New Myelofibrosis Drug Holds Promise

In a phase I clinical trial, physicians at The Mount Sinai Medical Center have identified the first drug that appears to stop the progression of myelofibrosis, a life-threatening blood cancer. The investigators found that, at low-doses, panobinostat (LBH589) successfully halted and reversed damage to the blood and bone marrow in several of the forty patients enrolled in the trial. Panobinostat, manufactured by Novartis, is a histone deacetylase inhibitor that affects the chromatin structure of malignant cells.

The study, led by Ronald Hoffman, MD, Albert A. and Vera G. List Professor of Medicine, and Director of the Myeloproliferative Disorders Research Program, and John O. Mascarenhas, MD, Assistant Professor of Medicine (Hematology and Medical Oncology), was published online in the January 21, 2013, issue of the British Journal of Haematology.

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