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<p>Over the past decade, AML therapy has evolved from the uniform “7 + 3” regimen toward a personalized approach. This transition integrates molecular genetic profiles with clinical fitness, driven by the genomic elucidation of AML and the development of selective targeted agents. Key advancements include the integration of FLT3 inhibitors into intensive chemotherapy and the emergence of venetoclax. When combined with hypomethylating agents, venetoclax has redefined the standard of care for older or unfit patients. Furthermore, IDH1/2 inhibitors and menin inhibitors have provided potent options for molecular subsets defined by <i>IDH1/2</i> mutations and <i>KMT2A</i> rearrangements or <i>NPM1</i> mutations, respectively. Innovations such as the liposomal formulation CPX-351 and oral formulations of CC-486 and oral decitabine/cedazuridine have further optimized treatment delivery and improved patient quality of life. Despite these breakthroughs, intrinsic clonal heterogeneity and drug-resistant mutations, particularly <i>TP53</i> mutation, remain significant challenges. Current research is actively exploring next-generation inhibitors, antibody–drug conjugates, and cellular immunotherapies such as BiTEs and CAR-T cells. This review summarizes the recent pharmacological evolution in AML and discusses how these emerging therapies bring us closer to the ultimate goal of achieving a definitive cure.</p>

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The new era of AML therapy: current standards and emerging targets

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2026年4月17日

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  • Naoko Hosono

摘要

Over the past decade, AML therapy has evolved from the uniform “7 + 3” regimen toward a personalized approach. This transition integrates molecular genetic profiles with clinical fitness, driven by the genomic elucidation of AML and the development of selective targeted agents. Key advancements include the integration of FLT3 inhibitors into intensive chemotherapy and the emergence of venetoclax. When combined with hypomethylating agents, venetoclax has redefined the standard of care for older or unfit patients. Furthermore, IDH1/2 inhibitors and menin inhibitors have provided potent options for molecular subsets defined by IDH1/2 mutations and KMT2A rearrangements or NPM1 mutations, respectively. Innovations such as the liposomal formulation CPX-351 and oral formulations of CC-486 and oral decitabine/cedazuridine have further optimized treatment delivery and improved patient quality of life. Despite these breakthroughs, intrinsic clonal heterogeneity and drug-resistant mutations, particularly TP53 mutation, remain significant challenges. Current research is actively exploring next-generation inhibitors, antibody–drug conjugates, and cellular immunotherapies such as BiTEs and CAR-T cells. This review summarizes the recent pharmacological evolution in AML and discusses how these emerging therapies bring us closer to the ultimate goal of achieving a definitive cure.