Novel Targeted Therapeutics in Acute Myeloid Leukemia: an Embarrassment of Riches.

ABSTRACT:

Purpose of review

Acute myeloid leukemia (AML) is an aggressive malignancy of the bone marrow that has a poor prognosis with traditional cytotoxic chemotherapy, especially in elderly patients. In recent years, small molecule inhibitors targeting AML-associated IDH1, IDH2, and FLT3 mutations have been FDA approved. However, the majority of AML cases do not have a targetable mutation. A variety of novel agents targeting both previously untargetable mutations and general pathways in AML are currently being investigated. Herein, we review selected new targeted therapies currently in early-phase clinical investigation in AML.

Recent findings

The DOT1L inhibitor pinometostat in KMT2A-rearranged AML, the menin inhibitors KO-539 and SYNDX-5613 in KMT2Ar and NPM1-mutated AML, and the

SUBMITTER: Grieselhuber NR 

PROVIDER: S-EPMC8515252 | biostudies-literature | 2021 Apr

REPOSITORIES: biostudies-literature