ALS (Lou Gehrig's disease)

Amyotrophic 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 a patient's intellectual capacity but increased susceptibility to pneumonia and respiratory failure results in half of all patients dying 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: 

• Drug discovery.  Launched in 2008, the new Collaborative ALS Drug Discovery Initiative (CADDI) is designed to accelerate the development of new, effective treatments for ALS patients. In the spring of 2008 we issued a nationwide request for drug discovery proposals to thousands of scientists who specialize in research related to neurodegenerative disease. The goal was to initiate a robust pipeline of promising ALS drug discovery projects. To date, three projects are already well underway at our drug discovery laboratory with the intention for significant program expansion during 2009.

• Understanding the disease.  Harvard NeuroDiscovery Center member Robert H. Brown, MD, PhD, and others teamed up to identify genes that impact the risk of developing the disease as well as its progression. This work will be published shortly. 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 treatment.

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 here. 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.