New Research Reveals Androgen receptors as Potential Target for Leukemia Treatment
University Park, PA – A study led by a Regeneron scientist, who began this research as a graduate assistant at Penn State, has uncovered a surprising link between sex hormones and acute myeloid leukemia (AML). The findings suggest that targeting androgen receptors could offer a novel therapeutic approach for leukemia patients, particularly those resistant to customary treatments.
Unexpected Findings in mouse Models:
The finding arose while the researcher,qian,was developing an AML mouse model. he observed a striking difference in disease progression between male and female mice: female mice were less likely to develop AML, while the disease was more aggressive in some males. Initially, Qian hypothesized estrogen was protective, but this was disproven when mice without ovaries still showed resistance to AML.This led him to investigate the role of androgen.
Androgen Receptor activity is Key:
Qian’s research revealed that androgen receptors are highly expressed in female donor leukemic cells. Interestingly, even in male mice with low androgen receptor levels in thier leukemia cells, high levels of dihydrotestosterone (a type of androgen) promoted AML development. This pattern held true when human AML cells were transplanted into the mouse model.
Drugs Show Promise in Slowing Progression:
The study demonstrated that finasteride,a drug that inhibits dihydrotestosterone,and apalutamide,which targets androgen receptor activity,both slowed AML progression in the mouse model.
A Novel Target for leukemia:
Currently, leukemia treatment relies on chemotherapy, radiotherapy, stem cell transplantation, and emerging immunotherapies. This research identifies androgen receptors – and their related pathways – as a previously unexplored target. Qian believes this could benefit patients who haven’t responded to conventional treatments.
Next Steps: Clinical Trials:
While promising, the drugs require further testing, including clinical trials with human patients. Qian plans to conduct these trials with strict inclusion criteria, focusing on patients with increased androgen receptor expression in their leukemic cells.
A Testament to Observational Skills:
Dr. Prabhu, a colleague, praised Qian’s meticulous observation skills, stating that his vigilance was crucial to uncovering this critically important discovery.
Research Team & Funding:
The study was a collaborative effort involving researchers from Penn State, Regeneron Pharmaceuticals, and Temple University. Funding was provided by the American Institute for Cancer Research, National Institutes of Health, and U.S. Department of Agriculture’s National institute of Food and Agriculture.
The research highlights the importance of continued federal funding for scientific innovation. Penn State emphasizes that research is vital for solving real-world problems and strengthening the economy, and recent funding cuts pose a threat to this progress. (Learn more at https://www.psu.edu/research/real-world-solutions).
