HOUSTON - An oral medication produces significant and lasting relief for patients with myelofibrosis, a debilitating and lethal bone marrow disorder, researchers at The University of Texas MD Anderson Cancer Center report in the Sept. 16 New England Journal of Medicine.
Myelofibrosis is caused by the accumulation of malignant bone marrow cells that trigger an inflammatory response, scarring the bone marrow and limiting its ability to produce blood, causing anemia.
"The problem with myelofibrosis is the lack of available therapies for patients - there are none approved for this disease today," said principal investigator Srdan Verstovsek, M.D., Ph.D., associate professor in M. D. Anderson's Department of Leukemia. Average life expectancy for people with this disease is 5 to 7 years. Available therapies approved for other diseases provide little response and are mainly palliative.
"This experimental drug is the first to target one of the underlying abnormalities in the malignant cells that cause myelofibrosis," Verstovsek said. "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."
Swollen spleens cause pain, malnutrition
"Interestingly, other organs, mainly the spleen, attempt to take over the production of blood cells. Bone marrow forms in the spleen," Verstovsek said. Malignant cells also accumulate there. "The growing spleen causes significant problems for the patient, and not just because it's painful. It compresses the stomach and bowels, so patients suffer malnutrition and lose weight. The ability to walk and to bend is affected, and the body deteriorates overall."
End-stage patients resemble the severely malnourished, with bloated abdomens and thin limbs. Patients on the study gained weight while on the medication.
The phase I/II clinical trial of INCB018424
|Contact: Scott Merville|
University of Texas M. D. Anderson Cancer Center