Currently approved therapies for myelofibrosis (MF) consist of JAK inhibitors, which produce meaningful improvements in spleen size and symptom burden but do not significantly impact leukemic progression. In addition, many patients develop resistance or intolerance to existing therapies and are left without meaningful therapeutic options. There has been recent rapid development of agents in MF that may be able to fill these unmet needs. Importantly, most treatments currently in clinical development have targets outside the JAK-STAT pathway, including BET, BCL-2/BCL-xL, PI3k, HDM2, PIM-1, SINE, telomerase, LSD1, and CD123. These therapies are being tested in combination with JAK inhibitors in the front-line setting and in patients with a suboptimal response, as well as a single agent after JAK inhibitor failure. This next generation of agents is likely to produce a paradigm shift in MF treatment with a focus on combination treatment targeting multiple areas of MF pathophysiology.
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