From Lab to Clinic and Back

  • MPNRF | March 23, 2022
    Yelena Ginzburg, MD, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai

    Dr. Yelena Ginzburg’s research illustrates how basic research, clinical data, and clinical trial development inform each other in what is often a continuous loop between lab and clinic.

    Funded by a 2019-2021 MPN Challenge award, her work focuses on understanding why iron deficiency is regulated differently in polycythemia vera. 

    Specifically, it looks at how red blood cell formation remains high, despite iron deficiency, and if iron metabolism is dysregulated in JAK2 mutated erythroblasts, the precursors of red bone marrow.

    Two presentations during ASH-2021 (the annual meeting of the American Society of Hematology) highlighted the promise of rusfertide in the treatment of PV, including the results of a phase 2 clinical trial that showed its safety and tolerability, while effecting hematocrit control with a reduced need for periodic therapeutic phlebotomy. 

    Rusfertide is a synthetic mimicker of the natural hormone hepcidin, which reverses iron deficiency while controlling hematocrit. Dr. Ginzburg’s work may help shed light on why rusfertide has been shown to be effective at reducing or eliminating a patient’s need for phlebotomies over time.

    Dysregulated iron metabolism plays a pivotal role in polycythemia vera

    As trials with rusfertide continue, Dr. Ginzburg postulates that in addition to minimizing phlebotomy, a reduction in thrombotic events may also be seen in patients taking this drug. 

    A phase 3 global, multicenter, randomized trial is underway which aims to enroll approximately 250 PV participants to assess the primary end points of a confirmed hematocrit of 45% or less. 

    Dr. Ginzburg recently spoke with Targeted Oncology about the basis and promise of rusfertide’s effectiveness.

    “I think the main point that we are hoping to see with the trial as it continues,” says Dr. Ginzburg, “is both a prolonged continuous either phlebotomy-free or phlebotomy-reducing effect of using rusfertide in the chronic setting that can also ultimately allow us to evaluate a potential reduction in the thrombotic events in this patient population.”