Surrounding neurons with healthy adjoining cells completely stops motor neuron death in some cases. Hence stem cell transplantation might represent a promising therapeutic strategy.
Stem cell treatment for ALS / Motor Neuron disease is still rather in its infancy but results of successful treatment are very promising, with patients having increased life span following treatment.
With the lack of effective drug treatments for amyotrophic lateral sclerosis, stem cell research has highlighted this disease as a candidate for stem cell treatment. Stem cell transplantation is an attractive strategy for neurological diseases and early successes in animal models of neurodegenerative disease generated optimism about restoring function or delaying degeneration in human beings. Autologous or allogeneic stem cells are the candidate source for local or systemic cell-therapies in ALS. Stem cells isolated and expanded in culture can be modified to release growth factors and generate glial cells following transplantation into the spinal cord or brain. As such, they might be able to both detoxify the local environment around dying motor neurons and deliver atrophic factors.
In a recent study patients received intraspinal injections of autologous mesenchymal stem cells at the thoracic level and were monitored for four years. No significant side effects were evident. No modification of the spinal cord volume or other signs of abnormal cell proliferation were observed. Four patients showed a significant slowing down of their respiratory function and improvement in their quality of life scores.
Our results seem to demonstrate that MSCs represent a good chance for stem cell based therapy in ALS and that intraspinal injection of MSCs is also safe in the long term. Recently it has been shown in animal models of amyotrophic lateral sclerosis that stem cells significantly slow the progression of the disease and prolong survival. All of our patients with ALS are treated under the protocols of a clinical trial in order to document the results for future patients.
Amyotrophic lateral sclerosis is a progressive, usually fatal, neurodegenerative disease caused by the degeneration of motor neurons, the nerve cells in the central nervous system that control voluntary muscle movement. Its symptoms are caused by the death of the anterior horn cell in the spinal cause.
The reason behind this is unknown; it is thought that reactive astrogliosis and microglia activation may play a role in causing this disease.
Motor neurons are nerve cells located in the brain, brainstem, and spinal cord that control and communicate between the nervous system and the voluntary muscles of the body. In ALS, both the upper motor neurons and the lower motor neurons degenerate or die, ceasing to send messages to muscles. Unable to function, the muscles gradually weaken, waste away (atrophy), and twitch (fasciculation) because of degeneration. Eventually, the ability of the brain to start and control voluntary movement is lost.
The onset of ALS may be so subtle that the symptoms are frequently overlooked. The earliest symptoms may include twitching, cramping, or stiffness of muscles; muscle weakness affecting an arm or a leg; slurred and nasal speech; or difficulty chewing or swallowing. These general complaints then develop into more obvious weakness or atrophy that may cause a physician to suspect ALS.
The parts of the body affected by early symptoms of ALS depend on which muscles in the body are damaged first. In some cases, symptoms initially affect one of the legs, and patients experience awkwardness when walking or running or they notice that they are tripping or stumbling more often. Some patients first see the effects of the disease on a hand or arm as they experience difficulty with simple tasks requiring manual dexterity, such as buttoning a shirt, writing, or turning a key in a lock. Other patients notice speech problems.
Regardless of the part of the body first affected by the disease, muscle weakness and atrophy spread to other parts of the body as the disease progresses. Patients have increasing problems with moving, swallowing (dysphagia), and speaking or forming words (dysarthria). Symptoms of upper motor neuron involvement include tight and stiff muscles (spasticity) and exaggerated reflexes (hyperreflexia), including an overactive gag reflex. An abnormal reflex commonly called Babinski’s sign (the large toe extends upward as the sole of the foot is stimulated in a certain way) also indicates upper motor neuron damage. Symptoms of lower motor neuron degeneration include muscle weakness and atrophy, muscle cramps, and fleeting twitches of muscles that can be seen under the skin (fasciculations).
To be diagnosed with ALS, patients must have signs and symptoms of both upper and lower motor neuron damage that cannot be attributed to other causes.
ALS causes weakness with a wide range of disabilities (see below). Eventually, all muscles under voluntary control are affected, and patients lose their strength and the ability to move their arms, legs, and body. When muscles in the diaphragm and chest wall fail, patients lose the ability to breathe without ventilatory support. Most people with ALS die from respiratory failure, usually within 3 to 5 years from the onset of symptoms. However, about 10 percent of ALS patients survive for 10 or more years.
Although the disease usually does not impair a person’s mind or intelligence, several recent studies suggest that some ALS patients may have alterations in cognitive functions such as depression and problems with decision-making and memory.
Although the sequence of emerging symptoms and the rate of disease progression vary from person to person, eventually patients will not be able to stand or walk, get in or out of bed on their own, or use their hands and arms. Difficulty swallowing and chewing impair the patient’s ability to eat normally and increase the risk of choking. Maintaining weight will then become a problem. Because the disease usually does not affect cognitive abilities, patients are aware of their progressive loss of function and may become anxious and depressed. A small percentage of patients may experience problems with memory or decision-making, and there is growing evidence that some may even develop a form of dementia.
Health care professionals need to explain the course of the disease and describe available treatment options so that patients can make informed decisions in advance. In later stages of the disease, patients have difficulty breathing as the muscles of the respiratory system weaken. Patients eventually lose the ability to breathe on their own and must depend on ventilatory support for survival. Patients also face an increased risk of pneumonia during later stages of ALS.
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