• MPNRF | July 3, 2004

    Summer, 2004

    I can’t believe it’s been five years since we started the MPD Foundation. Lately I have been reflecting on all we have done since our inception: raising money, building a volunteer staff, funding scientific research, and building a nationwide and worldwide community of people with similar interests. The question in all of our minds now is where we go from here, and how do we get there.

    We started with hope and not much else.

    We started the MPD Foundation towards the end of 1999 with several donations by board members, one research project, no public credibility, and lots of hope and hard work. Pretty much all we had was a blind faith that, somewhere along the line, we would gain public credibility and enough additional donations to support our ambitions. Many people expressed doubt about the whole enterprise, and specifically about our ability to make meaningful alliances with the LLS, or with Celia Miltz and Friends of ET. More were skeptical about the possibility of developing a web site, finding an executive director, and ever raising enough money to sustain and grow our organization in a meaningful way. It is now 2004 and together we have leapfrogged far ahead of all of these concerns.

    We’ve done a lot, with a lot of help.

    We have continually expanded the presence of the MPD Foundation and increased the number of medical research projects funded as a result of our efforts. Every year donations have increased. We are now responsible for 9 research grants and 2 organizational grants with a total dollar amount in play of approximately $3,000,000. Thanks to the critical evaluations of our esteemed medical advisory board, the grants we give are serious grants to serious researchers We have relationships with hematologists in America and elsewhere in the world. We work closely with some of the best MPD labs and professionals at highly regarded centers which include Mayo, John Hopkins, Baylor, and University of Ill in Chicago.

    In addition, we have been instrumental providing support money and energy towards the formation of the International MPD Research Consortium which is currently waiting for approval from the NCI for a $25 million MPD grant.

    We have assembled a remarkable volunteer board of directors where every member makes tangible contributions to our continuing success. The board has offered advice and consultation, provided gifts of money without which we cannot sustain our mission, and invested time and energy in a multitude of projects. Amy McMurray, our treasurer handles all the bookkeeping and correspondence, Woody Woodruff, with the assistance of Amanda Friedeman, makes our newsletters sleek and readable. Bob, Barbara, Celia, Sam, and David all contribute in meaningful ways throughout the year.

    I don’t know how we could have managed the last year without Felisse Sigurdson, our executive director. Her tireless energy and incisive mind have exponentially increased our ability to function effectively.

    We receive many hundreds of individual donations every year, some of them quite substantial. The grants we award are costly, and we cannot grow without this help.

    At last, for the first time, the Federal Government is taking an interest in the MPD’s; both the NIH and the Department of Defense are considering the possibility of making research money available.

    Many questions remain unanswered.

    However, our good news cannot make us complacent about the daunting task in front of us. Although many of us who are patients do well for long periods of time with these disorders, there is urgency for others and a great deal of uncertainty for all.

    There are issues that we can all relate to, and some issues which are specific only to certain patients. Blood clots remain a concern for almost everyone. Two people on our board have suffered serious, potentially life threatening thrombotic events. After all these years the medical community still does not know why MPD patients are prone to clotting, nor can anyone predict who is at the greatest of developing clots.

    There are newer medications in use; interferon, peg-interferon and Gleevec. We know these are active in many or most patients yet we do not have good enough information to determine long-term consequences, impact on longevity or why they help some patients and not others.

    There are no significant biomarkers to help understand who is at greatest risk and how to estimate longevity, or disease progression. Without good biomarkers treatments tend to be given without knowing which ones will work best for which patients. The doctors don’t agree on what drug treatments are appropriate for which disorders, and when and how to differentiate the needs between patients.

    Some of us have a disease called myelofibrosis which can be as urgent and acute as any malignancy anywhere, yet it is the focus of far too little primary research. We can hardly get decent research proposals on MF, yet I get calls at my office from recently diagnosed patients who want to know where to turn for help.

    Our patients see hematologists who misdiagnosis them, and who are not familiar with the MPD’s. We worry that there are not enough young researchers coming through the ranks to insure the best future scientific research and care.

    There has not been a large scale study group since the Wasserman study over 30 years ago. Enormous progress was made as a result of that work, and we need to do it again in the light of recent scientific progress in our understanding of the human genome. In spite of the efforts of Dr. Prchal and others, we do not yet have a conclusive handle on the genetic defects and molecular characteristics which cause MF, PV or ET. Without these we cannot have targeted therapies.

    Because the NIH does not track incidence and prevalence data for the MPD’s there is no credible data from which to establish priorities with potential funding sources to compete for pubic and private research dollars.

    We get donations from only a small minority of people who are on the MPD Foundation mailing list.

    Looking forward

    I was diagnosed with PV in the fall of 1997 after experiencing unexplained neuropathies in my toes and fingers for almost a year. At the time of diagnosis my hematocrit was 60 and the doctor asked if I had any pains in my chest or other heart symptoms. I was emotionally devastated for many months, and my family had difficulty coping with the fact of my illness. I couldn’t believe that in this day and age, there were no advocacy groups acting effectively to promote medical research in the MPD’s.

    With the passage of time I was able to regain my balance and direct my frustration into something constructive. With the help of many good people we are moving forward towards a time of better understanding and inevitably better treatments for our conditions.

    We are grateful for your support.

    Robert Rosen