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.
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Current MF Research and Clinical Trials
Some examples of treatments being studied include:
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.
Histone Deacetylase (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.
Immunomodulatory drugs (IMiDs)
A class of drugs that work against cancer cells by affecting the functions of the immune system. Three IMiDs are being investigated for the treatment of patients with MF. These are:
- Thalidomide (Thalomid®) and lenalidomide (Revlimid®), which are FDA approved to treat myeloma patients and
- Pomalidomide (Actimid®), which is currently being studied in clinical trials for the treatment of myeloma patients.
In MF patients, Thalomid has been associated with improvements in anemia, platelet count, enlarged spleen, and symptoms such as night sweats, weakness, fatigue and shortness of breath. Revlimid is active in treating anemia, thrombocythemia, enlarged spleen and marrow fibrosis. Actimid appears effective in treating anemia and improving low platelet counts. Patients taking Thalomid or Revlimid should be closely monitored for symptoms of peripheral neuropathy (a tingling or burning sensation in the hands and feet) and myelosuppression (decreased production of blood cells). These side effects are less frequent for patients taking Actimid and are linked to higher doses of the drug.