A breakthrough in the treatment of polycythemia vera, a rare and life-threatening blood cancer, has emerged with the introduction of a new drug named rusfertide.
Polycythemia vera falls under the category of chronic myeloproliferative neoplasms, which disrupt the normal production of blood cells within the bone marrow.
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In a clinical trial conducted by researchers from the Icahn School of Medicine at Mount Sinai, rusfertide showcased promising outcomes among 70 patients dependent on phlebotomy, a procedure to reduce blood volume.
Administered over 28 weeks, the drug effectively curbed the excessive production of red blood cells, the primary culprit behind the disease's complications.
Published in The New England Journal of Medicine, the trial demonstrated rusfertide's potential to decrease the necessity for phlebotomy while enhancing patients' quality of life, all while maintaining a favorable safety profile and high tolerance among participants.
Rusfertide emerges as a groundbreaking pharmaceutical intervention, acting upon hepcidin, a pivotal protein governing the body's iron levels. By augmenting hepcidin levels, rusfertide curtails iron accessibility for red blood cell synthesis, thereby diminishing red blood cell counts and mitigating polycythemia vera symptoms.
Dr. Marina Kremyanskaya, Associate Professor of Medicine and Medical Director for inpatient oncology at The Mount Sinai Hospital, who led the study, expressed optimism about the potential of rusfertide in revolutionizing treatment for polycythemia vera patients.
Dr. Kremyanskaya emphasized its novel mechanism, which restricts iron availability crucial for blood cell production. She underscored the importance of further clinical investigations, suggesting that this injectable agent could offer much-needed relief for patients grappling with the challenges of polycythemia vera management.
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Polycythemia vera falls under the umbrella of chronic myeloproliferative neoplasms, a spectrum of conditions influencing blood cell production within the bone marrow. Specifically, this disorder entails an overproduction of red blood cells, resulting in blood that is excessively thick and adhesive.
Consequently, individuals with polycythemia vera face heightened risks of blood clotting, heart attacks, strokes, and other severe complications. This condition is relatively rare, affecting an estimated 1-3 individuals per 100,000 in the United States, typically manifesting in older adults.
Current treatment modalities encompass aspirin, medications aimed at reducing red blood cell counts, and phlebotomy. Nonetheless, these approaches exhibit limitations and adverse effects, leaving many patients with inadequately managed conditions and unfavorable prognoses.
Findings of the Study
Researchers discovered that rusfertide effectively controlled hematocrit levels below 45% over 28 weeks and outperformed a placebo during a 12-week withdrawal period. Patients with symptoms like itchy skin, night sweats, difficulty concentrating, and fatigue experienced improvements.
Rusfertide mimics hepcidin, a liver-produced hormone regulating iron trafficking. It reduces red blood cell production by blocking iron export to the blood.
Dr. Kremyanskaya emphasizes rusfertide's potential to sustain hematocrit control and decrease the need for repeat phlebotomies. A phase 3 clinical trial, led by Mount Sinai, aims to confirm its efficacy and explore benefits in other blood disorders.
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