Strategy 2013

The MPN Research Foundation is rededicating itself to its roots: to stimulate original research in pursuit of new treatments and eventually a cure for MPNs, for the benefit of patients and their families. Our financial goal is to increase our fundraising significantly, to substantively accelerate the discovery of new treatments and potentially a cure. Without this investment, we believe important areas of research will be missed, and the potential MPN scientists of the future will direct their energies elsewhere.

The MPN World Has Changed

It’s hard to believe today that in 2000, when the MPN Research Foundation began its work, MPNs were largely ignored by the scientific community. The MPNs were often under- or misdiagnosed, and the first patient groups to take advantage of speaking about MPNs on the internet were frustrated by the lack of understanding among clinicians and the lack of treatment alternatives. It was in this world that the MPN Research Foundation made its first grant in 2000.

The next several years saw slow but steady progress in MPN science, but it was not until 2005, when the JAK2 mutation associated with so many cases of ET, PV and MF was discovered that things really took off. And the MPN Research Foundation was ready to invest moving this discovery forward by funding the preclinical testing that is now leading to FDA approved treatments that were a dream only a few years ago. With a total of 38 grant awards, many multiple year awards totaling almost $9 million, the Foundation has been a powerful force in MPN research.

But as rapid as this progress has been, we all know the work is far from over. Emerging drugs improve patient conditions but do not (yet) alter the course of the disease. Patients are still concerned about disease progression and the potential for conversion to acute leukemia. And the only true cure is still bone marrow transplant, a high-risk procedure that is not available to all MPN patients.

On the positive side, there are now many academic researchers working on these questions. And with the emergence of new FDA-approved drugs for MF, industry is now mobilizing to invest in MPN science. And most of all, patients have become increasingly engaged in the search for solutions, using online support groups, social media, patient seminars and support groups to communicate with each other and with the scientists and companies who will ultimately change their prognoses.

Time to Re-assess/Recalibrate/Refocus

As the MPN world, once so quiet and empty, becomes more filled with activity, the MPN Research Foundation felt the need to review its activities to determine what our mission should be in the year and years to come. We began this process by initiating a survey sent to patient groups, scientists, clinicians, and bio/pharma contacts. In the survey we asked responders to identify the unmet needs in MPN science and treatment, and to recommend where the Foundation’s energy should be focused. The response was strong and the results were clear; the messages we heard included the following:

  • That the Foundation must continue to fund innovative research, especially with new investigators, until effective treatments are delivered to patients. Reduced funding by NIH and other sources makes our dollars, focused specifically on MPNs, critical.
  • That the Foundation should work to bring scientists, clinicians, industry and patients together to identify unmet needs and opportunities, and to disseminate information broadly and clearly.
  • That the Foundation should move aggressively to raise the funds needed to have a significant impact on the advancement of research.

Along with these broad themes the survey elicited many other suggestions and comments. The Foundation met during the Fall to discuss strategies for moving forward, and as a result faces 2013 with a new energy and a sharpened focus.

A Commitment to Advancing and Accelerating Research

The MPN Research Foundation is rededicating itself to its roots: to stimulate original research in pursuit of new treatments and eventually a cure for MPNs, for the benefit of patients and their families. Our financial goal is to increase our fundraising significantly, to substantively accelerate the discovery of new treatments and potentially a cure. Without this investment, we believe important areas of research will be missed, and the potential MPN scientists of the future will direct their energies elsewhere.

In order to maximize our focus on funding researching we plan to work with other groups who bring patients together and provide education and support (rather than duplicating those efforts), so that the most comprehensive information is available to the greatest number of patients. We will promote the activities of these groups and encourage their input into our research agenda. We want all MPN patients to feel and act as a single, powerful force for change.

We are also determined to bridge the gap between academic science and the biotech and pharmaceutical companies which ultimately deliver solutions to patients, through information exchange and identification of appropriate private/public partnerships. We are following in the footsteps of other successful patient-run organizations in bringing these two forces together, and we have high expectations of the resulting acceleration of progress.

Plans for 2013

The MF Challenge, which we started funding in 2012, represents our unwavering focus on innovative, meaningful research. We have awarded grants to 4 researchers who presented innovative proposals for new concepts for stopping and reversing fibrosis in MF patients. In 2013 we will solicit additional new proposals, and will review progress of the first grants towards the end of 2013. It is our hope that these projects, in combination with MF drugs already in development, will provide real answers for MF patients.

But the MF Challenge is only one, not the last, of the focused grant programs we believe must be initiated with our support. In order to determine our next research target, we will convene representatives from all MPN constituencies (academic scientists, clinicians, industry representatives, and patients) to identify unmet needs that the Foundation’s efforts can significantly affect, and which other organizations are not addressing.

Summary

The MPN Research Foundation looks forward to the day we can stop sending fundraising letters, close the door, and turn off the lights. Our surveys and strategy sessions this year have convinced us that although there is much to be hopeful for, that day is not here yet. We look forward to 2013 as a year with great promise, and will do our best to contribute to that progress. Please join us by making a donation to the MPN Research Foundation. Happy New Year to you all.

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