"After my diagnosis I thought I could use my experience in business to promote MPN research and create scientific activity where very little had previously occurred." - Robert Rosen, Chairman, MPN Research Foundation

Strategy for 2014

In 2013, MPNRF rededicated itself to its primary mission: to stimulate original research in pursuit of new treatments and eventually a cure for the MPNs. During 2013, the MF Challenge continued into its second year, with 4 more awards made in partnership with the Leukemia & Lymphoma Society. To further bridge the gap between MPN academic and company researchers, MPNRF also hosted the first MFC Forum, at which academic and company scientists met to discuss research results for the MF Challenge to date and to identify the still-unmet need in MPN science.

Building on New Discoveries

Our strategy for 2014 builds on these results, and takes into account a number of game-changing recent developments in MPN and cancer research. These discoveries include:

• The discovery of a new mutation (CALR) associated with most JAK-negative MPN patients, opening a new avenue of potential treatment for these patients.

• Discovery and initial testing of new drugs based on new mechanisms of action (other than JAK2). These new drugs have potential by themselves or in combination with JAK inhibitors to greatly improve ET, PV and MF treatment.

• Advances in other cancers that may have applicability to MPNs, including immunotherapy and anti-fibrotic agents. It is our hope to bring these advances to MPN treatments.

As the MF Challenge moves into its third year, we are incorporating these new discoveries into the program by calling for proposals in five focus areas, making this year’s grant program the most ambitious and focused in the history of the Challenge.

Turning Up the Heat

In addition to requesting applications in specific focus areas, we believe the time is right to significantly increase our research efforts. In 2013, one of the leading JAK inhibitors failed in final testing, reducing the number of JAK alternatives and suggesting intrinsic limits in many JAK drugs. This, in tandem with the rapid development of alternative approaches, suggests that moving new discoveries faster is increasingly important. We therefore are raising our target from the 4 grants awarded in 2012 and 2013 to a possible total of 10 in 2014, assuming enough applications that merit such funding.

The MF Challenge Gets a New Name

When the MF Challenge was begun in 2012 our focus was simple: to identify the cause(s) and potential treatments for fibrosis in the bone marrow, the key characteristic of myelofibrosis (MF). We began this program because in spite of the current high level of activity in MPN research, there were important questions that remained unanswered – and the most urgent were those associated with MF, the most virulent of the MPNs.

However, we have realized that much of the research focused on MF overlaps research for PV and ET. In fact, it’s frequently hard to tell where one begins and the other ends. In all cases research is focused on some malfunction in the bone marrow, and discoveries in one area are highly likely to impact all the MPNs. As a result, we are renaming our grant program the 2014 MPN Challenge.

The goal of the 2014 MPN Challenge is to continue to search for the factor(s) that induce(s) fibrosis in bone marrow, and in addition to identify new avenues of research which will strengthen the overall understanding of the cause(s) and potential treatments for all of the MPNs (ET, PV and MF). We also encourage investigators in other fields whose research may have cross-over appeal to the MNP research community.

Summary

The MPN Research Foundation continues to look forward to the day we can stop sending fundraising letters, close the door, and turn off the lights. Our assessment is that in spite of encouraging advances in science, that day is not here yet. We look forward to 2014 as a year of great progress, and will do ou best to contribute to that progress. Please join us by making a donation to the Foundation at www.mpnresearchfoundation.org.

 

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