If this is not your name, click here.
| | If this is not your name, click here. | | |
| | Contact Us | Order Now | Journals | Bookstore | Register a colleague | | |
| |  Investigational Oral Agent Reduces Symptoms in Patients With Myelofibrosis: Presented at ASH By Ed Susman NEW ORLEANS -- December 7, 2009 -- The investigational drug INCB018424 shows dramatic ability to reduce symptoms related to myelofibrosis and improve patient quality of life, according to phase 2 trial findings report here. At the American Society of Hematology (ASH) 51st Annual Meeting and Exposition, doctors said the drug, being developed by Incyte Corporation, Wilmington, Delaware, targets the Janus-activated kinase (JAK) 2 gene. “The results have been quite amazing in terms to benefit to the patient,” said Srdan Verstovsek, MD, PhD, University of Texas M.D. Anderson Cancer Center, Houston, Texas, during a press briefing held December 5. “More than 75% of the patients are still on the study after an average of 16 months.” The ability for patients to remain on treatment long-term, he said, indicates that treatment is safe as well as effective. Myelofibrosis presents with swelling of the spleen and sometimes the liver, and can be severely debilitating, explained Dr. Verstovsek. Uncontrolled growth of bone marrow cells elicits a cytokine reaction which in turn creates an inflammatory response causing fibrosis and scarring. Because the bone marrow cannot produce enough blood cells, the body puts greater demand on the spleen and liver to produce the cells, leading to splenomegaly. “Because of that, patients have very poor quality of life,” said Dr. Verstovsek. The researchers enrolled 155 patients with either primary myelofibrosis or myelofibrosis occurring after polycythemia vera or essential thrombocythemia. He said the study was the largest ever performed in the disease. Patients received an individually optimised dose of 10 mg, 15 mg, or 25 mg of INCB018424 twice a day. Efficacy of INCB018424 was assessed by measuring spleen volume at 1, 3, and 6 months using magnetic resonance imaging. Patients also were surveyed about the impact of the treatment on their health and well-being. The impact of INCB018424 on exercise capacity was measured with a standardised 6-minute-walk test. “There are no approved therapies for this disease,” Dr. Verstovsek said. The development of INCB018424 followed the late-2004 discovery of a mutation in the JAK2 gene that was observed in about 50% of the patients with myelofibrosis. The mutated gene creates a protein that contributes to uncontrolled cell growth. Dr. Verstovsek said the treatment “benefit is seen primarily in the significant and rapid reduction in splenomegaly in these patients. Half of the patients have experienced a reduction of their palpable spleens by at least 50%.” He added that symptoms of pruritus, night sweating, abdominal discomfort, bone pain, and fatigue also are reduced or eliminated. “They stop their weight loss and gain weight and actually can walk more,” he said. “They start to enjoy their life and have a normal quality of life again.” In addition, INCB018424 treatment improved 6-minute-walk test outcomes, with median increases from baselines of 33, 58, and 70 meters after 1, 3, and 6 months of therapy, respectively. The trial was funded by Incyte Corporation. [Presentation title: Long-Term Follow-up and Optimized Dosing Regimen of INCB018424 in Patients With Myelofibrosis: Durable Clinical, Functional and Symptomatic Responses With Improved Hematological Safety. Abstract 756]
|
