Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), also known as "Lou Gehrig's Disease" after the famous baseball player who developed the illness while at the peak of his career, is an often-fatal neurodegenerative disorder characterized by the progressive degeneration of nerve cells in the central nervous system that controls voluntary muscle movement.
The cause of ALS is largely unknown and there is no cure or proven therapy for the disease. People afflicted with ALS usually die within five years of diagnosis. While ALS is generally not an inherited disease, five to 10 percent of cases are known as familial (inherited) ALS.
It is the third most prevalent neuromuscular diseases worldwide, even though only one in 50,000 individuals are diagnosed each year. Affecting more men than women, ALS is most commonly diagnosed in middle age. However, younger people can also develop a "juvenile" form of the illness. Juvenile ALS often progresses more slowly than the adult form of the disease.
CMMT is at the forefront of ALS research. Recently, we led an international team of researchers, with Dr. Joh-E Ikeda, Scientific Director of NeuroGenes International Cooperative Project, Tokai University, Japan, and the University of Ottawa, which discovered a gene that causes Juvenile ALS. We found this gene by analyzing the genomes of two families—one in Kuwait and one in Tunisia—whose members suffer from the juvenile form of familial ALS.
We remain committed to uncovering the genetic and molecular mechanisms of ALS and Juvenile ALS. In close collaboration with geneticists and clinicians from around the world, we are currently conducting a series of DNA screening studies involving hundreds of individuals, including ALS sufferers, to determine which other genes can trigger the disease.
We are also seeking to develop new treatments for ALS by generating and studying mice that have the same symptoms and genetic abnormalities as the human form of the disease. A wide range of therapeutic strategies can be tested in these mice in order to find treatments that will be effective in humans.
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