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

    September 2025 in “ Blood Advances
    Fenghua Qian, Deborpita Sarkar, Brooke E. Arner, Vanessa M Peduzzi, Yuting Bai, Baiye Ruan, Bei Jia, Hong Zheng, Robert F. Paulson, K. Sandeep Prabhu
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    Studysummary This study found that targeting androgen receptors with prostate cancer drugs ARN509 and finasteride induced remission and increased survival in a leukemia mouse model, suggesting a potential new treatment approach for acute myeloid leukemia. Our plain-language summary of this paper — not a Tressless recommendation.
    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.
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