Frank G. Haluska, MD, PhD (Damon Runyon Fellow ‘94-‘96) of ARIAD Pharmaceuticals, Cambridge, and colleagues, reported the success of the investigational targeted therapy Ponatinib (AP24534) in treating patients with resistant types of blood cancers, including chronic myeloid leukemia (CML) and a subtype of acute lymphoblastic leukemia (Ph-positive ALL).  The drug blocks the kinase BCR-ABL, including its mutated form (T315I) which is resistant to existing kinase inhibitor drugs.  In this Phase I trial, nearly all patients responded to treatment.  Ponatinib is a promising new treatment for patients who have no other treatment options.  The Phase I trial results were reported in The New England Journal of Medicine. The FDA approved ponatinib (Iclusig) for the treatment of these two forms of drug-resistant leukemia. Approval was based on a single phase II trial, results of which were reported last week at the American Society of Hematology’s annual meeting.