March 2022
Under the Microscope
From Lab to Clinic and Back
Research Spotlight on Yelena Ginzburg, MD, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai 

Dr. Yelena Ginzburg's research focuses on how iron metabolism is regulated differently in polycythemia vera (PV). Specifically, it looks at how red blood cell formation remains high, despite iron deficiency, and how iron metabolism is altered in JAK2 mutated erythroblasts, the precursors of red bone marrow. This work was funded by an MPNRF 2019-2021 Challenge award.
 
Her work illustrates the continuous loop that often informs and connects lab and clinic. What's observed in humans (serum ferritin levels, blood counts, red blood cell size and hemoglobin content, timing of changes, etc.) can provide leads for how normal function can be altered in different diseases, for further study using patient samples and mouse models of polycythemia vera. At the same time, what she learns in the laboratory contributes to the design and development of clinical trials. 
 
Currently in phase 2 and recently started phase 3 clinical trials, rusfertide is a synthetic mimicker of the natural hormone hepcidin, which regulates iron availability in humans. Because in polycythemia vera the majority of iron is used for hemoglobin synthesis, causing iron deficiency in all other organs, adding rusfertide reverses iron deficiency systemically 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 PV patient's need for phlebotomies, and why it may ultimately also lead to a reduction in thrombotic events. READ FULL STORY 
ICYMI Webinar on Progression
The science of progression was the subject of a recent webinar hosted by MPN Advocacy & Education International, in partnership with the MPN Research Foundation. The recording is now available, featuring Raajit Rampal, MD, PhD, of Memorial Sloan Kettering Cancer Center, and MPNRF Chief Executive Officer Kapila Viges. Dr. Rampal serves as the foundation's medical advisor, including for the MPN Progression Network. zzzzzz

MPN Progression: What We Know and What We Hope to Learn
ESH MPN Conference Now Virtual
The European School of Hematology's 9th Translational Research Conference on MPNs will now be held virtually May 12-15th, with no charge for registration and content access until June 15th.

The conference brings together scientists and clinicians worldwide to present and discuss
new data on the cellular and molecular biology of MPNs, as well as their diagnosis and
treatment. The scientific program is designed to encourage close interaction between
global MPN authorities and registered participants, some of whom received scholarships
from the MPNRF to attend, originally an in-person conference in Budapest. 

Specific topics will include:
• Molecular pathogenesis of MPNs 
• Novel MPN therapeutics 
• MPN initiation and clonal evolution 
• Murine MPN models and pathogenesis/therapeutic studies 
• Molecular pathogenesis and therapy of MPN progression and leukemic transformation 
• Recent advances in therapeutics 
Save the Date: Patient Webinar on Updated National Comprehensive Cancer Network Guidelines for MPNs 
Updated clinical practice guidelines are critical to quality cancer care, but for patients, it can be challenging to understand the changes and impacts. To add clarity, the MPN Research Foundation is sponsoring a patient guide to the NCCN Guidelines for MPNs updated in January 2022, which is being prepared by the NCCN Foundation. On April 13th, one of our valued board members, Stephanie Cindric, ARNP, will speak to the updates and implications of the new patient guide in a free webinar hosted by NCCN.  
Clinical Research Trial Highlights
Elotuzumab 
Sponsor: MD Anderson 
Phase 2 trial for MF patients with JAK2 mutation who are able to travel to the trial site in Houston, TX. Elotuzumab is an anti-SLAMF7 monoclonal antibody that was previously approved by the FDA to treat another blood cancer. 

For a full list of currently recruiting MPN trials, view this list on our website.  
Bomedemstat 
Company: Imago 
Phase 2b trial of an LSD1 inhibitor for ET patients who have high platelets and did not benefit from at least one standard therapy. 
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