NGS-Guided Targeted Therapies Reshape the Treatment Landscape for Acute Myeloid Leukemia
Next-generation sequencing has revealed the mutational hierarchy driving AML, enabling development of targeted agents against specific driver proteins such as mutant IDH and FLT3. This review from Blood surveys emerging drugs including novel chemotherapy formulations, antibody-drug conjugates, and mutation-targeted inhibitors. The work highlights how molecular profiling is becoming essential for treatment stratification in AML, with implications for laboratory workflows in hematologic malignancy testing.
The original study
Emerging therapeutic drugs for AML.
- Authors
- Stein EM, Tallman MS
- Journal
- Blood
- Type
- Journal Article, Review
- PMID
- 26660428
Original abstract
Multiple new drugs are being developed to treat acute myeloid leukemia (AML), including novel formulations of traditional chemotherapy-antibody drug conjugates and agents that target specific mutant enzymes. Next-generation sequencing has allowed us to discover the genetic mutations that lead to the development and clinical progression of AML. Studies of clonal hierarchy suggest which mutations occur early and dominate. This has led to targeted therapy against mutant driver proteins as well as the development of drugs such as CPX-351 and SGN-CD33A whose mechanisms of action and efficacy may not be dependent on mutational complexity. In this brief review, we discuss drugs that may emerge as important for the treatment of AML in the next 10 years.