Amyotrophic lateral sclerosis (ALS; Lou Gehrig’s disease) is a neurodegenerative disease that attacks nerve cells (neurons) that are responsible or controlling voluntary muscles. According the National Institute of Neurological Disorders and Stroke (NINDS), approximately 20,000 Americans have ALS, and an estimated 5,000 additional people are diagnosed with ALS each year. Unfortunately, ALS is a rapidly progressive disease that is fatal.

In patients suffering with ALS, the upper and lower motor neurons degenerate and/or die, resulting in a loss of muscle function. Eventually, the muscles that are responsible for respiration fail, and the ability to breath is lost. The cause of ALS remains unknown, and in approximately 90-95% of all ALS cases, the disease appears to occur randomly. However, some recent theories hypothesize that there is interplay between genetic or metabolic factors and lifestyle.

To date, there is one drug that has been approved by the Food and Drug Administration (FDA). This drug, Riluzole, is thought to reduce the damage to motor neurons and thus extend the life of these neurons and, especially, in patients that present with difficulty swallowing. Riluzole acts as an anti-glutamatergic agent, which inhibits the release of the neurotransmitter glutamate.

A recent article published in the Journal Neuropsychiatric Disease and Treatment reviewed the current literature in order to assess emerging treatments for ALS. Studies from 1986 to August 31st, 2009 were included in this evaluation; of these studies, 48 drugs were identified and studied. Although several of the drugs in these studies showed effective results in animal models, none of the compounds, excluding Riluzole, significantly prolonged the survival rate, or improved the quality of life, in patients suffering with ALS. The authors of this review proposed that more potent Riluzole analogs should be explored as well as “cocktail therapies” that include Riluzole and other drugs.