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Currently there are only two FDA approved treatments for the treatment of amyotrophic lateral sclerosis (ALS). These treatments are only modestly effective, partly because ALS is not completely understood by healthcare professionals. Recently at Massachusetts General Hospital there has been research into the inflammatory processes that may contribute to the onset and progression of the disease. This research has led to a study of cromolyn sodium and its affect on ALS in mice. The results of this study were promising, and they give researchers hope that there will soon be a new treatment for ALS.
About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic lateral sclerosis (ALS) is a progressive, neurological disease that destroys nerve cells in the brain stem, brain, and spinal cord. Because of this loss of nerve cells, muscles weaken and people lose control and voluntary movement. As the disease progresses, eventually it weakens the muscles necessary for breathing, leading to death. There are two types of this disease: sporadic and familial. Sporadic is the most common, with 90-95% of cases falling into this category. The latter means that it is inherited. ALS is a rare disease, affecting 3.9 of every 100,000 people in the United States. While all races and sexes are affected, white males aged 60-69 are at the highest risk.
The cause of ALS is unknown. In familial cases it is known that there is a mutated gene that is inherited from parents, but it is still not fully understood and only accounts for 5-10% of cases. It is believed that there is a connection between frontotemporal dementia and ALS. Other theories point to exposure to certain substances or toxins could lead to the development of ALS.
Symptoms of ALS vary from patient to patient. They also worsen as the disease progresses. Symptoms may begin as difficulty with small movements and everyday things like walking. At the beginning of the disease, people may trip and feel weakness in their arms, hands, and legs. As it progresses, people experience difficulties with speaking and swallowing, slowed and slurred speech, twitches and cramps in the muscles, and difficulty holding good posture. At the end of the progression people will be unable to move their muscles gradually, which affects the entire body. This inability affects movements like blinking. While people with ALS experience loss of muscle function, they do not lose any of their cognitive abilities. Their senses are generally not affected either.
Diagnosis comes after a physician notices the symptoms and performs tests to rule out other conditions like lyme disease, HIV, or multiple sclerosis. They often conduct blood tests to accomplish this. After other conditions are no longer possibilities, doctors will use electromyographies (EMGs), nerve conduction studies (NCSs), and magnetic resonance imaging (MRIs). After a diagnosis is obtained, treatment is often symptomatic. There is no cure for ALS. Treatment includes physical and speech therapy, nutritional and ventilation support, medication for depression or anxiety, medication to relieve tenseness and pain in muscles, hospice care, and riluzole, which reduces damage to the motor neurons.
About the Study
Cromolyn sodium is an FDA approved drug used for the treatment of asthma and other conditions. Researchers from Massachusetts General Hospital and the Healey Center found that this drug exhibited neuroprotective effects in ALS models in mice and at the cellular level. These researchers discovered these effects by injecting the drug into male and female mice with and without the mutation for ALS. They noticed that the cromolyn sodium delayed the development of symptoms, protected neurons from degeneration, helped to maintain the connection between the nerves and muscles, and reduced inflammation around the muscles.
They also found that inflammation around the muscles may play a larger role in the progression of ALS than previously thought. This insight may lead to new methods of treatment along with the use of cromolyn sodium. Although it is not yet known if the results of the mouse model will translate to humans, researchers are hopeful that this new information will lead to the better treatment of ALS.
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