A previously invincible mutation in chronic myeloid leukemia (CML) has been thwarted by an investigational drug in a phase I clinical trial.

12 patients in a 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. 
Twelve patients with acute myeloid leukemia also participated in the trial. A separate paper will address those results.

T315I is present in up to 20 percent of patients and blocks the docking station where three other successful CML drugs normally connect to the mutant protein.

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