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The State of Research |
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For many years medical research into the cause and treatments of MPD's had been limited. The last full scale medical research study, the Polycythemia Study Group (PVSG) was completed over 30 years ago. Many important medical decisions for treatment of the MPD's are based on information from this study. This strongly suggests the need for more current research, particularly in the light of new understanding about the human genome.
Prior to the founding of the MPD Foundation, there were no advocacy groups
working effectively to stimulate and support research in this area. The
foundation was started in an effort to rectify this situation. Since inception
the Foundation has awarded 15 medical research grants, an epidemiology
grant, and two organization grants. More than $5 million has been raised
of which more than 95% has been put to work on behalf of MPD patients
and their families.
In the last few years it is clear that the world of MPD research has started
to attract a great deal more attention. Then the recent discovery of the
JAK2 V617F mutation gene has ignited a great deal of new and exciting
scientific discourse on the genetic causes of the MPD's and much speculation
on the possibility of finding new genetically targeted drugs and therapies.
JAK2 V617F is found in over 90% Polycythemia patients and approximately
50% of Myelofibrosis and Essential Thrombocythia patients.
In addition, the NIH has recently awarded a $19.6 million grant to an international consortium which will focus on understanding the genetic underpinnings of these diseases, the effectiveness of existing therapies, and the acceleration of basic research. The foundation is proud of the two organizational grants it awarded to this group in the early stages of application for the grant and many members of the consortium continue to have close working relationships with many members of the MPD Foundation.
The recent formation of the MPD Research Alliance marks a major initiative to accelerate the development of new drugs for the MPD"s. Patient involvement at all stages of the process keeps our focus on the extension of life expectancies and the improvement of patient quality of life. Funding for the Research Alliance commenced in early 2006. The Research Alliance is funded by the MPD Foundation which in turn is funded by donations from MPD patients and their families and friends.
In spite of the growing recognition that intensified research into these diseases is both necessary and appropriate, there are still significant barriers which limit the potential effectiveness of this research.
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Insufficient numbers of patients area available at a single medical institution to permit the pursuit of phase III trials, or rapid completion of phase I/II trials. |
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Clinical and bench research expertise in these disorders is limited to a small group of individuals at a limited number of institutions worldwide. |
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Large cooperative cancer treatment groups are not traditionally interested in chronic malignancies and do not provide an appropriate environment for the pursuit of basic research in such disorders. |
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There is a lack of clinical biomarkers to assess prognosis of MPD patients and to stratify patients for appropriate therapeutic interventions. |
