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Drug under study shows relief for bone marrow disorder

September 20, 2010
Texas researchers say an experimental oral drug now being studied has provided significant relief for those with myelofibrosis, a lethal bone marrow disorder.

Myelofibrosis is caused by a growth in malignant bone marrow cells that lead to inflammation, scarred bone marrow and anemia caused by the body's inability to produce blood. In the September issue of the New England Journal of Medicine, researchers from the University of Texas MD Anderson Cancer Center say a clinical trial of a JAK1 and JAK2 genetic inhibitor is the first to target the malignant cells that cause myelofibrosis.

"It provides unprecedented reduction of enlarged spleens that are a central characteristic of the disease, and relieves pain, fatigue and other symptoms, improving quality of life," said Dr. Srdan Verstovsek, of the cancer center's Department of Leukemia.

Patients enrolled in the clinical trial of the drug, identified as INCB018424 developed by the Incyte Corporation, have experienced weight gain while on the medication. Late-stage myelofibrosis patients often appear malnourished, with bloated abdomens and thin limbs.

The pediatric bone marrow transplantation program at Cohen Children's Medical Center is one of a select group nationwide that has been accredited by the Foundation for the Accreditation of Cellular Therapy.
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