Taking part in a clinical trial may be the best treatment choice for some myelofibrosis (MF) patients. Patient participation in clinical trials is important in the development of new and more effective treatments for MF and may provide patients with additional treatment options.
Click here to read more about clinical trials.
Current MF Research and Clinical Trials
Some examples of treatments being studied include:
- JAK Inhibitors target abnormal JAK pathway signaling, which is present in all MF patients, largely due to either JAK2, CALR or MPL mutations. Several JAK inhibitors are now in clinical trials and are showing effectiveness in reducing spleen size and symptoms such as night sweats and fatigue and possibly improving anemia. These new treatments include momelotinib (CYT387), pacritinib (SB1518) and NS-018.
- Histone deacetylase (HDAC) inhibitors play an important role in the regulation of gene expression. A clinical study of panobinostat (Farydak®), which is approved by the FDA for the treatment of multiple myeloma, in combination with ruxolitinib in patients with MF is ongoing. Pracinostat is another HDAC inhibitor that is being studied in combination with ruxolitinib.
- Antifibrotic agents interfere with the process of tissue repair and fibrosis. PRM-151 is an antifibrotic therapy that is being tested in MF. Lysin oxidase like-2 antibody is another antifibrotic medication that is being studied in a clinical trial.
- Therapies that target other pathways that may be abnormally activated in MF are also being tested. LCL-161 is an oral therapy that blocks the activity of inhibitor of apoptosis (IAP) proteins, which promote cell survival. SL-401 is a therapy that targets the IL-3 receptor, which is found on the surface of MF cells.
- Immune checkpoints inhibitors (nivolumab, ipilmumab) are a new class of drugs that harness the body’s immune system to fight cancer. Nivolumab (Opdivo®), which is approved by the FDA for the treatment of melanoma and non-small cell lung cancer, is now being tested as a therapy for MF.
- Sotatercept (ACE-011) is a therapy that stimulates the production of red blood cells and is being tested to treat anemia in MF.
- Imetelstat is a telomerase inhibitor, which affects ability of dividing cell to repair loss of DNA that happens during cell division. It is being studied in MF to possibly improve bone marrow and normalize blood cell count.