ALS Therapy Development Institute Partners With Clinical Centers To Establish Pathological Mechanisms Of Lou Gehrig's Disease

Main Category: Neurology / Neuroscience
Also Included In: Clinical Trials / Drug Trials
Article Date: 20 Aug 2008 - 2:00 PDT

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ALS Therapy Development Institute (ALS TDI) announced the acceleration of an ALS biomarker effort using blood samples collected through an expanded network of clinical partnerships. These ongoing projects are part of the Institute's extensive effort to identify the altered expression of genes and proteins, at the onset of, and throughout the progression of the fatal neurodegenerative disease known as amyotrophic lateral sclerosis (ALS, commonly known as Lou Gehrig's disease). The knowledge generated through this effort will be crucial to the identification of potential biomarkers which could be used for diagnosis and monitoring of disease progression, and will be useful for researchers worldwide in the effort to develop truly effective therapeutics. To facilitate additional ALS patient participation, ALS TDI has recently executed or submitted protocols to several additional hospitals and clinics for the collection of additional blood samples, as well as muscle and adipose tissue. The program is designed to include all ALS patients, regardless of whether they have a sporadic or inherited form of the disease.

"These types of ongoing projects, made possible only through collaborations with our clinical partners, are critical to determining the molecular signatures of this extremely complicated disease. The efforts of our clinical partners to carefully collect these biological samples, as well as the relevant clinical information from each patient, is central for the success of this project," said Steve Perrin, Ph.D., chief scientific officer of ALS TDI.

ALS is a neurodegenerative disease resulting in progressive paralysis and is considered fatal. The disease typically strikes without an identifiable cause, indiscriminately affecting a new family every 90 minutes in the U.S. That incidence rate is similar to that of multiple sclerosis, but the typical survival prognosis given to an ALS new patient is only 2-5 years from the date of diagnosis. Currently there is no known cure for ALS and only one FDA-approved drug, with marginal efficacy, for treating the disease.

The data analysis from this developing translational database has already begun and will be compared with the ALS TDI transcriptome database for the SOD1 mouse collected last year. To date, the Institute has cataloged and analyzed the world's largest collection of information on the molecular changes in the leading mouse model in the effort to establish the fundamental pathological mechanisms of this devastating disease. These ongoing projects are made possible as part of a major, three-year, $18 million funding and scientific collaboration with the Muscular Dystrophy Association (MDA) and its Augie's Quest Initiative, entered into at the beginning of 2007.

"We're pleased to be part of this project that gives our patients an opportunity to participate in very significant research. By collaborating with ALS TDI, a broad spectrum of people living with ALS are now involved in the creation of therapeutics," said Stanley Appel, M.D., co-founder & co-director of the Methodist Neurological Institute in Houston, Texas.

At the time of this announcement, ALS TDI had three clinical locations set up to collect blood donations and clinical information from ALS patients; Methodist Neurological Institute, University of California - Irvine, and Mount Sinai Medical Center. Mount Sinai has approved both blood and muscle tissue collection protocols. All three of these clinics are MDA/ALS Centers. ALS TDI plans to continue to accelerate its biomarker program and has submitted several additional protocols for review and approval to hospitals and clinics in other parts of the U.S.

About the ALS Therapy Development Institute

The ALS Therapy Development Institute (ALS TDI) (http://www.als.net), based in Cambridge, Mass., operates the world's largest research and development program focused exclusively on ALS. At ALS TDI, a staff of over 30 scientists and research technicians work on behalf of ALS patients to discover and advance novel therapeutics for treating, and ultimately curing, ALS. The nonprofit biotechnology institute excels in identifying novel disease targets, discovering compounds that may act against these targets, and screening potential treatments for clinical development.

About Augie's Quest

Fitness pioneer Augie Nieto started Augie's Quest (http://www.augiesquest.org) 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 (http://www.mda.org) is the world's largest provider of ALS services and funder of ALS research. Over the years, it has expended $230 million in this effort. It operated 225 clinics across the county, 38 of which are ALS-specific research and care centers.

ALS Therapy Development Institute
http://www.als.net