NIH Reporter #1P50CA298970-01A1

Project 1 Targeting Menin in Leukemia

This award funds research on Menin inhibitors for treating acute myeloid leukemia, focusing on overcoming resistance and developing combination therapies, including a Phase I clinical trial.

Description

Menin inhibitors, which disrupt the Menin-KMT2A complex and reverse leukemic gene expression, are novel targeted agents currently in clinical development for the treatment of genetically defined subsets of acute myeloid leukemia (AML). As single agents are virtually never curative and resistance to Menin inhibitors has been reported, we propose to characterize adaptive resistance and the minimal residual disease state with Menin inhibitor treatment, while investigating future combination therapeutic strategies with Menin inhibitors in AML. The proposed studies will define a path forward for new combination approaches and a Phase I clinical trial of combinations with Menin inhibitors will be performed in order to advance new therapeutic opportunities for patients with AML.

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