☆ 4.7 Review

New Targeted Agents in Acute Myeloid Leukemia: New Hope on the Rise

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)

Journal

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Volume 20, Issue 8, Pages -

Publisher

MDPI

DOI: 10.3390/ijms20081983

Keywords

AML; targeted therapy; FLT3; IDH1; 2; BCL2; hedgehog; immunogenic treatment

Categories

Biochemistry & Molecular Biology Chemistry, Multidisciplinary

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Abstract

The therapeutic approach for acute myeloid leukemia (AML) remains challenging, since over the last four decades a stagnation in standard cytotoxic treatment has been observed. But within recent years, remarkable advances in the understanding of the molecular heterogeneity and complexity of this disease have led to the identification of novel therapeutic targets. In the last two years, seven new targeted agents (midostaurin, gilteritinib, enasidenib, ivosidenib, glasdegib, venetoclax and gemtuzumab ozogamicin) have received US Food and Drug Administration (FDA) approval for the treatment of AML. These drugs did not just prove to have a clinical benefit as single agents but have especially improved AML patient outcomes if they are combined with conventional therapy. In this review, we will focus on currently approved and promising upcoming agents and we will discuss controversial aspects and limitations of targeted treatment strategies.

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