Efficacy of Hedgehog inhibitors in acute myeloid leukemia and establishment of valid biomarkers

Abstract

Hedgehog (Hh) signaling pathway plays an important role in embryonic patterning and organ development. Its aberrant activation is involved in many types of cancers and is required for the maintenance of patients with leukemic stem cell (LSC). Clinical phase trials of SMO (a positive regulator in the Hh signaling pathway) inhibitors are currently underway in hematologic malignancies, such as acute myeloid leukemia (AML).