Targeted therapy of acute myeloid leukemia

Benedito A. Carneiro, Jessica K. Altman, Jason B. Kaplan, Gert Ossenkoppele, Ronan Swords, Leonidas C. Platanias, Francis J. Giles

Research output: Contribution to journalReview articlepeer-review

9 Scopus citations


Advances in the understanding of the genetic underpinnings of acute myeloid leukemia are rapidly being translated into novel treatment strategies. Genomic profiling has highlighted the importance of the epigenetic machinery for leukemogenesis by identifying recurrent somatic mutations involving chromatin-modifier proteins. These genetic alterations function as dynamic regulators of gene expression and involve DNA-methyltransferase 3A, methyltransferase DOT1L, enhancer of zeste homologue 2, isocitrate dehydrogenases 1 and 2 and bromodomain-containing proteins. New therapeutic targets are also emerging from further delineation of cell signaling networks in acute myeloid leukemia blasts mediated by PIM kinases, polo-like kinase 1, cell surface protein CD98 and nucleocytoplasmic shuttling receptors, among others. Early results of targeted therapies directed at these molecular mechanisms are discussed in this review and their potential to improve the outcomes of patients by allowing the use of more effective and less toxic treatments.

Original languageEnglish (US)
Pages (from-to)399-413
Number of pages15
JournalExpert review of anticancer therapy
Issue number4
StatePublished - Apr 1 2015
Externally publishedYes


  • acute myeloid leukemia
  • chromatin-modifier proteins
  • targeted therapy
  • treatment

ASJC Scopus subject areas

  • Oncology
  • Pharmacology (medical)


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