Prominent Researchers at Columbia University Receive Significant Funding to Combat Leading Genetic Killer of Infants and Toddlers
With an estimated 55,000 people afflicted with the disease in the United States, Europe and Japan, spinal muscular atrophy, once among the least understood diseases in medicine, has recently emerged as one of the genetic conditions closest to a treatment. Researchers at Columbia University have received more than $3 million in funding from the Spinal Muscular Atrophy Foundation to conduct research on spinal muscular atrophy (SMA), a neuromuscular disease that is the leading genetic killer of infants and toddlers. The funding will enable the institution to significantly expand its already substantial SMA research.
New York, NY (PRWEB) March 3, 2005 -- Researchers at Columbia University have
received more than $3 million in funding from the Spinal Muscular Atrophy
Foundation to conduct research on spinal muscular atrophy (SMA), a neuromuscular
disease that is the leading genetic killer of infants and toddlers. The funding
will enable the institution to significantly expand its already substantial SMA
research.
With an estimated 55,000 people afflicted with the disease in
the United States, Europe and Japan, spinal muscular atrophy, once among the
least understood diseases in medicine, has recently emerged as one of the
genetic conditions closest to a treatment. The National Institute of
Neurological Disorders and Stroke (NINDS) has selected SMA to serve as the
prototype for a translational research project that is expected to yield drug
candidates for investigational new drug application filings by 2007. The SMA
Foundation has been instrumental in raising awareness and supporting ground
breaking SMA projects in biotechnology, government and academia which will
advance translational research overall, including the efforts underway at NIH.
The SMA Foundation’s donation to Columbia University research is part of
a broader strategy to integrate basic, translational and clinical research
efforts at leading institutions into coordinated efforts aimed at facilitating
drug discovery.
Loren Eng, Co-Founder and President of the SMA
Foundation, said, “Columbia University was an obvious place to launch this
effort given the institution’s leadership position in neuroscience and
neurology. In the past four years Columbia neuroscientists have won two Nobel
prizes. There is also a strong commitment to neuroscience from Columbia’s
administration and research community. One third of all Columbia researchers
study the brain and nervous system and they generate more research funding than
any other group of neuroscientists in the country. Given the strengths of the
institution and its people, it was evident that Columbia was the place for us to
develop an SMA research nucleus.”
Most recently, Columbia University
president Lee Bollinger and Columbia University Medical Center (CUMC) executive
vice president and dean Gerald Fischbach, M.D. announced the creation of a
Neuroscience Institute which will bring together researchers and clinicians to
expedite discoveries from the laboratory to the bedside.
“This a
remarkable time in the field of neuroscience and SMA research,” said Dr.
Fischbach. “Given all that is already known about SMA scientifically, the
expanded research that this funding supports will greatly advance the
probability of a treatment in the near term. The funding provided by the
Foundation will enable us to provide critical support to rapidly advance our
research and clinical care to patients with SMA.”
Key Columbia faculty
members who have been funded by the SMA Foundation or are involved in SMA
research include:
Umrao Monani, Ph.D., Assistant Professor of Neurology
in the Center of Neurobiology and Behavior at CUMC is responsible for
identifying drug targets. He was instrumental in the development of a key mouse
model of SMA, which is essential to genetic studies of the disease. His research
addresses the question of why a decreased amount of Survival Motor Neuron (SMN)
protein leads to SMA and provides an opportunity to test the drugs that Dr.
Stockwell identifies.
Brent Stockwell, Ph.D., Assistant Professor in the
Department of Biological Sciences and the Department of Chemistry at Columbia
University leads drug discovery efforts. He focuses on chemical genetics, drug
discovery and is screening tens of thousands of existing compounds to identify
drugs that will stimulate the creation of SMN protein, which is deficient in SMA
patients.
Christopher E. Henderson, Ph.D., a neurobiologist specializing
in central nervous system development and neuronal degeneration will be joining
the institution. Dr. Henderson will establish a Center for Motor Neuron Biology
and Disease at CUMC focused on high-level translational motor neuron research.
