OUR RESEARCH IMPACT
MPN Research Highlights
- Funding six current pilot projects directed at assessing new therapeutic targets and risk biomarkers for disease progression – 2020 MPN Progression Pilot grants.
- Contributed to development and funding of ongoing multi-institutional retrospective study to understand impact of genomic testing on post-stem cell transplant outcomes for MF patients, led by Memorial Sloan Kettering – 2020 Emerging Trends research. Also supported development of Stem Cell Transplant Spectrum Timing Tool to guide patients and physicians around transplant timing.
- Funded early groundbreaking research showing MPN driver mutations were often acquired decades before diagnosis, suggesting possible opportunities to detect early changes and potentially intervene to prevent or slow MPN development – 2019 MPN Challenge grant – Jyoti Nangalia, MD, Wellcome Sanger Institute and University of Cambridge.
- Funded research on pivotal role of dysregulated iron metabolism in patients with PV. Research outcomes provide premise for ongoing clinical trial of PTG-300. 2019 MPN Challenge grant – Yelena Ginzburg, MD, Icahn School of Medicine at Mt. Sinai.
- Gained insight into understanding why and how interferon works and sometimes doesn’t work in MPN patients through three-year global MPNRF Interferon Initiative. Results released November 2021.
- Funded molecular target studies presenting strategy to selectively eliminate JAK2 mutated cells, using their own intracellular degradation machinery and sparing non-mutated JAK2. 2017 and 2021 MPN Challenge grant – James Griffin, MD, and Sara Buhrlage, PhD, Dana Farber Cancer Institute.
- Multiple research projects funded to understand role of inflammation, inflammatory mediators and bone marrow microenvironment in pathogenesis and progression of MPNs, laying groundwork for clinical trials targeting inflammatory pathways in MPNs – 2014 and 2017 MPN Challenge grants – Angela Fleischman, MD, UC, Irvine; Stephen Oh, MD, PhD, Washington University School of Medicine at St. Louis. Clinical Trials: An Optimal Dose Finding Study of N-Acetylcysteine in Patients With Myeloproliferative Neoplasms and Pevonedistat in Combination With Ruxolitinib for Treatment of Patients With Myelofibrosis
- Initial laboratory work funded by MPNRF made possible the first CALR vaccine-based MPN clinical trial in the US – an investment driven by understanding the potential impact such work will have when translated to the clinic. 2015 MPN Challenge grant – Camelia Iancu-Rubin, PhD, Icahn School of Medicine at Mount Sinai.
- Funded one of two teams that discovered mutations in CALR gene, found in the great majority of JAK2 and MPL negative MPN patients, leading to better understanding of why large subset of patients with ET and MF have the disease. The team continues to use these findings to develop CALR-based immunotherapeutic approaches. 2011 and 2014 MPN Challenge grants – Robert Kralovics, PhD research team, Austria.
- Pioneering work funded on use of barcoding to study clonal hematopoiesis in zebrafish. This system provides a more rapid approach to understanding why and how to control mutated stem cells from taking over bone marrow. 2014 MPN Challenge grant – Len Zon, MD, Harvard Medical School.
- Translational research funded into roles of TGF-beta and PIM kinase as therapeutic targets for myelofibrosis, supporting preclinical proof of concept for clinical trials now using this approach. 2012 and 2014 MPN Challenge grants – Amit Verma, MD, Albert Einstein School of Medicine and Gary Reuther, PhD, Moffit Cancer Center, respectively. Clinical Trials: MPN-RC 118 AVID200 in Myelofibrosis and A Study of Oral TP-3654 in Patients With Myelofibrosis
- Funded numerous grants to better understand and establish therapeutic targets and risk biomarkers for fibrotic transformation of chronic MPNs, including fibrotic transformation targets such as thrombopoietin, SHP-2, HMGA2, CXCL4 and PDGF-R, TNF-alpha, IL-1beta and NFkB. 2012, 2014 and 2017 MPN Challenge grants.
- Funded multi-year, multi-institutional MPD Research Alliance, enabling preclinical studies of JAK inhibitors in MPN animal models, MPN stem cell characterizations, and development of large-scale tissue bank and associated data bank, leading to rapid testing of new JAK2 inhibiting drugs – 2006 and 2009 MPD Research Alliance grants
Together We’re Making a Difference
We’re proud of the contributions we’ve made to the field of MPN research. But we know that we still have a long way to go. Sign up to receive the latest news and updates or donate now and join us in our efforts to advance treatments and ultimately, find a cure for MPNs.
