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Updated: May 18, 2026

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Published on: June 6, 2025
Role of additional novel therapies in myeloproliferative neoplasms
Warren Fiskus1, Siddhartha Ganguly, Suman Kambhampati
1The University of Kansas Medical Center, 3901 Rainbow Boulevard, Robinson Hall 4030, Mail Stop 1027, Kansas City, KS 66160, USA.
Abstract:
The recent approval of ruxolitinib (INCB018424) for myelofibrosis and the preclinical/clinical development of several additional janus kinase (JAK)-targeted agents have ushered in an era of novel therapies for advanced myeloproliferative neoplasms (MPN), which are associated with constitutive activation of the JAK-signal transducer and activation of transcription (STAT) signaling pathway. Collectively, these novel therapeutic approaches could rapidly broaden the spectrum of available therapies, with potential for improved clinical outcome for patients with advanced MPN. This review covers the recent developments in the testing of novel therapeutic agents other than JAK inhibitors that target signaling pathways in addition to JAK/STAT, or target the deregulated epigenetic mechanisms in MPN.
Insights
Novel therapies targeting Janus kinase (JAK) and other pathways offer new hope for advanced myeloproliferative neoplasms (MPN). This review explores emerging treatments beyond JAK inhibitors for improved patient outcomes.
Area of Science:
- Hematology
- Oncology
- Pharmacology
Background:
- Advanced myeloproliferative neoplasms (MPN) are characterized by constitutive activation of the JAK-STAT signaling pathway.
- Ruxolitinib approval and development of other JAK inhibitors mark a new era in MPN therapy.
Purpose of the Study:
- To review novel therapeutic agents for advanced MPN beyond JAK inhibitors.
- To discuss agents targeting additional signaling pathways and epigenetic mechanisms in MPN.
Main Methods:
- Literature review of preclinical and clinical studies.
- Analysis of emerging therapeutic strategies for MPN.
Main Results:
- Several novel therapeutic agents are in development for MPN.
- These agents target pathways beyond JAK/STAT and address epigenetic dysregulation.
Conclusions:
- Novel therapies, including non-JAK inhibitors, show promise for improving clinical outcomes in advanced MPN.
- Targeting diverse signaling and epigenetic mechanisms broadens therapeutic options for MPN patients.
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