- Posted Monday March 20, 2006
TGen to Scan 1,000 Patient Samples in Year-long Study
TUCSON, Ariz., March 20, 2006 -- The largest screening of genes
that may be involved in causing, exacerbating or increasing
vulnerability to amyotrophic lateral sclerosis (ALS, or Lou
Gehrig's disease) has been funded by the Muscular Dystrophy
Association (MDA), the organization announced today.
MDA will provide $650,000 to the non-profit Translational Genomics Research Institute (TGen) in Phoenix, to scan the genomes of 1,000 patient samples collected from several clinical sites around the country to identify ALS-causing genes.
The one-year project is focused on identifying regions in the human genome that harbor genes that predispose individuals to sporadic ALS. Much insight has been gained into other forms of ALS, but there is still little understanding of the common causes of ALS, which occur with no prior history.
"We are optimistic that this study will enable us to identify those genes that contribute to sporadic ALS. That knowledge will bring us a step closer to developing a diagnostic test and hopefully improved therapeutics for battling ALS," said Dr. Dietrich Stephan, head of TGen's Neurogenomics Division and project director.
ALS is a progressive, degenerative disease of the nervous system that usually begins in middle or late adulthood, causes total paralysis, and most often leads to death within five years. It's called Lou Gehrig's disease after the famed New York Yankees baseball player who died of the disease in 1941. Since 1993, scientists, many of whom have received funding from MDA, have uncovered a few genes that, when flawed, can directly cause ALS, and have suspected several others of increasing vulnerability to the disorder or accelerating its course.
Projects funded by MDA that are smaller in scope have provided researchers with several hunches concerning biochemical pathways that are likely to contribute to ALS, but this is the first project that will scan the entire human genome (full set of human genes) for variants that may be more common in the ALS population than in the general population.
"Identifying such variants is likely to provide leads to additional biological pathways involved in the disease and provide targets for drug development," said Sharon Hesterlee, MDA Director of Research Development. "This is a very powerful approach that should yield results."
MDA's financial support was made available through Augie's Quest, an aggressive campaign aimed at raising funds for ALS research that will lead to treatments and a cure for the disease that affects more than 30,000 Americans.
"TGen is uniquely suited to leverage the information in the human genome to accelerate the development of novel diagnostics and smarter treatments for patients with ALS," said TGen's president and scientific director, Dr. Jeffrey Trent. "We are pleased to be partnering with MDA to identify susceptibility genes for this devastating disease."
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MDA (www.mdausa.org), founded in 1950, is a voluntary health agency working to defeat more than 40 neuromuscular diseases through programs of worldwide research, comprehensive services, and far-reaching professional and public health education.
About Augie's Quest
Fitness pioneer Augie Nieto started Augie's Quest (www.augiesquest.org) in conjunction with MDA's ALS Division. Nieto is co-founder and former president of Life Fitness, and chairman of Octane Fitness. He and his wife, Lynne, serve as co-chairpersons of MDA's ALS Division. Nieto received a diagnosis of ALS in March 2005.
The Translational Genomics Research Institute (TGen) is a non-profit 501(c)(3) organization focused on developing earlier diagnostics and smarter treatments. Translational genomics research is a relatively new field employing innovative advances arising from the Human Genome Project and applying them to the development of diagnostics, prognostics and therapies for cancer, neurological disorders, diabetes and other complex diseases. TGen's research is based on personalized medicine. The institute plans to accomplish its goals through robust and disease-focused research.
Director of Public Information
Muscular Dystrophy Association
Translational Genomics Research Institute