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ALS (Lou Gehrig's disease)

Amyloid Lateral Sclerosis (ALS or Lou Gehrig's disease) is a progressive, fatal disorder. ALS usually appears between the ages of 40 and 70, and affects more men than women. About 30,000 Americans currently suffer from ALS.  Approximately 5,600 Americans are newly diagnosed each year.

Initial signs of ALS include twitching, cramping, weakness in the legs and arms, and difficulty speaking, chewing, or swallowing. As symptoms spread throughout the body, weight loss, fatigue, exaggerated reflexes, and decreased coordination become common. Ultimately, patients cannot walk, stand, eat, or breathe without assistance. The disease does not impact the senses or memory. Increased susceptibility to pneumonia and respiratory failure causes half of all patients to die within three to five years of their diagnosis.

ALS strikes the cells in the brain and spinal cord (motor neurons), which send signals to move muscles. We do not know how damage to motor neurons occurs. In some cases, a gene causes a mutation in a protein (called SOD1) that normally "cleans" up toxic particles inside a cell. When SOD1 is mutated, toxic particles accumulate inside motor neurons causing them to malfunction. But this mutation only explains a few percent of cases of ALS. Other mechanisms must be involved.

There is no cure for ALS and there is currently only one approved medicine (Riluzole) to treat the disease. While Riluzole may slow the disease for a few months, it has no lasting effect. 

But progress toward understanding ALS is being made and we are investigating new treatment possibilities. Examples of the Harvard NeuroDiscovery Center's initiatives to fight ALS include: 

  • Understanding the disease.  Harvard NeuroDiscovery Center member Robert H. Brown, MD, PhD, and others have teamed up to identify genes that impact the risk of developing the disease as well as its progression. Other projects are probing the basic mechanism of ALS. Through a deeper understanding of why and how the disease strikes, we will identify clues for an effective cure.

  • Testing new treatments and cures.  Several drugs are undergoing clinical trials to determine if they can treat ALS safely and effectively. The Harvard NeuroDiscovery Center is supporting the first ever trial of ceftriaxone, a compound that has shown promising results in animal models. We also are supporting the development of a test that will allow physicians to monitor disease progression.

  • Drug discovery.  Through the new ALS Drug Discovery Research Program, the Harvard NeuroDiscovery Center has issued a nationwide request for ALS drug discovery projects from the ALS research community. 

The Havard NeuroDiscovery Center is focused on accelerating the discovery of effective treatments and cures for ALS. For information about our many important initiatives and how to support our approach, click on "Advance The Cause" at left.  For information about coping with the disease, the ALS Association website may be of interest: ALS Association. Please also visit Harvard NeuroDiscovery Center partner, Project ALS.