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Experimental Drug Shows Promise for Bone Marrow Disorder
Date:9/15/2010

WEDNESDAY, Sept. 15 (HealthDay News) -- In ongoing early stage research, an experimental drug appears to go some distance toward reducing the tell-tale inflammatory impact of the deadly bone marrow disorder known as myelofibrosis.

Indications that the oral drug -- called INCB018424 -- shows promise are reported in the Sept. 16 issue of the New England Journal of Medicine.

"The problem with myelofibrosis is the lack of available therapies for patients; there are none approved for this disease today," study lead author Dr. Srdan Verstovsek, an associate professor at the M.D. Anderson Cancer Center's department of leukemia in Houston, said in a news release from the center.

"This experimental drug is the first to target one of the underlying abnormalities in the malignant cells that cause myelofibrosis," Verstovsek added. "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."

For reasons as yet poorly understood, myelofibrosis occurs when aggressive bone marrow cells "scar" bone marrow, the soft, fatty tissue inside bones. The process so severely curtails the bone marrow's ability to produce blood cells that anemia results.

In turn, the liver and spleen attempt to take up the blood production slack, causing these organs to swell to painful proportions and impinging on the stomach and bowels. This prompts malnutrition, weight loss, fatigue, and a progressively debilitating loss of the ability to bend and move properly, according to background information in the news release.

An estimated 3,000 Americans are newly afflicted with the disease each year, with an average life expectancy of between five to seven years. There is no known cure or effective treatment, leaving doctors few resources for the alleviation of pain and suffering.

The drug currently being tested is desi
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