Pacritinib: A novel therapeutic agent for treating myelofibrosis

Selective inhibitors are an important advancement in clinical medicine, providing a method to target proteins within signal transductionpathways. One such inhibitor, pacritinib, targets Janus Kinase 2 (JAK2) to treat patients with myelofibrosis (MF). In MF patients diagnosedwith a specific point mutation (JAK2V61F), the JAK/STAT pathway is over-activated, leading to an increase in cell proliferation rates.Pacritinib restores normal JAK/STAT activity by targeting aberrant activity of JAK2V61F mutant proteins and inhibiting cell proliferation inbone marrow tissue. In recent studies, pacritinib has shown better results at treating MF than the best available therapy. Though pacritinibis a potentially effective treatment option, it does not address all aspects of MF and should be supplemented with other treatments.