A previously invincible mutation in chronic myeloid leukemia (CML) has been thwarted by an investigational drug in a phase I clinical trial reported in The New England Journal of Medicine. All 12 patients in the trial with chronic phase CML and the T315I mutation had a complete hematologic response (absence of CML cells in the blood) after treatment with ponatinib. Eleven had a major reduction in CML cells in the bone marrow and nine achieved a complete cytogenetic response – no cells in the marrow. Read more here.
Study mentioned: Cortes JE, et al. Ponatinib in Refractory Philadelphia Chromosome–Positive Leukemias. N Engl J Med 2012; 367:2075-2088.
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