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 or investigational therapies in myeloproliferative neoplasms include BLU-285, JQ1, and suberoyl anilide hydroxamic acid. Each of these investigational agents carries a unique mechanism of action on myeloid cells. The agents are currently in clinical trials.

Future or investigational therapies

• BLU-285: This medication is a selective KIT D816V inhibitor that is currently in clinical trials in mastocytosis. 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.

• 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.