Novel therapies for myelofibrosis

Brady L. Stein*, Francisco Cervantes, Francis Giles, Claire N. Harrison, Srdan Verstovsek

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

7 Scopus citations


Myelofibrosis (MF), including primary, post-essential thrombocythemia and post-polycythemia vera MF, associates with a reduced quality of life and shortened life expectancy. Dysregulation of the Janus kinase (JAK)/signal transducer and activator of transcription (STAT) pathway is prominent, even in the absence of the JAK2V617F mutation. Therefore, all symptomatic MF patients may potentially derive benefit from JAK inhibitors. Despite the efficacy of JAK inhibitors in controlling signs and symptoms of MF, they do not eradicate the disease. Therefore, JAK inhibitors are currently being tested in combination with other novel therapies, a strategy which may be more effective in reducing disease burden, either by overcoming JAK inhibitor resistance or targeting additional mechanisms of pathogenesis. Additional targets include modulators of epigenetic regulation, pathways that work downstream from JAK/STAT (i.e. mammalian target of rapamycin/AKT/phosphoinositide 3-kinase) heat shock protein 90, hedgehog signaling, pro-fibrotic factors, abnormal megakaryocytes and telomerase. In this review, we discuss novel MF therapeutic strategies.

Original languageEnglish (US)
Pages (from-to)2768-2778
Number of pages11
JournalLeukemia and Lymphoma
Issue number10
StatePublished - Oct 3 2015


  • Molecular genetics
  • myeloproliferative disorders
  • signaling therapies

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research


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