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ALS TDI Completes Gene Expression Profile of Lou Gehrig's Disease in Mice

Largest Target-Discovery Program Aimed at New Treatments for Fatal Disease

CAMBRIDGE, Mass., Oct. 2 /PRNewswire/ -- The ALS Therapy Development Institute (ALS TDI) today announced that it has completed the first phase of a comprehensive gene-expression database for ALS with the profiling of the SOD1 mouse model of amyotrophic lateral sclerosis -- ALS, or Lou Gehrig's disease. The second phase, profiling of human samples, is scheduled for completion by February of 2008. This study is part of the largest known target-discovery program aimed at developing new treatments for the fatal neuromuscular disease.

"This dataset is the foundation that will enable scientists at ALS TDI to understand the biological mechanisms associated with disease progression," said Steve Perrin, Ph.D., chief scientific officer at ALS TDI. "Additional gene expression profiling and proteomics analysis from other mouse models of neurodegeneration, as well as human samples collected at ALS clinics, will help us focus on the most relevant biological pathways to target for drug development."

The gene-profiling study involved looking at which genes are turned on over a period of time in various tissues of the SOD1 mouse. Comprising more than 250 samples from four different tissues, the comprehensive database reflects gene expression from eight time points during disease onset and progression. Scientists at Gene Logic Inc., of Gaithersburg, Md., isolated total RNA from the tissue samples, generated high quality genomic data using the Affymetrix GeneChip(R), and used their expert bioanalytical capabilities to identify the activated genes implicated in ALS.

"The assembly of this data set is an important milestone. We could not have gotten to this point without the critical support of Congressman Capuano, Senator Kennedy, the Department of Defense, the MDA and the nearly 250 families that have invested in our unique approach," said Sean Scott, president of ALS TDI, "However, there are several more hurdles to overcome and milestones to be reached."

Unprecedented in scale, the gene-expression profiling project is a cornerstone of the ALS TDI research plan. The collaboration was made possible in part by funding received earlier this year as part of a historic three- year, $36 million partnership with the Muscular Dystrophy Association (MDA) through its fast-track ALS research initiative, Augie's Quest.

"This is the first step in an extensive plan to map fully all of the gene and protein changes that lead to the disease," said Sharon Hesterlee, Ph.D., vice president of translational research at the Muscular Dystrophy Association. "As the data are analyzed and the results published, we think this project will greatly benefit the field."

Ultimately, the Institute plans to make this gene-profiling dataset many times larger, and to incorporate information from other neurodegenerative animal models. It has already initiated relationships with several ALS clinics to conduct similar experiments on samples donated by ALS patients.

About ALS Therapy Development Institute

The ALS Therapy Development Institute (, based in Cambridge, Mass., was founded in 1999 as the ALS Therapy Development Foundation. Re-named in 2007 as a result of its collaboration with MDA's Augie's Quest, the Institute continues its mission to discover and develop drugs to treat ALS. It excels in identifying novel disease targets, discovering compounds that may act against these targets, and screening these potential treatments toward bringing viable therapeutics to the clinic.

About Augie's Quest

Fitness pioneer Augie Nieto started Augie's Quest ( in conjunction with MDA's ALS Division. Nieto is co-founder and former president of Life Fitness of Chicago, 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.

About MDA

MDA ( is the world's largest provider of ALS services and funder of ALS research. Over the years, it has expended almost $200 million in this effort. It operates 225 neuromuscular disease clinics across the country and 37 ALS-specific research and care centers.

SOURCE ALS Therapy Development Institute
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