MGH announces new ALS research venture
$1.7 million donation from CVS/pharmacy will support research collaborative

BOSTON - October 31, 2003 - The MassGeneral Institute for Neurodegenerative Disease today announced a new research initiative against ALS (Lou Gehrig's disease). Using some of the latest scientific approaches, the program is designed to clarify how the disease develops and to develop potential new treatments. It is made possible through the donation of $1.7 million raised by CVS/pharmacy.

"More exciting new strategies against ALS are evolving now than at any time in the past, and we are cautiously optimistic that there will be a major change in the outlook for this disease in the near future," says Robert H. Brown Jr., MD, PhD, director of the Cecil B. Day Laboratory for Neuromuscular Research at MGH, who will lead the research program.

The new initiative will bring together an international consortium of ALS researchers to take advantage of new technologies and information provided by the Human Genome Project. A major focus will be searching for normal genetic variants that, in combination, may lead to increased risk for developing sporadic ALS.

"The same sorts of investigations are underway for diabetes, asthma and hypertension," says Brown. "We hope that identifying such gene variants may lead us to the molecular pathways causing cell death, and that those findings will provide insight into potential ways of blocking those pathways."

That sort of research approach involves large-scale screenings of thousands of patient samples and a similar number of potential treatments, a task that could be daunting for a single research center. Therefore the MGH-led initiative has developed collaborations with many research groups, starting with the members of the ALS Therapy Alliance - which also includes investigators from Harvard College, Harvard Medical School, Boston University, Massachusetts Institute of Technology, Beth Israel Deaconess Medical Center, Brigham & Women's Hospital and the University of Massachusetts.

Amyotrophic lateral sclerosis is characterized by degeneration of the motor neurons of the central nervous system. Without affecting intelligence or memory, the disease leads to progressive paralysis of the limbs, the diaphragm, and the muscles needed for eating and speaking, usually leading to death within five years. Only 10 percent of ALS is inherited; 90 percent of cases occur in patients with no other affected family members. Like Alzheimer's, Huntington's and Parkinson's diseases, ALS is the result of the abnormal death of cells in the brain or spinal cord.

Brown's MGH lab has made many key advances in ALS research, including the 1993 discovery of the first gene responsible for the inherited form of the disease. His team subsequently developed both animal and cellular models of ALS, which could be essential to developing potential treatments, and helped identify gene defects causing a juvenile form of inherited ALS and a slowly progressive adult form.

The CVS donation includes funds collected from the company's employees and customers to fight this devastating neurologic disorder. ALS research was chosen as a philanthropic target because it has directly affected company employees and family members.

"As a health care company, this donation is part of our ongoing effort at CVS to help improve the health and well-being of the communities where we live and work," said Jack Kramer, senior vice president for CVS/pharmacy.

"Today we are poised to find new treatments and cures for devastating diseases like ALS, but we can't do it alone," says Anne B. Young, MD, PhD, MGH chief of Neurology. "The support of friends and organizations like CVS can really make the difference by providing the flexible funding that allows investigators to move decisively towards the newest line of research."

Massachusetts General Hospital, established in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH conducts the largest hospital-based research program in the United States, with an annual research budget of more than $350 million and major research centers in AIDS, cardiovascular research, cancer, cutaneous biology, medical imaging, neurodegenerative disorders, transplantation biology and photomedicine. In 1994, the MGH joined with Brigham and Women's Hospital to form Partners HealthCare System, an integrated health care delivery system comprising the two academic medical centers, specialty and community hospitals, a network of physician groups and nonacute and home health services.

Media Contact: Sue McGreevey, MGH Public Affairs

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