December 18, 2007 

Renewed Funding for ALS Biomarker Discovery Highlights Importance of Reliable Disease Indicators

The ALS Association is pleased to announce approval of renewal funding for verification and further analysis of biomarkers in ALS. The studies supported by this grant will build on previous pioneering work to discover new ways to understand and track ALS disease progression.

“A key unmet need in ALS clinical trials is a biomarker of the disease and its response to treatment,” according to Lucie Bruijn, Ph.D., science director and vice president of The Association. A biomarker is an easy-to-measure indicator that reliably changes with the disease. Examples of biomarkers are the prostate-specific antigen for prostate cancer, or changes in blood pressure for assessing the risk of heart attack. So far in ALS, there are no convenient and accurate biomarkers that can be used in clinical trials of new treatments.

To meet this need, The Association has supported a consortium of researchers focused on biomarker discovery, including Metabolon, Inc., of Durham, NC; Robert Bowser, Ph.D., of University of Pittsburgh School of Medicine; and Merit Cudkowicz, M.D., of Massachusetts General Hospital. Additional funding was provided by the Jim “Catfish” Hunter ALS Foundation in Hertford, NC.

Pilot studies have indicated that useful biomarkers may exist in the blood or cerebrospinal fluid of people with ALS. The renewal funding will expand the number of participants studied, and rigorously determine the best sample collection procedures for these biomarkers.

“This expanded study will allow us to test whether these compounds are associated with ALS in most patients,” said Dr. Cudkowicz. Dr. Cudkowicz and colleague Swati Aggarwal, M.D., will lead the clinical investigation arm of the study. “Finding a validated biomarker for ALS could be a real step forward in conducting future clinical trials.” The samples taken in this study will also be made available to other scientists for ALS-related research.

Samples will be collected at 18 sites in the TREAT ALS/Northeast ALS (NEALS) Clinical Trials Network. The Network is a partnership between The Association and NEALS clinical research centers throughout the country, devoted to expanding clinical trials to treat the disease.

The NEALS Consortium comprises 70 research and treatment centers. TREAT ALS (Translational Research Advancing Therapy for ALS) is a drug discovery program and clinical trials process created by The ALS Association that accelerates discovery and testing of clinical candidates.

Centers participating in the biomarkers study include:

Site	Principal Investigator
Columbia University Medical Center	Hiroshi Mitsumoto, MD
Dartmouth Hitchcock Medical Center	Jeffrey Cohen, MD
Duke University Medical Center	Richard Bedlack, MD
Emory University	Jonathan Glass, MD
Johns Hopkins University	Nicholas Maragakis, MD
Lahey Clinic	James Russell, DO
Massachusetts General Hospital	Swati Aggarwal, MD
Baylor College of Medicine	Ericka Simpson, MD
Providence ALS Center	Kimberly Goslin, MD
Saint Louis University	Ghazala Hayat, MD
SUNY Upstate	Jeremy Shefner, MD
University of Arizona	Timothy Miller, MD
University of California, Irvine	Tahseen Mozaffar, MD
University of Chicago	Kourosh Rezania, MD
University of Miami	Khema Sharma, MD
University of Pittsburgh	David Lacomis, MD
University of Utah	Mark Bromberg, MD
Wake Forest University	James Caress, MD

“We are excited about the continued funding of this project” said John Ryals, Ph.D., President and CEO of Metabolon. “Using our metabolomics platform, our initial studies discovered novel biomarkers potentially useful for diagnosing ALS.  By expanding this study, we will be able to refine and validate those biomarkers, bringing us even closer to a clinical diagnostic.”

About ALS

Amyotrophic lateral sclerosis (ALS) is a motor neuron disease, first identified in 1869 by the noted French neurologist Jean-Martin Charcot. Although the cause of ALS is not completely understood, the past two decades have brought a wealth of new scientific understanding about the disease. ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and the spinal cord. Motor neurons reach from the brain to the spinal cord and from the spinal cord to the muscles throughout the body. The progressive degeneration of the motor neurons in ALS eventually leads to their death. When the motor neurons die, the ability of the brain to initiate and control muscle movement is lost, leading to progressive paralysis.

Lou Gehrig, with whom ALS is most commonly associated, first brought national and international attention to the disease in 1939 when he abruptly retired from baseball after being diagnosed with ALS.

About The ALS Association

The ALS Association is the only national, not-for-profit voluntary health organization devoted solely to fighting ALS through research, patient services, advocacy and public education and information.

The ALS Association’s TREAT ALS (Translational Research Advancing Therapy for ALS) program combines efficient new drug discovery with priorities set for existing drug candidates, to accelerate clinical testing of compounds with promise for the disease. For more information on TREAT ALS, please see The ALS Association's web site under the research tab (www.alsa.org/research/article.cfm?id=1048) and Laboratory Models in ALS (http://www.alsa.org/research/article.cfm?id=812).