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Targeting IDH1 and IDH2 Mutations in Acute Myeloid Leukemia

  • Acute Myeloid Leukemias (H Erba, Section Editor)
  • Published:
Current Hematologic Malignancy Reports Aims and scope Submit manuscript

Abstract

Purpose of Review

Over the past decade, the pathogenic role of mutations in isocitrate dehydrogenases (IDH) 1 and 2, affecting approximately 20% of patients with AML, has been defined, allowing for the development of specific therapeutic strategies for IDH-mutant AML. In this review, the landscape and progress of targeted therapeutics aimed at IDH mutations in AML and related myeloid malignancies will be described.

Recent Findings

Since 2013, several mutant IDH-targeted inhibitors have been developed, and nearly a dozen clinical trials have opened specifically for IDH-mutant hematologic malignancies. Preliminary results for several of these investigations have shown evidence of safety, tolerability, and encouraging evidence of efficacy.

Summary

Targeting IDH mutations in AML is a biologically informed and rational strategy to promote clinical responses, primarily through differentiation and maturation of the malignant clone. The use of IDH targeted therapy is expected to soon become part of a genomically defined and individualized AML treatment strategy.

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Funding

Supported in part by the MD Anderson Cancer Center Support Grant CA016672 and NIH T32 Training Grant CA009666

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Authors and Affiliations

  1. Department of Hematologic Oncology and Blood Disorders, The Levine Cancer Institute, Carolinas HealthCare System, Charlotte, NC, USA

    Brittany Knick Ragon

  2. Department of Leukemia, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard Unit 428, Houston, TX, 77030, USA

    Courtney D. DiNardo

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  1. Brittany Knick Ragon
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Contributions

Brittany Ragon and Courtney DiNardo participated in the discussion, wrote, and have reviewed and approved the current version of the manuscript.

Courtney DiNardo is responsible for the overall content as guarantor.

Corresponding author

Correspondence to Courtney D. DiNardo.

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Conflict of Interest

Brittany Knick Ragon declares that he has no conflicts of interest.

Courtney D. DiNardo has served on advisory boards for Agios, Celgene, and Novartis.

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.

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This article is part of the Topical Collection on Acute Myeloid Leukemias

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Ragon, B.K., DiNardo, C.D. Targeting IDH1 and IDH2 Mutations in Acute Myeloid Leukemia. Curr Hematol Malig Rep 12, 537–546 (2017). https://doi.org/10.1007/s11899-017-0418-6

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  • DOI: https://doi.org/10.1007/s11899-017-0418-6

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