Advances in the Treatment of ALS/Lou Gehrig`s Disease
ALS/Lou Gehrig's Disease
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a progressive disease that affects motor nerve cells in the brain and spinal cord. Motor neurons degenerate and eventually die, causing the brain to lose its ability to control muscle movement. The progressive loss of muscle strength and coordination eventually interfere with the ability to perform routine activities, such as going up steps, swallowing, or rolling over. Eventually it leads to death, often because it affects the muscles needed for breathing. ALS, however, has no effect on a person's ability to think or reason.124 It is usually fatal within five years of diagnosis.125
Symptoms usually do not develop until after age 50. In about 10 percent of cases, ALS is caused by a genetic defect. In other cases, the cause of the nerve deterioration is unknown.126 ALS affects approximately 30,000 people in the United States, and about 5,000 new cases are diagnosed each year.127
Patient Perspective: A Pitch for ALS
PHARMACEUTICAL ADVANCES
Breakthrough Treatment a First After 126 Years
ALS was first identified in 1869, but the first drug, riluzole, was not approved for treatment until 1995. It is the first drug to show any increase in survival for ALS patients.128 The increase is a modest three months on average, but it offers hope.129 The drug is based on a theory that the disease is caused by toxic levels of glutamate in the brain. Neurons in the central nervous system use glutamate to communicate with one another. The brain normally regulates glutamate levels, but glutamate has been found at abnormally high levels in the cerebrospinal fluid of some ALS patients. Scientists theorize that glutamate toxicity might be killing motor neurons, leading to progressive muscle degeneration in people with ALS. Riluzole, however, inhibits the release of glutamate in the brain.
According to Dr. Jeffrey Rothstein of Johns Hopkins University School of Medicine, this medicine offers great hope despite the fact that the improvement is not dramatic: "This is against the background of decades when no drug ever did anything for the disease. Initial therapies for many diseases, like leukemia and other cancers, had the same kind of effect... a modest increase in survival. But they were followed by better therapies that, over time, increased patients' survival... It's a daunting task, but I envision that someday it will be possible to develop drugs that will not only stop motor neurons from dying but replace them and reverse the course of ALS."130
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An "orphan" or rare disease affects fewer than 200,000 people in the
United States.131 |
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Endnotes:
124 ALS Association, What Is ALS?, http://www.alsa.org/als/what.cfm?CFID=1107473&CFTOKEN=16335905 (accessed 12 September 2005).
125 National Organization for Rare Disorders, "Amyotrophic Lateral Sclerosis," Rare Disease Database, http://www.rarediseases.org/search/rdblist.html (accessed 2 June 2005).
126 National Library of Medicine, MedlinePlus Medical Encyclopedia, Amyotrophic Lateral Sclerosis, http://www.nlm.nih.gov/medlineplus/ency/article/000688.htm (accessed 12 September 2005).
127 National Organization for Rare Disorders, "Amyotrophic Lateral Sclerosis," op. cit.
128 Food and Drug Administration, "FDA Approves First Drug for Lou Gehrig's Disease," press release, 12 December 1995, http://www.fda.gov/bbs/topics/NEWS/NEW00522.html (accessed 26 September 2005).
129 Food and Drug Administration, "Glimmer of Hope for People with ALS," FDA Consumer Magazine (September 1996), http://www.fda.gov/fdac/features/796_als.html (accessed 12 September 2005).
130 Ibid.
131 National Organization for Rare Disorders, http://www.rarediseases.org/info/about.html.
