How MPN patients have accelerated research for PV, ET, and MF

By Andrew Schafer, MD, Chairman of Medicine, Weil Cornell & MPN Research Foundation Scientific Advisory Board Chair

Since 2000, the MPN Research Foundation has provided nearly $9 million in research funding to advance the treatment of the myeloproliferative neoplasms (essential thrombocythemia, polycythemia vera and myelofibrosis.) It is a modest amount relative to the magnitude of the problem and the scale of research needed to tackle it. But the Foundation has had a remarkable track record of success in optimally allocating these limited funds to achieve the highest impact for MPNs. In fact, the Foundation has provided key support for virtually every major advance that has been made in the MPNs since 2000.

NIH funding gets harder to come by every single year, more so for diseases like MPNs, which are less “glamorous”. But our grants have created the foundation on which researchers have gone on for advanced funding from the NIH to take their projects to the next level. It has also helped launch the MPN research careers of our currently most productive investigators in the field, as well as incentivizing already successful scientists in related areas to turn their attention to curing the MPNs.

The following is a list of researchers funded by the MPN Research Foundation, and by extension, patients themselves. If you're inspired by this, please consider becoming a member of the Foundation. It's free and will keep you up to date with the latest MPN community and research news.


 Dr. Josef Prchal (2000-2002)               Used Foundation support to become the first to discover a loss-of-heterozygosity of chromosome 9q in polycythemia vera, which provided the basis for the subsequent discovery of JAK2-V617F, the diagnostic mutation which is now known to be located on chromosome 9q.
 Dr. Robert Kralovics (2011-2013)  A student of Dr. Prchal, subsequently became himself a major innovator in MPN research and has received independent Foundation support to advance the genomics of MPN. Dr. Kralovics was among the first to identify the JAK2-V617F mutation in a widely cited, first-authored article in the New England Journal of Medicine.
 Dr. Ronald Hoffman (2003-2011)  Is a world-renowned hematologist who made several key discoveries during his Foundation funding period in defining the molecular underpinnings of the MPNs and then using this basic knowledge to develop and test novel agents in preclinical studies.
 Dr. Ruben Mesa (2003-2005) Was trained by Dr. Tefferi, is making the first breakthroughs in the treatment of myelofibrosis, including leading the investigation of the clinically effective JAK1 and JAK2 inhibitor, ruxolitinib.
 Dr. Ayalew Tefferi (2006-2011)  Established the leading biobank for peripheral blood and bone marrow samples from consenting MPN patients, which has been used as the indispensable core facility for genetic studies of MPNs by investigators throughout the world.
Dr. Francois Delhommeau (2009-2011) Worked in Dr. William P. Vainchenker’s lab, where he was involved in developing a novel liquid culture system to grow polycythemia vera erythroid cells in the absence of added growth factors; this was the technical advance that allowed the development of JAK2 inhibitors. Dr. Delhommeau has been searching for the “driver mutations” of MPNs and reported in the New England Journal of Medicine during his Foundation funding period that TET2 mutations in the stem cells of MPN patients precede the JAK2-V617F mutation.
 Dr. Gary Gilliland (2006-2008) He made the discovery of JAK2-V617F simultaneously and used Foundation support to pioneer our understanding of the genetic basis of MPNs. Dr. Gilliland, in turn, trained Dr. Ross Levine (2011-2013), discoverer the MPLW515 mutation of the thrombopoietin receptor, Dr. Ben Ebert (2011-2013), and Dr. Ann Mullally (2012-2013), all of whom in their own rights have developed into world-class, independent researchers in the MPNs.
MF Challenge Grantees  During the past two years, the Foundation has partnered with the Leukemia & Lymphoma Society to, for the first time, focus a grant program exclusively on myelofibrosis called the MF Challenge. This is perhaps the most poorly understood and stubborn of all the MPNs, and conversion is a concern to those with PV and ET. We’re awaiting progress reports for year 1 of the MF Challenge while we ramp up to start funding the 2nd year’s grants.


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