October 29, 2007
Major donation boosts muscular dystrophy
research efforts
A $7.1 million gift to the Medical Center from
New York developer and philanthropist Richard Fields will create a
research and clinical center of excellence for facioscapulohumeral
dystrophy (FSHD), a common form of muscular dystrophy. The gift,
the largest private donation for a specific disease program in the
Medical Center’s history, was announced by Medical Center CEO
Bradford Berk during a press conference on October 25.
“I want to express my deep gratitude to
Richard Fields for his generous gift and his vision to bring
together the people and resources necessary to find new ways to
understand, treat, and perhaps even cure this
disease,” says Berk. “Our scientists
strongly believe that new breakthroughs are within our grasp, and
Richard’s support will help catalyze their efforts.”
The new Fields Center for FSHD &
Neuromuscular Research will be an international collaboration
between the Department of Neurology and Leiden University Medical
Center in the Netherlands. The long-term, uninterrupted funding
will accelerate what has generally been an uncoordinated and
fragmented research effort. It will also enable the Medical Center
to develop the clinical programs that will make the University a
national referral center for patients with the disease.
“This is a very difficult disease to
figure out in terms of what is going on,” says neurologist
Rabi Tawil, director of the Fields Center. “Research has been
hampered by the fact that there are very few centers involved, and
the centers that are don’t have access to patients and
patient resources necessary to move research
forward.”
The new center will be the hub of an
international network of scientific talent and resources necessary
to propel FSHD research through greater coordination,
collaboration, financial resources, and access to patients.
FSHD is one of the more common forms of
muscular dystrophy. Most symptoms do not appear until the teen
years, but the disease does appear, albeit more rarely, in very
young children and infants. The condition, which is genetic in
origin, is characterized by a progressive weakness of muscles
starting in the face, shoulder blades, and upper arms. While the
disease is understood to be genetic in origin, the precise
mechanism is unknown.
The Department of Neurology is home to the
National Registry of Myotonic and FSHD Patients and Family Members.
The registry will enable Fields Center researchers to reach out to
a large number of patients and recruit them into research projects.
Because FSHD cannot yet be replicated and studied in animal models,
collection of blood DNA and muscle biopsy samples are essential for
the success of the research projects. These samples will provide
scientists with critical genetic clues as to the origin of the
disease and possibly new treatment approaches.
“This is a wonderful opportunity
to move forward a whole area of research and clinical care for an
underserved disease,” says neurologist Richard Moxley,
director of the Wellstone Center and the National Registry.
“The real key will be our ability to interact with a large
population of patients who will be able to guide us toward a more
clearly focused understanding of the disease.”
“The University of Rochester has long
been a leader in muscular dystrophy research, so they were a
natural fit to establish a center to focus on genetic and clinical
research to FSHD patients,” says Fields, chairman and founder
of Coastal Development. “I see this gift as a significant
step in giving this disease the medical attention it deserves, and
hope that the Fields Center will be a center for leadership and
innovation in the field for many years to come.”
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