Driving groundbreaking research. Improving lives.
For 21 years, the MPN Research Foundation has delivered on a bold commitment to fund global pioneers studying innovative approaches to prevention, new therapies and improved quality of life for people living with essential thrombocythemia, polycythemia vera, and myelofibrosis.
Born of Necessity
The Internet was in its infancy when we were founded in 2000. People diagnosed with an MPN now had a place to go to look for information. But there was little to learn once they got there, because meaningful research was scarce.
We’re proud of where we started, where we are, and where we’re going. Our entrepreneurial attitude forced us outside the box from day one, and that continues to drive our unique impact − on MPN research, clinical treatments, biopharma drug development, and most importantly MPN patients.
Raised by Determination
Science is an exploration that has twists and turns, some exciting developments, and sometimes disappointments. The answers aren’t always front and center.
That’s why our commitment to collaboration is critically important. We find and fund investigators across the globe, across institutions and academic disciplines, and bring their questions and answers, successes and redirected interests to the collective MPN table. Then we ask what’s next and who is on board.
We’re determined to remain flexible, nimble, swift to adjust. If a direction isn’t proving productive, or better yet others are finding success there, we aren’t afraid to stop, take a breather, re-evaluate and better position our resources where most impactful.
Driven to Mind the Gap
We never lose sight of our roots. We were founded by patients, for patients. And this continues to drive everything we do.
What does that mean in practice? It means we don’t just fund promising research. We look at critical gaps in current research that may move answers from the laboratory to the clinic, faster. We play a uniquely independent role in the exploding MPN community, convening academia, industry, patients, advocates and regulatory agencies, all to improve the lives of people living with MPNs.
Our only end game is to be out of the game—because we can stop MPNs in their tracks.
mpn leaders on our research impact
“As we chart the course for the next 20 years, we will continue to convene global thought leaders to help steer our vision for future promise of disease treatment and prevention, and the clinicians, researchers, industry leaders, patients and their advocates will help us invest our funds for the most impactful outcomes. That means new treatments, new options, better quality of life, and ultimately prevention of progression.
We will continue to invest in our core principles for high risk, high reward research with near-term outcomes. At the same time, we’ll be setting our future on a path of innovation toward aspirational, long-term and sustainable outcomes for ET, PV, and MF patients.”
Kapila Viges, MPNRF Chief Executive Officer
MPN Research Foundation stimulates original research in pursuit of new treatments — and eventually a cure — for the blood cancers polycythemia vera, essential thrombocythemia, and myelofibrosis, known collectively as myeloproliferative neoplasms.
The foundation has played a key role in funding some of the more important work that has been done over the last two decades, translating ideas into meaningful impact for patients. For example, identification of some of the mutations that are involved in the disease, and specifically some of the driver mutations, these were in part funded by the foundation. We used to put all of our patients in one bucket, then say some are high risk, some are low risk. But now with the advent of all of the mutational work, it helps guide us in treatment decisions and outcome predictions.
Raajit Rampal, MD, PhD
Sloan Kettering Cancer Center and MPNRF Scientific Advisor
Message from Barbara Van Husen, MPNRF Board Chair
When in 2005 the single point gene mutation associated with MPNs was discovered (Jak2 V617F), we were convinced this would change everything. And, in fact, it has. From the challenge of finding someone who could spell myeloproliferative disorder (now neoplasm), MPNs are now among the hottest things on the blood cancer front.
Today, we have so many research thought leaders knocking on our proverbial doors, we have to decide with whom and how most effectively to invest our precious, hard won dollars.
For more than three years, we funded researchers to pound away at preclinical testing for JAK inhibitors. We funded very early full genome sequencing projects at a time when the human genome project first suggested this might be feasible. One of the projects we funded resulted in identification of a second mutation called Calreticulin (CALR), which we now know accounts for the one-third of MPN cases not associated with Jak2.
In fact, a significant number of MPNRF research projects resulted in major scientific findings. And some of the people who received our earliest awards to new investigators are now leaders in the field.
While we’re proud of every one of our wins, we won’t rest on our laurels. As most people who are involved with MPNs know, these are chronic, complicated, heterogeneous diseases. And until we are confident that all MPN patients have treatment options that will change their disease, we’re not done.
“In our early years, MPNs were on nobody’s radar. We placed ads in medical journals: ‘We want to fund research into myeloproliferative disorders. Come and get it!’ We expected a stampede for dollars. The first year, we had two submissions.”
When we held our first MPN Roundtable in 2012, we brought together investigators with very different perspectives. We just sat back and listened to the fireworks fly. The scientists said they had never been to a meeting like this, where across the table was a colleague who was approaching the same problems, possibly in completely different ways, yet they had not ever spoken. The dynamics and sidebar conversations of the annual roundtables remain extraordinary.
Barbara Van Husen, MPNRF Board Chair
Patient Impact - Cathy
Cathy’s grandkids know she has a ‘blood problem.’ They also know that a lot of dedicated individuals are working to understand how to make her better. In the meantime, despite the daily fatigue and a series of life-threatening blood clots, Cathy exemplifies why answers to preventing and treating symptoms and dangerous complications are as critical as a cure.
OUR RESEARCH IMPACT
We’ve awarded more than $16 Million for MPN blood cancer research since 2000.
This investment continues to accelerate our understanding of MPNs – their causes, prevention and progression. Our primary goal: the development of new treatment options, improving patients’ quality of life, and ultimately a cure.
