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Updated: Jan 21, 2026

Transduction-Transplantation Mouse Model of Myeloproliferative Neoplasm
Published on: December 22, 2016
Novel Therapies in Myeloproliferative Neoplasms (MPN): Beyond JAK Inhibitors
Minas P Economides1, Srdan Verstovsek2, Naveen Pemmaraju3
1Department of Internal Medicine, The University of Texas School of Health Sciences at Houston, Houston, TX, USA.
Purpose Of Review:
With increased understanding of the pathobiology of myeloproliferative neoplasms (MPNs), multiple new agents are now being investigated. We aim to cover some of the current treatment options for MPNs and discuss new agents in development.
Recent Findings:
The introduction of ruxolitinib improved the treatment of many patients with intermediate and higher risk myelofibrosis. However, ruxolitinib monotherapy does not benefit all patients, and not all patients can receive this therapy due to limiting cytopenias. The unraveling of new molecular abnormalities and cellular pathways led to the development of several novel targeted agents that are currently under investigation in clinical trials. These agents have different mechanisms of action and are being used either alone or in combination with ruxolitinib. Novel targets include inhibition of apoptosis, the tumor microenvironment, telomerase enzyme action, immunotherapy, and fibrosis with associated cytokines. We comprehensively review and summarize the available preclinical and clinical trials with novel agents for MPNs.
Insights
New targeted agents are being developed for myeloproliferative neoplasms (MPNs) beyond current treatments like ruxolitinib. These novel therapies address unmet needs in MPN treatment by targeting various molecular pathways and cellular mechanisms.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Myeloproliferative neoplasms (MPNs) are a group of blood cancers with evolving treatment landscapes.
- Ruxolitinib has improved outcomes for myelofibrosis but has limitations in efficacy and tolerability for some patients.
Purpose of the Study:
- To review current treatment options for MPNs.
- To discuss novel targeted agents under investigation for MPNs.
Main Methods:
- Comprehensive review of preclinical data and clinical trials.
- Summary of novel agents targeting various molecular pathways.
Main Results:
- Ruxolitinib offers benefits but does not benefit all MPN patients.
- Novel agents target apoptosis, tumor microenvironment, telomerase, and fibrosis.
- Investigational agents are being evaluated alone or in combination with ruxolitinib.
Conclusions:
- Understanding MPN pathobiology drives the development of new therapeutic strategies.
- Novel targeted agents show promise for improving MPN treatment outcomes.
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