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Taking part in a clinical trial may be the best treatment choice for some myelofibrosis (MF) patients. Today's standard treatments for cancer are based on earlier clinical trials. LLS continues to invest funds in MPN research.

Clinical trials can involve new drugs, new combinations of drugs or approved drugs being studied to treat patients in new ways such as new drug doses or new schedules to administer the drugs. Clinical trials are conducted worldwide under rigorous guidelines to help doctors find out whether new cancer treatments are safe and effective or better than the standard treatment.

Current MF Research and Clinical Trials

Some examples of treatments being studied include:

JAK inhibitors. These drugs target abnormal pathway signaling caused by a specific mutation in the JAK2 gene. Several JAK inhibitors are now in clinical trials and are showing effectiveness in reducing spleen size and improving anemia and symptoms such as night sweats and fatigue. These potential treatments include CYT387, SAR302503 (TG101348), AZD1480, CEP-701, SB1518 and LY2784544.

Thalidomide (Thalomid®). This agent is an immune modulator currently being used to treat myeloma and other conditions. In studies with MF patients, thalidomide has been associated with improvements in anemia, platelet count, an enlarged spleen and symptoms such as night sweats, weakness, fatigue and shortness of breath.

Lenalidomide (Revlimid®). This drug is similar to thalidomide, and currently FDA approved to treat myeloma patients, but generally has fewer side effects and is more potent than thalidomide. Lenalidomide is being studied as a potential therapy for MF patients to treat anemia, thrombocythemia, an enlarged spleen and marrow fibrosis.

Pomalidomide (Actimid®). This drug is currently being studied in clinical trials for the treatment of myeloma patients. In MF patients, Actimid appears effective in treating anemia and improving low platelet counts.

Histone deacetylase (HDAC) inhibitors. HDAC inhibitors play an important role in the regulation of gene expression. These HDAC inhibitors are in clinical studies for MF: panobinostat (LBH589), givinostat (ITF2357) and vorinostat (Zolinza®), which is FDA approved for treatment of cutaneous T-cell lymphoma.

Reduced-intensity allogeneic stem cell transplantation. Doctors are studying a reduced-intensity stem cell transplantation, also called a nonmyeloablative transplantation. This type of stem cell transplant is being used to treat some patients with leukemia, lymphoma and myeloma. Unlike standard transplantation, reduced-intensity transplantation doesn't destroy the patient's diseased marrow and relies on donor immune cells to fight the disease. The intention is to achieve the benefits of allogeneic stem cell transplantation while reducing the procedure's risks. The use of nonmyeloablative transplantation has shown promise in a small number of patients. Additional investigation is needed to compare long-term outcomes of this treatment to outcomes of other types of therapy for MF patients.

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last updated on Friday, April 27, 2012
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