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Novel therapeutic agents provide hope for patients with hard-to-treat blood disorders
Date:12/9/2012

h and without the T3151 mutation build on previous evidence and imply that ponatinib is a TKI that may work across a wide range of mutations associated with TKI resistance and also in instances where mutations are not detected.

Remarkably, complete cytogenetic response (no Ph+ cells measured in the body) was achieved in 46 percent of patients with chronic phase CML, with higher response rates observed in patients who were exposed to fewer prior TKIs and those with shorter disease duration. The therapy was also well tolerated in all cohorts, as evidenced by the minimal toxicities observed. The most common adverse events observed were skin toxicity (including rash or dry skin), elevation of pancreatic enzymes and/or pancreatitis, and myelosuppression (a side effect of cancer treatment that lowers blood cell count). At the time of analysis, 52 percent of patients remained on the therapy. These results demonstrate ponatinib's efficacy in CML and Ph+ALL patients with no other viable treatment options.

"This therapy may be able to transform highly fatal forms of leukemia into a curable disease in these patients we have simply never had any treatment produce such high rates of durable response in such a heavily treated group of patients," said Jorge Cortes, MD, lead author, Professor of Medicine, Deputy Chair of the Department of Leukemia, and Chief of the CML and AML Sections at The University of Texas MD Anderson Cancer Center in Houston. "Our next step is to test ponatinib's potential as an initial therapy in an attempt to prevent the occurrence of relapse that may decrease the prospects of a normal lifespan for patients."

Dr. Cortes will present this study in an oral presentation on Sunday, December 9, at 4:30 p.m. EST at the Georgia World Congress Center in Room A411-A412, Level 4, Building A

Phase I/II Study of Weekly MLN9708, an Investigational Oral Proteasome Inhibitor, in Combination with Lenalidomide and Dexamethaso
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Contact: Andrea Slesinski
aslesinski@hematology.org
614-352-5096
American Society of Hematology
Source:Eurekalert

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