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Revolutionary new drug successfully treats deadly blood cancer

A new treatment for polycythemia vera, a potentially deadly blood cancer, has shown promising results in a recent clinical trial. (CREDIT: Creative Commons)

A new treatment for polycythemia vera, a potentially deadly blood cancer, has shown promising results in a recent clinical trial led by the Icahn School of Medicine at Mount Sinai.

The trial focused on a drug called rusfertide, which was found to effectively control the excessive production of red blood cells, a hallmark of the disease, over a 28-week period.


These findings, published in The New England Journal of Medicine, indicate that rusfertide could offer a viable alternative to therapeutic phlebotomy, a burdensome treatment for many patients.

According to Marina Kremyanskaya, MD, PhD, Associate Professor of Medicine at Icahn Mount Sinai and lead author of the study, "Rusfertide appears to represent a significant step forward in treating polycythemia vera through its unique approach of limiting the amount of iron available for blood cell production."


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Dr. Kremyanskaya, along with her colleagues Dr. Ronald Hoffman and Dr. Yelena Ginzburg, played crucial roles in translating laboratory findings into clinical applications.

Polycythemia vera is a rare form of chronic myeloproliferative neoplasm, affecting 1-3 new cases per 100,000 people annually in the United States. It results from the overproduction of blood cells in the bone marrow, leading to thickened blood and increased risks of heart attack, stroke, and blood clots.


Current treatments include aspirin, medications like hydroxyurea and interferon, and phlebotomy, which involves blood withdrawal to reduce blood volume. However, frequent phlebotomies can be taxing for patients, exacerbating iron deficiency symptoms and posing risks of complications.

Graph displaying hematocrit levels over time. (CREDIT: The New England Journal of Medicine)

The REVIVE trial, conducted at 16 centers in the United States and India and funded by Protagonist Therapeutics, Inc., assessed the safety and efficacy of rusfertide in 70 phlebotomy-dependent polycythemia vera patients.


Results showed improved and sustained control of hematocrit levels below 45 percent, a key marker for the disease, during the treatment period. Moreover, rusfertide was superior to placebo in maintaining these improvements during the subsequent withdrawal phase.

Patients also reported alleviation of symptoms such as itchy skin, night sweats, difficulty concentrating, and fatigue.


Rusfertide, administered via injection, mimics hepcidin, a liver hormone regulating iron traffic. By blocking iron export to the blood, rusfertide induces functional iron deficiency and reduces red blood cell production. Importantly, the drug can be self-administered by patients at home, offering convenience and potentially reducing the need for repeated phlebotomies.

Dr. Kremyanskaya emphasizes the promise of rusfertide in achieving sustained hematocrit control and decreasing the reliance on phlebotomies. "Some patients remained virtually free of the procedure for more than two and a half years," she notes.


With the success of the phase 2 trial, a phase 3 trial sponsored by Protagonist Therapeutics is now underway at global sites, with Mount Sinai continuing its leadership in advancing polycythemia vera treatment.

For more science news stories check out our New Innovations section at The Brighter Side of News.


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