New Discoveries and Honors in Cancer Research

Read the latest cancer research and recognition from the members of the Damon Runyon scientific circle.
December 14, 2012
New targeted therapy effective in treating resistant leukemias

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.