A Cure in Focus

A Cure in Focus
cureJAK2 Mutation CALR Mutation Fibrosis & the Bone Marrow Niche MPN Allele Immunotherapy MPN Science
In 2013 the MPN Research Foundation convened a group of scientists from academia and industry to ask two questions: What is the state of MPN science, and what can MPN Research Foundation do to accelerate discoveries that will lead to new and better treatments for PV, ET, and MF? The ensuing discussion crystalized around five concepts which became the focus of the MPN Challenge grant program. This year we awarded $1 million to ten projects focusing on these five cutting-edge areas of research all of which show promise helping to find a cure for PV, ET, and MF.
Please click on any focus area at left for more information on each area and grant recipients.

With this focus area we want to help find clues about how the JAK2 mutation affects your body's blood production system. Current JAK2 inhibitors are not selective because they inhibit both the mutated and unmutated JAK2 gene and inhibit the body's ability to create needed blood cells. If these projects are successful, the new treatment options that arise would inhibit the over production of blood cells, but allow the body to produce blood cells when needed.
 
Grantees

Jean-Luc Villeval, PhD of INSERM (France); Sandra Pellegrini PhD and Stefan Constantinescu MD, PhD of Universite Catholique de Louvain (Brussels)
Gary W. Reuther, PhD of Moffitt Cancer Center

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In 2013, research partly funded by the MPN Research Foundation revealed a previously unknown mutation involved in MPNs: the Calreticulin or CALR exon 9 mutation. In addition to contributing to our basic understanding of how MPN develops and progresses, the study of CALR could lead to new treatments for PV, ET and MF. Because CALR is found close to the surface of the myeloid cell, it may be relatively easy to target. These projects could results in the development of antibodies that target the CALR mutation in the hopes of creating a new treatment for CALR mutant derived MPNs.
 

Leonard Zon, MD of Boston Childrens Hospital
Jean-Luc Villeval, PhD of INSERM (France); Sandra Pellegrini PhD and Stefan Constantinescu MD, PhD of Universite Catholique de Louvain (Brussels)
Robert Kralovics, PhD of CEMM, Austria
Michael W. Deininger, MD, PhD; Thomas O'Hare, PhD; Djordje Atanackovic, MD; Tim Leutkens, MD of University of Utah Cancer Center

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Understanding the role the bone marrow environment or niche plays in the formation of fibrosis is key to understanding what causes and how to treat myelofibrosis. We know that changes in the stem cells can contribute to leukemia and MPNs. But we don't know how these changes occur. There are potential therapies waiting to be discovered in this microscopic environment. In addition to unlocking new opportunities for treatments, one of these proposals is exploring whether non-invasive imaging can be used in place of a bone marrow biopsy to confirm fibrosis.
 
Grantees

Leonard Zon, MD of Boston Childrens Hospital
Angela Fleischman, MD, PhD and Richard A. Van Etten, MD, PhD of University of California, Irvine
Lei Deing, PhD of Columbia University School of Medicine
Katya Ravid, MD of Boston University School of Medicine

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In a subset of MPN patients, interferon has shown the ability to reduce the mutant allele burden, be it JAK2 V617F or other disease alleles which mark the disease clone. How does Pegasys target the JAK2 mutation? Does it also target CALR positive hematopoetic stem cells? Why are some patients resistant to Pegasys? We hope these researchers will discover answers that lead to a better understanding of this important treatment option.
 

Leonard Zon, MD of Boston Childrens Hospital
Angela Fleischman, MD, PhD and Richard A. Van Etten, MD, PhD of University of California, Irvine
Jean-Luc Villeval, PhD of INSERM (France); Sandra Pellegrini PhD and Stefan Constantinescu MD, PhD of Universite Catholique de Louvain (Brussels)

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Your immune system is your body's own defense against illness. Today, cancer biologists are learning how to harness the body's own immune system to fight back against the catalysts that cause cancer to form. Some subset of MPNs can be effectively treated with interferon (Pegasys), which works with your immune system. In some cases of CML, cures are possible using adoptive T-cell transplantation. We solicited proposals for immunotherapeutic approaches to the broader spectrum of MPNs.
 

Angela Fleischman, MD, PhD and Richard A. Van Etten, MD, PhD of University of California, Irvine
Robert Kralovics, PhD of CEMM, Austria

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Since opening our doors in 2000, the MPN Research Foundation has provided $10 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, but through smart investment weve been involved in nearly every major breakthrough in the MPNs since we began funding research. The Foundation has had a remarkable track record of success, including funding the discovery of the CALR mutation, preclinical testing of JAK inhibitors, attracting experienced investigators to research MPNs, and providing vital funding to investigators to encourage them to stay in the field of MPNs. In an era of unprecedented cuts to NIH funding, as the only organization solely dedicated to funding research into PV, ET, and MF, it is essential to keep pace with the demands of people everywhere living with an MPN.

For more information on the research we've funded, please visit our Research Funded page.

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