Gilead, Adienne, Italfarmaco, Pierre Fabre Pharmaceuticals with an unrestricted educational contribution to AREO—Associazione Ricerche Emato-Oncologiche (Genoa) and AMS—Associazione Malattie del Sangue (Milan) for the purpose of advancing research in acute and chronic leukemia.

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Perrotti, D. Where are we going with CML research?. Leukemia Suppl 1 (Suppl 2), S51–S53 (2012). https://doi.org/10.1038/leusup.2012.26

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