ALS (Lou Gehrig’s Disease)
Amyotrophic Lateral Sclerosis (ALS) is a devastating disease that progressively destroys nerve cells, called motor neurons, in the brain and the spinal cord, eventually causing paralysis and death. Baseball great Lou Gehrig first brought national attention to the disease in 1939 when he retired from baseball after being diagnosed with ALS. He died two years later, but ALS is still commonly referred to as “Lou Gehrig’s Disease.”
People who have ALS steadily lose their ability to control muscle movement. Patients in the later stages become totally paralyzed, although their minds are often unaffected. The average life expectancy of a person with ALS is two to five years from time of diagnosis. Many ALS sufferers die within a few years due to failure of the nerve cells that control breathing.
The cause of ALS is unknown and there is currently no cure. One FDA-approved drug, Rilutek, helps slow the progression of ALS, but no existing treatment halts or reverses the disease.
Human and Social Costs
- An estimated 30,000 Americans suffer from ALS.
- Every day, an average of 15 more people are diagnosed with ALS – more than 5,600 people per year.
- Aggregate direct cost to the healthcare system is estimated to total more than $6 billion per year.
The financial cost to families of persons with ALS is extremely high. In advanced stages of the disease, caring for an ALS patient can cost up to $200,000 a year, imposing an enormous financial and emotional toll on affected families.
Total economic impact is difficult to measure because — in addition to the cost of medicine, devices, and hospital visits — most of the care involved in ALS is provided at home by family members. Many individuals quit their jobs in order to stay at home with the patient at later stages of the disease or must pay for hospice care.
Potential for Cures
Scientists are exploring many new directions in ALS research. Studies indicate that stem cells can generate healthy new nerve cells. Thus, they could someday be transplanted into a patient’s body to treat or cure diseases like ALS, by generating healthy cells to replace diseased and damaged cells.
Another strategy involves administering a drug that may perhaps encourage the recipients’ own stem cells to divide and ultimately replace lost or damaged neural cells. This technique does not involve the transplantation of embryonic or adult donor stem cells.
Stem cell research is strongly supported by the overwhelming majority of medical researchers, medical organizations like the American Medical Association, and disease and patient advocacy groups like the ALS Association, ALS Therapy Development Foundation, Project ALS and Jack Orchard ALS Foundation.
Neurons made from human embryonic stem cells can both send and receive nerve impulses when transplanted into the mouse brain, scientists at University of Wisconsin-Madison have announced. The discovery provides some of the strongest evidence that human embryonic stem cell-derived neurons, which could be used to treat a variety of neurological disorders such as ALS, Parkinson’s disease, epilepsy and stroke, can fully integrate and behave like regular neurons when transplanted into the brain. (The Scientist, Nov. 21, 2011)
Using both embryonic and adult stem cells, researchers at the University of Wisconsin-Madison have created astrocytes, star-shaped cells in the brain that act like bodyguards for neuron brain cells and play an important role in diseases of the central nervous system. Astrocytes are more common than neurons but have been hard to grow in the lab. Being able to study them could help researchers understand their role in normal brain functioning, and help find new treatments for diseases such as ALS, in which the neurons are overworked. Transplanting healthy astrocytes could rescue the injured neurons. (LiveScience/FOX News, May 23, 2011)
Scientists from the Universities of Edinburgh, Cambridge and Cardiff in the United Kingdom have created a range of motor neurons – nerves cells that send messages from the brain and spine to other parts of the body – from human embryonic stem cells in the laboratory. The process, which will enable scientists to create different types of motor neurons and study why some are more vulnerable to disease than others, could help scientists better understand ALS, primary lateral sclerosis (PLS), progressive muscular atrophy (PMA) and other motor neuron diseases. (University of Edinburgh, March 1, 2011)