The Myeloproliferative Disorders Foundation has awarded $750,000 to establish the MPD Research Alliance, a collaboration of the University of Illinois at Chicago, Harvard Medical School and Mayo Clinic to accelerate drug development for incurable, progressive bone marrow disorders.
The Foundation plans to donate $10 million to the Research Alliance over the next five years to advance treatment options for the myeloproliferative disorders polycythemia vera, myelofibrosis and essential thrombocythemia.
Myeloproliferative disorders are an overproduction of platelets, white blood cells, or red blood cells in the bone marrow, which may result in serious complications that include enlargement of the spleen and liver, anemia, bleeding, stroke, heart attack or leukemia. The survival rate varies greatly, from 3 to 20 years, depending on the type of disorder and which complications ensue.
"The traditional model of medical research has not yielded significant progress fast enough," says Robert Rosen, an MPD patient and president of the MPD Foundation. "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."
The MPD Research Alliance was conceived to bring together leading MPD researchers to advance scientific knowledge and discovery, focus on developing new treatments, and involve patient advocates.
Research teams led by Dr. Ronald Hoffman, professor of medicine at UIC; Dr. Gary Gilliland, associate professor of medicine at Brigham and Women's Hospital and Harvard Medical School; and Dr. Ayalew Tefferi, professor of medicine at Mayo Clinic College of Medicine; will work to expedite drug discovery and clinical trials.
"By working together, instead of competing, we will ensure a streamlined research process among the three institutions," said Hoffman. "During the next three to five years I expect we will have identified potential compounds for clinical study in humans."
In 2005, researchers made a major breakthrough by discovering a genetic mutation, known as JAK2, in many MPD patients.
The goals of the Research Alliance include identifying additional genetic mutations that may cause myeloproliferative disorders, conducting pathogenic studies, identifying existing drugs with the potential to treat MPD disorders, and identifying novel compounds for pre-clinical studies and, eventually, clinical trials in humans.
"MPDs are classified as orphan diseases because they affect fewer than 200,000 people," said Rosen. "These disorders have been noticeably underserved with respect to research funding, but we now have the ability to change the future for people with MPDs."
The MPD Foundation was established in 1999 by a group of patients who began raising funds to support research. The Foundation has raised over $4 million and has awarded 12 multi-year medical research grants.