FROM LAB TO CLINIC
Ruben Mesa, MD, UT Health San Antonio MD Anderson Cancer Center
Ann Mullally, MD, Brigham & Women’s Hospital/Harvard Medical School
Alison Moliterno, MD, Johns Hopkins Medicine
Raajit Rampal, MD, PhD, Memorial Sloan Kettering
It is unusual that we have been effectively using interferon for the treatment of MPNs for more than 30 years without fully understanding how it works. That is why the MPNRF’s Interferon Initiative is so important. Interferon appears to uniquely affect the MPN stem cells and has other valuable attributes.
Richard T. Silver, MD
Director Emeritus, Silver MPN Center at Weill Cornell Medicine
Our virtual boardroom is packed with pride. We both empower internationally renowned blood cancer researchers and support investigators during their early interest in MPNs, fortifying a lifelong commitment to MPN research. Our investments in their work lead to follow-on funding from both public and private sources, and in many cases, have positioned them as today’s leaders in the field.
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 Mount 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 grants – 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.
Patient Impact - Taja
Despite being told there were potential serious risks to a pregnancy with her PV, Taja followed the science, the advice of MPN specialists, and her dream. She gave birth to her daughter, Miracle, in 2020. In the year that followed, she was diagnosed with myelofibrosis, hospitalized twice for blood clots in her heart and lungs, and late in 2021 had a stem cell transplant, the only current cure for an MPN.
“This photo was a good day for me. The issue with MPN is that it is invisible and it’s hard for you or anyone to see exactly what you’re dealing with internally.”
Leaders don’t just set goals, they understand the power of results. That’s the ethos that defines the MPNRF. It’s what propels us. It’s an unwavering commitment to deliver on answers from the lab to the treatment room. And it keeps us laser focused on our mission.
What's next for the MPNRF?
We’re ready to drive another decade of tremendous impact.
We’ll focus on targeted laboratory, clinical and translational research funding and coordination of talented investigators around the globe, including both renowned MPN researchers and those just jumping in to explore these rare blood cancers.
We’ll gather them together, talk openly about what we’ve learned, and discuss how their groundbreaking work might translate to clinical trials — with hope for new and better treatments for MPNs.
We’ll actively support key research topics that have historically been underfunded. And we’ll keep at it until the questions we’ve been asking no longer remain unanswered.
We’re now leading a large research commitment to MPN progression. And as our understanding increases, we’re hopeful that new therapies down the road will be able to slow disease progression, or even prevent it. Our ultimate goal is to have little more to do – because MPNs are no longer a life-threatening blood cancer.
Brandon Goetzman, MPNRF Board Member
Invest. Convene. Advocate.
Yes, we’re primarily a research foundation, but we also take our commitment to advocacy to heart. We convene all types of stakeholders to better inform and advocate for the specific needs of people living with MPNs. It may be advocating for more research dollars into ET, PV or MF, or the urgency of approving more and better drug therapies. Our success reflects our broad reach and our commitment to bring together the voices of researchers and advocacy organizations from across the globe, government regulators, biopharma, and patients and their life and care partners.
The MPN Challenge is our open request for research proposals which are diligently reviewed by scientific peers and leading experts. The focus areas change each round so we can fund the most pressing MPN science and respond to new unmet needs. Since 2012, we’ve funded studies into immunotherapy, selective JAK2 inhibition, inflammation, interferon and more. Five project awards announced in 2021 will each receive up to $200,000 over two years.
This isn’t just another scientific meeting. Since our initial MPNRF Roundtable in 2012, it has become an event that invited MPN research stakeholders, including patients, look forward to each year. The unique gathering results in vital, sometimes unexpected questions and conversations, and ultimately important collaborations between leaders in academia and industry.
MPN Voice of the Patient
This Patient Focused Drug Development Meeting with the FDA was a long awaited platform for the MPN community to convey the challenges that ET, PV and MF patients face every day. Patients shared what it means to have an MPN diagnosis, which of their symptoms should be prioritized, and how their disease impacts their quality of life. A detailed report of the two-day meeting was delivered to the FDA in 2021, a collaboration with MPN Advocacy & Education International, The Leukemia & Lymphoma Society and MPN Cancer Connection.
Patient Impact - Ruth
When in 2019 MPNRF held an MPN patient focused informative meeting with the FDA, Ruth was on a patient panel. It was a first ever event, MPNRF advocating for more and better therapies for MPNs, and the first time Ruth shared her 30-year story of progression from ET to PV to MF. “I just want to dance at my son’s wedding,” she said. Three months later, she entered a clinical trial, then became a global MPN advocate. And in 2021, she beamed as her son Zach twirled her across the dance floor.
Our Progress Perpetuates our Continued Need
When Chicago businessman Robert Rosen was diagnosed with polycythemia vera in 1997, he had to search for information the old fashion way. He was shocked to discover how little research was conducted on myeloproliferative neoplasms – then called blood disorders.
So Rosen pulled together a small number of other MPN patients, established the MPN Research Foundation (then MPD Foundation) and set out to catalyze research leading to advanced treatments for people living with these rare blood conditions.
Today, after 21 years of MPN leadership and research, our investments have paid off with extraordinary progress. While we celebrate our impact, we commit ourselves to continue to drive answers to the biology, treatment and progression of MPNs.
“When my dad founded the MPN Research Foundation, little was known about these rare blood disorders and there was virtually no research being done. So he set out to change that. And the result 21 years later is astounding!”
Rebecca Rosen Shapiro