In 1869, French neurologist Jean-Martin Charcot discovered several conditions affecting the lateral horn of the spinal cord. These conditions became known as Maladie de Charcot (Charcot's disease). Further investigations isolated a disease resulting in the death of motor neurons and in England this illness was referred to as motor neuron disease (MND). In the US it was named Amyotrophic Lateral Sclerosis (ALS), more commonly known as Lou Gehrig's disease after it killed the 37 year-old Hall of Fame baseball legend in 1941.
ALS is a progressive fatal neurodegenerative illness that attacks motor neurons. When motor neurons die, the ability of the brain to control muscle movement is lost, leading to paralysis. Unable to function, muscles atrophy. Eventually, all muscles under voluntary control are affected and patients in time lose their ability to walk, talk, swallow, and breathe. During this horrible process, the mind remains completely intact observing the loss of each function. When the diaphragm and chest muscles fail, patients stop breathing on their own. The majority of people with ALS die of respiratory failure within 3 to 5 years of noticing the symptoms.
The incidence of ALS is approximately 2 out of every 100,000 people. In the developed world, where disease statistics are tracked, it is believed that over 128,000 people are living with ALS. Applying the incidence to the 2005 world population, there could be more than 640,000 people who have the disease. Therefore, each year over 128,000 people contract ALS and the same number of people die from it.
While the disease is not contagious, people of all races and ethnic backgrounds are susceptible. ALS typically strikes people between the ages of 40 and 60, but it is known to affect younger and older people as well. The disease also occurs more often in men than women. In 2004, the Harvard School of Public Health conducted a study that showed US military veterans are 60% more likely to contract ALS than non-veterans.
The root cause of the disease is unknown. Only 5 - 10% of the cases can be linked to a family history of ALS. In the early 1990's, a team of researchers was able to identify a defective gene, SOD1, on chromosome 21 as the cause of many of the familial cases. This also enabled the development of animal models of the disease for testing potential treatments.
According to the US National Institute of Health (NIH), ALS is an orphan disease because it affects less than 200,000 people in the US. This gives the disease special status in accordance with the Orphan Drug Act of 1983. Because treatment development for orphan diseases are financially disadvantageous relative to other diseases, the government will reward companies that develop drugs for orphan diseases with tax reductions and a guaranteed patent monopoly for up to seven years. Drugs have been developed under this act for conditions like cystic fibrosis and HIV/AIDS.
There is only one drug currently on the market that treats ALS. French pharmaceutical company Sanofi-Aventis created riluzole, which carries the commercial name Rilutek and is aimed at symptomatic ALS relief. However, studies have shown that Rilutek does not improve patient quality of life and may only extend life an average of 2 months. Rilutek costs patients over $10,000 per year to maintain the daily recommended dosage.
Current ALS research is conducted primarily in academic research centers and is funded through government grants and nonprofit donations. Tens of millions of dollars are spent on ALS research fellowships and grants each year. All of these efforts have resulted in numerous scientific findings and publications about the disease, but no new effective treatments have made it from the laboratory to the patient. Prize4Life will change this...