New drug shows promise in controlling deadly blood cancer
Polycythemia vera is a type of chronic myeloproliferative neoplasm, a group of disorders that affect the production of blood cells in the bone marrow.
A new drug called rusfertide has shown promising results in a clinical trial for polycythemia vera, a rare and life-threatening blood cancer. The drug lowered the excess production of red blood cells, the main cause of the disease and its serious complications.
The trial, led by researchers from the Icahn School of Medicine at Mount Sinai, involved 70 patients who were dependent on phlebotomy, a procedure that removes blood from the body to reduce its volume. The patients received rusfertide injections for 28 weeks and were monitored for their blood counts and symptoms.
The results, published in The New England Journal of Medicine, showed that rusfertide effectively reduced the need for phlebotomy and improved the patient's quality of life. The drug also had a favorable safety profile and was well tolerated by most patients.
A novel approach to treat polycythemia vera
Polycythemia vera is a type of chronic myeloproliferative neoplasm, a group of disorders that affect the production of blood cells in the bone marrow. In polycythemia vera, the bone marrow makes too many red blood cells, which can make the blood thick and sticky. This can lead to an increased risk of blood clots, heart attack, stroke, and other serious problems.
The disease affects about 1–3 people per 100,000 in the United States and is usually diagnosed in older adults. The current treatments include aspirin, drugs that lower the red blood cell count, and phlebotomy. However, these treatments have limitations and side effects, and many patients still have uncontrolled disease and poor outcomes.
Rusfertide is a novel drug that works by binding to a protein called hepcidin, which regulates the body's iron. By increasing hepcidin levels, rusfertide reduces the availability of iron for red blood cell production, thus lowering the red blood cell count and alleviating the symptoms of polycythemia vera.
A breakthrough for blood cancer research
The study's lead author, Dr. Marina Kremyanskaya, an Associate Professor of Medicine and Medical Director for inpatient oncology at The Mount Sinai Hospital, said that rusfertide could be a game-changer for polycythemia vera patients.
"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," she said. "Pending further clinical studies, this injectable agent could become a valuable therapeutic tool for a disease which many patients and their physicians struggle to bring under control."
She also acknowledged the contributions of her co-investigators, Dr. Ronald Hoffman and Dr. Yelena Ginzburg, both Professors of Medicine at Icahn Mount Sinai and experts in the field of myeloproliferative neoplasms and iron metabolism, respectively.
"This groundbreaking work would not have been possible without their close collaboration. We were able to take laboratory findings from the bench to bedside," she said.
The researchers found that the drug was associated with improved and sustained control of hematocrit levels below 45% during the 28-week dose-discovery period and was superior to placebo during the 12-week withdrawal period. Patients with a high burden of symptoms such as pruritis (itchy skin), night sweats, difficulty concentrating, and fatigue also reported improvements following treatment with rusfertide, which was well tolerated.
Rusfertide is an injectable medication that patients can self-administer at home. It works in the body by mimicking hepcidin, a hormone produced by the liver that serves as a master regulator of iron trafficking. It blocks the export of iron to the blood in a dose-dependent manner, which results in functional iron deficiency and decreased red blood cell production.
"Rusfertide shows great promise for achieving sustained hematocrit control in polycythemia vera patients. Just as importantly, it decreased the need for repeat phlebotomies, with some patients remaining virtually free of the procedure for more than two and a half years," notes Dr. Kremyanskaya.
A phase 3 clinical trial is now underway at global sites, with Mount Sinai again playing a leadership role. The researchers hope to confirm the long-term efficacy and safety of rusfertide in polycythemia vera patients and to explore its potential benefits in other blood disorders.
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