TDF IS NOW TDI
COLLABORATION FUNDS HISTORIC $36M ALS DRUG SEARCH
Partnership Combines Vision of Hope and Science to Become
World’s Largest Drug Discovery Program for ALS

CAMBRIDGE, Mass. and TUCSON, Ariz., January 16, 2007 — The Muscular Dystrophy Association (MDA), through its Augie’s Quest initiative, and the ALS Therapy Development Institute today announced they are establishing the world’s largest research program dedicated to discovering drugs to treat amyotrophic lateral sclerosis — ALS, or Lou Gehrig’s disease.
Under the agreement, the collaboration will be funded by MDA’s Augie’s Quest and by the Institute (formerly the ALS Therapy Development Foundation). MDA will grant at least $6 million a year for the next three years; the Institute will add its $6 million annual budget to the project. Additionally, MDA will provide access to patients through its nationwide network of 225 clinics. MDA’s funding for this collab¬oration is above and beyond its existing ALS research expenditures. The research will take place at the Institute’s 16,000 square-foot facility in Cambridge.
As a result of the collaboration, the ALS Therapy Development Institute elected Augie Nieto chairman of its board of directors. Nieto also serves as co-chairman of MDA’s ALS Division and is an MDA national vice president. The program also will add a drug discovery team and appoint former Biogen Idec executive, Steve Perrin, Ph.D., as chief scientific officer of the Institute.
A major goal of the ALS Therapy Development Institute will be to license its intellectual property on relevant pathway and drug leads to biotechnology and pharmaceutical companies for further develop¬ment and commercialization. “Our collaboration with MDA’s Augie’s Quest is an unprecedented initiative of speed, efficiency and focus on drug discovery,” said Sean A. Scott, president of the Institute, who, with Nieto and Sharon Hesterlee, Ph.D., MDA’s vice president of translational research, spear¬headed the collaboration. “With the expansion of our capacity for target validation and the addition of state-of-the-art drug discovery under Dr. Perrin, we’re now able to apply the necessary resources to take the disease apart at the molecular level and target appropriate therapeutics.”
The ALS Therapy Development Institute combines the passion and dedication of a nonprofit organi¬zation with the entrepreneurial and scientific spirit of a biotechnology company. “This initiative represents a natural fit between two organizations with complementary strengths, a common vision and a relentless entrepreneurial spirit,” explained Nieto. “This shows what can be done when you combine smart money with smart science to focus on an unmet medical need and a potential business opportunity.”
MDA’s Dr. Hesterlee added, “ALS has been a major part of our program since Eleanor Gehrig first sought MDA’s help in stopping the disease that claimed her husband. We look forward to seeing that promise fulfilled.”
Dr. Perrin brings to the ALS Therapy Development Institute more than 20 years of experience in genomics and proteomics applied to translational medicine — the “translation” of basic research into medical treatments. Prior to joining the Institute, he spent nearly seven years at Cambridge-based Biogen Idec, Inc., most recently as associate director, responsible for target discovery and biomarker characteri¬zation, using transcription profiling and proteomics for the company’s research and development. Dr. Perrin also was project team leader of Biogen Idec’s ALS initiative.
Uncovering the Cause of ALS
Through the MDA collaboration, the Institute’s expanded research and drug discovery program will integrate both animal and human research based on cutting-edge bioinformatics, and genomic and proteomic technologies. Applying its expertise in the newest mass profiling and screening approaches to ALS pathology, the Institute’s scientific objectives will be to:

Identify physiological pathways and molecules in animal models of disease progression
Generate therapeutic hypotheses around these pathways, and modulate the function of key molecules using novel drug delivery strategies to target the central nervous system
Create a comprehensive translational medicine initiative to identify ALS biomarkers for disease staging and prognosis, as well as markers of drug efficacy and patient response

About ALS Therapy Development Institute

The ALS Therapy Development Institute (www.als.net), based in Cambridge, Mass., was founded in 1999 as the ALS Therapy Development Foundation. Re-named in 2006 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. A leader in ALS transgenic mouse studies, it excels in identifying novel disease targets, and screen¬ing potential treatments for ALS, as well as related disorders like Alzheimer’s, Parkinson’s and Hunting¬ton diseases.

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 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 (www.mda.org) 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.

Media Contacts

Charles Versaggi, Ph.D.
Versaggi Biocommunications®
415/806-6039
cv@versaggibio.com

Sharon Hesterlee, Ph.D.
Muscular Dystrophy Association
520/529-5433
shesterlee@mdausa.org