Androgen Receptor Signaling as a New Target for Intervention in Acute Myeloid Leukemia

The study investigates the role of Androgen receptor (AR) signaling in acute myeloid leukemia (AML) using a murine model and patient-derived cells. It was found that high AR expression in leukemia initiating cells (LICs) led to more severe disease, with a notable difference between sexes—female LICs had higher AR expression than males. AR signaling was linked to key pathways like PI3K/AKT/MTOR and SRC/HIF-1α. The study demonstrated that using AR antagonists, such as ARN509 and finasteride, which are FDA-approved for prostate cancer, resulted in significant remission and increased survival in AML models. These findings suggest that anti-androgen therapy could be repurposed to enhance AML treatment efficacy.