Working collaboratively with each laboratory and contributing to the
translational research process, the new center will facilitate transfer of
screening techniques, active chemical molecules and knowledge of disease causing
pathways.
Darryl De Vivo, M.D., the Sidney Carter Professor of Neurology
at CUMC and the director of the SMA Clinic, will direct the next step toward
bringing new therapies to SMA patients through clinical trials. Dr. De Vivo is
leading a multi-center network of investigators – the Pediatric Neuromuscular
Clinical Research Network – that includes CUMC, Harvard University and the
University of Pennsylvania researchers, as well as data management from the
University of Rochester. Beginning in 2005, Dr. De Vivo and the network will
conduct clinical trials in SMA patients with drugs that are currently under
study as well as any compounds newly discovered by Dr. Stockwell, Dr. Monani and
others. It is hoped that one more of these drugs candidates will significantly
reduce the devastating effects of SMA in patients.
James L. Manley,
Ph.D., a molecular biologist and Julian Clarence Levi Professor of Life Sciences
in the Department of Biological Sciences at Columbia University is studying,
together with his colleague Tsuyoshi Kashima, M.D., the basic biology of the SMN
gene to find ways in which to recover its activity in patients. He is interested
in how information in the chromosome is converted into messenger RNA to produce
the SMN protein and is trying to understand the defect in that process that
results in SMA disease.
Thomas Jessell, Ph.D., Investigator at the Howard
Hughes Medical Institute and Director of the Neuroscience Institute at Columbia
University, is focused on defining the molecular mechanisms that control motor
neuron differentiation and the formation of sensory-motor circuitry in the
developing spinal cord. Recent studies together with Hynek Wichterle, Ph.D., now
in the Department of Pathology at Columbia University Medical Center, have been
able to convert embryonic stem cells into functional motor neurons, opening up
new strategies for cell based chemical screens for drugs that can prevent the
degeneration of motor neurons associated with SMA.
Committed to
supporting research with the highest probability of advancing potential drug
candidates, the SMA Foundation has forged collaborations with leading medical
institutions and industry in the United States and internationally. Since its
inception, the Foundation has funded more than $15 million in research
initiatives, formed a number of partnerships with biotechnology companies,
reduced barriers in the drug development process by providing open access to
mouse models of SMA and has been instrumental in working with both Congress and
the National Institutes of Health in creating awareness and increasing federal
funding for the disease.
About SMA Foundation
The SMA Foundation is a
nonprofit organization founded in 2003 dedicated to finding a treatment or
potential cure for spinal muscular atrophy (SMA). The Foundation provides
funding for the full range of research from basic to clinical work conducted in
academic laboratories as well as corporate therapeutics development. In
addition, the Foundation is committed to raising awareness through education and
increasing federal funding and support for the disease. For more information on
the Spinal Muscular Atrophy Foundation, visit www.smafoundation.org or
call (646) 253-7100.
About Columbia University
Columbia University in
the City of New York, founded in 1754 as King’s College by royal charter of King
George II of England, is the oldest institution of higher learning in the state
of New York and the fifth oldest in the United States.
Columbia
University Medical Center provides international leadership in basic,
pre-clinical and clinical research, medical education, and health care. The
medical center trains future leaders in health care and includes the dedicated
work of many physicians, scientists, nurses, dentists, and other health
professionals at the College of Physicians & Surgeons, the School of Dental
& Oral Surgery, the School of Nursing, the Mailman School of Public Health,
the biomedical departments of the Graduate School of Arts and Sciences, and
allied research centers and institutions. With a strong history of some of the
most important advances and discoveries in health care, its researchers are
leading the development of novel therapies and advances to address a wide range
of health conditions.
Media Contact:
Bryan deCastro, (631)
495-9177
SMA Foundation
e-mail protected from spam bots
# # #
Source : http://www.prweb.com/releases/2005/3/prweb214571.htm