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MPD RESEARCH ALLIANCE: HEADING FOR NEW TREATMENTS
For the first time ever, leading MPD researchers from three renowned institutions have joined forces to accelerate the development of new treatments for polycythemia vera, essential thrombocythemia and primary myelofibrosis.
On April 1, 2006, the MPD Foundation awarded $750,000 to fund the first year of the MPD Research Alliance. The lead investigators are Gary Gilliland, PhD, MD, Harvard Medical School; Ronald Hoffman, MD, PhD, Mt. Sinai School of Medicine, New York; and Ayalew Tefferi, MD, Mayo Clinic, Rochester.
An Ambitious Goal: Extending Our Lives
The primary goal of the MPD Research Alliance is to accelerate the development of new targeted therapies that will materially benefit MPD patients. These treatments will address all the Philadelphia chromosome negative myeloproliferative disorders (essential thrombocythemia, polycythemia vera and primary myelofibrosis) and include patients with and without the JAK2 mutation. (In 2005, researchers made a major breakthrough by discovering a genetic mutation - technically known as JAK2 V617F - that occurs in most PV patients and many ET and MF patients.)
Research Alliance Priorities
Identify the best candidates for development among compounds available today for JAK2 positive patients, and quickly move them through the drug development pipeline and into clinical trails for JAK2 positive patients.
Identify and validate new targets in MPDs that are JAK2 negative and develop new compounds that are effective against these targets.
Initiate Phase I and Phase II clinical trials to provide a better understanding of which compounds work best in which patients.
We plan to work with biotechnology and pharmaceutical companies to carry the most promising compounds through the large and expensive Phase III trials required for FDA approval.
Highly Qualified Independent Advisory Board
In support of the MPD Research Alliance, the MPD Foundation has recruited an impressive Scientific Advisory Board to provide an independent assessment of research progress against objectives, and to make recommendations where improvements or changes may be needed.
A Different - and Better - Approach to MPD Research
The MPD Research Alliance is a novel approach to MPD medical research because of the unusual collaboration of researchers at three different institutions. Also, many traditional research models are aimed at advancing the science without a specific focus on improved treatments. Key to this effort is that patients are involved in the process. In addition to raising money, widespread patient involvement provides researchers with easy access to a broad range of tissue samples and volunteers for clinical trials.
"The traditional model of medical research has not been yielding progress nearly fast enough," says Robert Rosen, President of the MPD Foundation and an MPD patient. "The existing treatments for MPDs were originally developed to treat other diseases, and they all can have severe side effects. Our goal is to make new targeted treatments available that will help current patients as well as future generations."
