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

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Collaboration Funds Historic $36 Million ALS Drug Search
Posted on : Tue, 16 Jan 2007 14:43:01 GMT | Author : Muscular Dystrophy Association
News Category : PressRelease



TUCSON, Ariz., Jan. 16 /PRNewswire-USNewswire/ -- The largest ALS drug discovery project in history was launched today as a joint venture by the Muscular Dystrophy Association (MDA), through its Augie's Quest initiative, and the ALS Therapy Development Institute.

The three-year, $36 million project will attempt to identify biochemical targets and find drugs that work on them in ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).


The collaboration will be funded by MDA's Augie's Quest, a fast-track ALS research program, and by the Institute (formerly the ALS Therapy Development Foundation). MDA, through Augie's Quest, 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. MDA's funding for this collaboration is above and beyond its existing ALS research expenditures.

"This project represents a declaration of war on ALS, and this is a war we intend to win by building the best scientific and technologic army in the world," said Augie Nieto, co-chairman with his wife, Lynne, of MDA's ALS Division. "Amazing things can happen when you combine the drive to succeed with money and technology."

Research will take place at the Institute's 16,000-square-foot lab in Cambridge, Mass. James Heywood, who started the organization in 1999 after his brother Stephen received an ALS diagnosis, said, "This collaboration allows for a massive application of cutting-edge technology, combined with proven drug development techniques. There's no doubt in my mind that ultimately we are going to be successful."

Stephen Heywood died in November when the ventilator that allowed him to breathe malfunctioned while he slept.

ALS is a progressive neurological disease that affects the nerves that send signals to the muscles. It causes paralysis of all voluntary muscles and ultimately death, usually within three to five years. The cause is unknown.

"The causes of ALS have been a tragic mystery for too long," said MDA National Chairman Jerry Lewis, noting that Lou Gehrig's widow, Eleanor, held a voluntary leadership position with MDA for many years. "This project is a giant step toward a solution to that mystery and a cure for ALS."

Augie Nieto has been named chairman of the board for the ALS Therapy Development Institute and Steve Perrin, formerly an executive at the biotechnology company Biogen Idec, will be its chief scientific officer. Former Institute vice president of drug discovery Sean Scott will serve as its president.

About Augie's Quest
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Waging Lou Gehrig’s Ultimate Fight

Two non-profits sign a $36 million collaboration deal to develop drugs for the disease that claimed the life of the famed New York baseball player.
January 16, 2007

By Rachel Barron



Legendary Yankee baseball player Lou Gehrig earned eternal fame for two things: First for his ability to play 2,130 consecutive games, and second, for what would eventually force him to leave the field forever. He had the disabling and fatal neuromuscular disease amyotrophic lateral sclerosis, or ALS for short. Today, folks diagnosed with what is also called Lou Gehrig’s disease have only one prospect when it comes to drug treatment.



But two non-profits have joined forces to boost that single number of ALS medications on the market. The Muscular Dystrophy Association and the ALS Therapy Development Institute said Tuesday they have entered into a $36 million research collaboration.



The deal calls for the association to dole out at least $6 million a year for the next three years, and for the institute give $6 million of its annual budget to the project.



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Funding will go to further research, like drug discovery, at the institute’s 16,000 square-foot facility in Cambridge.



More specifically, the institute will use the financing in part to go back to basics, like identifying physiological pathways of disease progression.



The institute has already focused heavily on evaluating previously discovered pathways, notably those in published research. The process also involved testing drugs approved to treat other diseases.



“To the extent that the existing body of literature leads to one drugabble target, we’ve exhausted it,” said Sean Scott, president for ALS Therapy Development Institute. So far, the institute has evaluated 800 drugs.



But that’s not to say there hasn’t been some success. Currently, the institute has three drugs in early stage trials. Among them includes Abbott Laboratories’ HIV drug Ritonavir, and Bristol-Myers Squibb’ leukemia drug Hydroxyurea.



As well, the institute spun off the ALS drug developing company ALSGEN in 2004. The company has so far raised $1.75 million from private venture philanthropists.



About 30,000 people in the United States and 300,000 internationally suffer form ALS. This progressive neurodegenerative disease leads to paralysis. The disorder that causes muscle weakness and atrophy eventually leaves patients trapped in their bodies unable to speak, eat, or breathe on their own. Symptoms commonly appear in middle to late adulthood, with death in two to five years.



Currently there is no known cure. So far, only one drug, Aventis Pharmaceuticals’ Riluzole, has been federally approved to treat the disease. But the therapeutic effects are modest, offering patients three more months on average to their life.



“When you are the relative of the patent and look around at who is in charge of curing your loved one. The answer is no one,” Mr. Scott says, which in part it why the collaboration has been spurred.



A big goal of the collaboration will be to see the institute license intellectual property and drug leads to biotech and pharma companies for further development.



As Mr. Scott sees it, the private sector hasn’t been as eager to come on board to develop ALS drugs due to the risk involved. “In a disease with no validated target, you’re up against a world of unknowns,” he said. But the day that changes, Mr. Scott anticipates big interest by the private sector. If a company can develop an effective ALS drug, he believed it will also tap a potential market worth $9.5 billion.