Ponatinib in the therapy of chronic myeloid leukemia.

INTRODUCTION: Chronic Myeloid Leukemia (CML) is a myeloproliferative disorder that has become the neoplastic poster child for understanding the disease biology of a malignant disease and targeting effective therapy. The targeted therapy of BCR-ABL inhibition by tyrosine kinase inhibitors (TKI) has provided the epitome for "Ehlrich's magic bullet" postulated decades ago. AREAS COVERED: Due to the therapy with these drugs, the survival of newly diagnosed patients with this disease now approaches that of age matched controls. Progression to advanced phases of CML had decreased over the years, though resistance has now been increasingly identified. Expert commentary: Ponatinib is a third generation TKI, which has shown to be effective in both early and advanced phases of CML and those bearing resistant mutations, specifically T315I. However, new side effect considerations need to be balanced with the efficacy, to establish the role of ponatinib in the therapy of CML.