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An iron-binding drug, deferiprone, that is currently FDA-approved for the treatment of other diseases, shows promise as a novel way to target ovarian tumors. This finding comes from a preclinical study led by researchers from Weill Cornell Medicine, published in Cancer Discovery. The study focused on the significance of iron in ovarian cancer, as cancer cells typically require more iron than normal cells for their growth and proliferation. By tightly binding to iron, deferiprone prevents cancer cells from utilizing it, potentially inhibiting their growth.

Dr. Juan Cubillos-Ruiz, the senior author of the study, explained that the researchers first examined human ovarian tumor samples to confirm the increased expression of iron-related genes in ovarian cancer cells, which correlated with poor patient prognosis. Additionally, the fluid surrounding ovarian tumors was found to contain high levels of iron that could be readily taken up by cancer cells. To further validate their findings, the team used advanced mouse models of metastatic ovarian cancer, which allowed them to study the immune system response to the disease.

The researchers discovered that as ovarian cancer progressed in the animal models, there was an accumulation of iron in the tumors, with cancer cells specifically over-expressing iron-related gene signatures. Treatment with deferiprone proved to be more effective than cisplatin, the current standard chemotherapy for ovarian cancer, and showed direct action within the cancer cells. The combination of cisplatin and deferiprone significantly increased the survival of the mice with metastatic ovarian cancer, suggesting a synergistic effect of the two drugs in combating the disease.

Dr. Tito Sandoval, the lead author of the study, highlighted that by depriving cancer cells of iron, deferiprone induces a cellular stress response that triggers the immune system to attack the cancer cells. This mechanism, combined with the action of cisplatin on DNA replication, appears to enhance the efficacy of the therapy. The researchers are now collaborating with clinical partners to design human trials to further investigate the potential of combined cisplatin and deferiprone therapy for ovarian cancer.

Dr. Cubillos-Ruiz emphasized the importance of optimizing the therapeutic effects of deferiprone for ovarian cancer in the clinical trial design. While the drug is already approved for other conditions, its use against ovarian cancer requires careful consideration to maximize its benefits. The team is focused on determining the most efficacious way to incorporate deferiprone into the treatment regimen for ovarian cancer patients. The study’s findings may open up new avenues for targeted therapy in ovarian cancer, leveraging the unique iron-related vulnerabilities of cancer cells to improve patient outcomes.

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