Credit score: Blood Advances (2025). DOI: 10.1182/bloodadvances.2024012639
Two U.S. Meals and Drug Administration (FDA)-approved medication for treating prostate most cancers can also be efficient in opposition to acute myeloid leukemia (AML), in keeping with a brand new examine by researchers at Penn State. AML, a most cancers of blood and bone marrow, can have an effect on folks of all ages however is the commonest kind of leukemia in adults.
The analysis, just lately revealed within the journal Blood Advances, examined the power of the 2 medication—apalutamide and finasteride—to deal with leukemia in experimental fashions. They discovered that the medication had been efficient in treating mice with AML in addition to patient-derived AML cells.
Ok. Sandeep Prabhu, head, division of veterinary and biomedical sciences, and professor of immunology and molecular toxicology within the Faculty of Agricultural Sciences and a lead creator on the paper, mentioned the medication work by inhibiting the pathway that’s activated by dihydrotestosterone—an androgen hormone stronger than testosterone.
“Although androgen signaling is well-studied in prostate cancer and has successfully guided molecular-targeted therapies, its role in AML has been under-explored,” Prabhu mentioned. “In our study, these two drugs effectively suppressed AML progression in both male and female mice, highlighting its potential for new uses for the drugs.”
Robert Paulson, professor of veterinary and biomedical sciences at Penn State and co-author on the paper, mentioned the findings supply a promising new route for AML remedy.
“AML is a tough disease,” Paulson mentioned. “Many patients relapse and the treatments haven’t really changed. It is exciting to identify a new target, the androgen receptor, where there are already FDA-approved drugs. The next steps will be using these drugs in clinical trials. We are excited to see what happens in patients.”
Earlier than the present examine, the connection between leukemia and androgen receptors was beforehand unknown, in keeping with the researchers, who’ve additionally registered a patent for utilizing the androgen receptor inhibitors to deal with AML.
Fenghua Qian, a senior scientist at Regeneron who led the examine as a graduate assistant at Penn State, the place he earned his doctorate in pathobiology, mentioned the invention happened when he was engaged on establishing a way to develop an AML mouse mannequin. This can be a widespread method for finding out varied illness progressions and potential therapeutic targets, Qian mentioned. Whereas creating the AML mannequin, Qian discovered that among the feminine mice did not develop AML, however the illness was unusually aggressive in among the male mice.
His first thought was that estrogen may be defending the feminine mice in opposition to AML, Qian mentioned, however discovered that mice with out ovaries—and subsequently no estrogen—nonetheless had a decrease likelihood of creating AML.
“This was when I switched gears to analyzing the potential role of androgen,” Qian mentioned. “I started to analyze female and male donor leukemic cells, as well as female and male recipients, and was able to demonstrate that androgen receptors are highly expressed in female donor leukemic cells. And the interaction of androgen and androgen receptors could potentially be a target for the treatment of leukemia.”
Within the present examine, the researchers discovered that in male mice, excessive dihydrotestosterone promoted the event of AML regardless of low androgen receptors of their leukemia cells. In the meantime, though feminine mice had low dihydrotestosterone, this was offset by excessive ranges of androgen receptors of their leukemia cells. The crew discovered this held true for AML cells transplanted from human sufferers into the mouse mannequin, as nicely.
Finasteride slowed AML development by inhibiting dihydrotestosterone, the researchers mentioned, whereas apalutamide labored by focusing on a barely completely different pathway in androgen receptor exercise.
Qian mentioned that presently, remedies for leukemia embrace chemotherapy, radiotherapy and stem cell transplantation together with rising immunotherapies. Nonetheless, androgen receptors have by no means been thought of as a goal.
“This study provides a novel targetable pathway for leukemia patients,” he mentioned. “Not only can androgen receptors be the direct target, but also its upstream and downstream interactors. It could also potentially benefit those patients who are resistant to traditional treatments.”
The researchers added that whereas the examine was promising, the medication will nonetheless want further testing, together with in people. Qian mentioned he expects to check each medication in scientific trials on leukemia sufferers with strict inclusion standards equivalent to elevated androgen receptor expression in leukemic cells.
Prabhu mentioned the examine is an efficient instance of how consideration to element can result in new, vital scientific discoveries.
“Qian was so vigilant, and he was the one who saw that there was a problem here,” Prabhu mentioned. “He had the presence of mind as well as a close observation of what he was seeing, and he managed to catch this. And that is what changed everything.”
Extra data:
Fenghua Qian et al, Androgen receptor signaling as a brand new goal for intervention in acute myeloid leukemia, Blood Advances (2025). DOI: 10.1182/bloodadvances.2024012639
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