Myeloproliferative neoplasm future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]

Overview

Future of investigational therapies

  • BLU-285: This medication is a selective KIT D816V inhibitor that is currently in clinical trials in mastocytosis.[1] The preliminary data presented at the American Society of Hematology meeting in 2017 showed promising results. It is generally well-tolerated. BLU-285 remains in clinical trials.
  • JQ1: This medication is a bromodomain-containing protein 4 (BRD4) inhibitor that is currently being studies in advanced mastocytosis. JQ1 works by blocking the interaction between BRD4 and acetylated histones. It functions as an epigenetic modifying agent, since it influences histone activity and thereby regulates gene transcription. JQ1 triggers apoptosis in cells.[1]
  • Suberoyl anilide hydroxamic acid (SAHA): This medication is a histone deacetylase inhibitor that downregulates the expression of kit. This medication is also known as vorinostat, which is used in cutaneous T cell lymphoma.[1]

References

  1. 1.0 1.1 1.2 Vaes M, Benghiat FS, Hermine O (2017). "Targeted Treatment Options in Mastocytosis". Front Med (Lausanne). 4: 110. doi:10.3389/fmed.2017.00110. PMC 5517467. PMID 28775983.

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