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Current Hematologic Malignancy Reports

, Volume 14, Issue 5, pp 460–468 | Cite as

Novel Therapies in Myeloproliferative Neoplasms (MPN): Beyond JAK Inhibitors

  • Minas P. Economides
  • Srdan Verstovsek
  • Naveen PemmarajuEmail author
Myeloproliferative Neoplasms (B Stein, Section Editor)
Part of the following topical collections:
  1. Topical Collection on Myeloproliferative Neoplasms

Abstract

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.

Summary

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.

Keywords

Ruxolitinib Myeloproliferative neoplasms Novel therapies JAK inhibitors Novel drugs 

Notes

Funding

This research is supported in part by the M. D. Anderson Cancer Center Support Grant P30 CA016672.

Compliance with Ethical Standards

Conflict of Interest

Minas P. Economides declares no conflict of interest.

Srdan Verstovsek declares the following: Consulting/honorarium from Constellation, Pragmatist, Sierra, Incyte Corporation, Novartis, and Celgene. Research funding/clinical trials support from Incyte Corporation, Roche, NS Pharma, Celgene, Gilead, Promedior, CTI BioPharma Corp., Genentech, Blueprint Medicines Corp., and Novartis.

Naveen Pemmaraju declares the following: Consulting/honorarium from Celgene, Stemline, Incyte Corporation, Novartis, MustangBio, Roche Diagnostics, and LFB. Research funding/clinical trials support from Stemline, Novartis, Abbvie, Samus, Cellectis, Plexxikon, Daiichi-Sankyo, Affymetrix, and SagerStrong Foundation.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.

References

Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  • Minas P. Economides
    • 1
  • Srdan Verstovsek
    • 2
  • Naveen Pemmaraju
    • 2
    Email author
  1. 1.Department of Internal MedicineThe University of Texas School of Health Sciences at HoustonHoustonUSA
  2. 2.Department of LeukemiaThe University of Texas MD Anderson Cancer CenterHoustonUSA

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