Spinal muscular atrophy, more commonly known as SMA, afflicts millions of individuals around the world. Considered a neuromuscular disease, the condition causes a degeneration or destruction of motor neurons, which transmit signals from the brain to the muscles. The condition causes eventual atrophy or wasting away of muscles throughout the body, and can affect all age groups and genders.
Muscle weakness, impaired movement and range of motion are the most common symptoms of SMA, caused by the inability of motor neurons from the spinal cord to reach the muscles. Patients may experience relatively minor symptoms to fatal symptoms, depending on the stage and manifestations of the disease process in any given individual.
While there is no known cure to date for SMA, stem cell therapies and technologies have shown positive results in treating a variety of symptoms associated with the condition. Gene replacement and stem cell replacement therapies involving the implantation of healthy and completely functional motor neurons into individuals diagnosed with the condition are showing promising results, especially in the increase of the production of the specific SMN 2 gene responsible for healthy motor neuron development.
Stem cell research facilities in treatment centers around the world have begun treating SMA symptoms with embryonic stem cell transplants and implantation therapies, offering remission in up to 75% of cases, as well as helping to diminish the symptoms of neurogenic dystrophy in patients in 90% of cases in some clinical and treatments studies.
Clinical trials in stem cell treatments and therapies for spinal muscular atrophy are focused on determining efficacy data, assuring safety, and quality assurance in the implementation of testing, currently under way in multiple facilities throughout the United States, including the University of California, California Stem Cell, Inc., and John Hopkins. Facilities in the Ukraine, Western Europe, Asia, and Latin America are also conducting clinical trials, therapies and treatments utilizing stem cells in the treatment of spinal muscular atrophy.
To date, spinal muscular atrophy and Lou Gehrig's disease, most commonly known as ALS, or amyotrophic lateral sclerosis, have attracted attention by companies around the world as having the greatest potential in motor neuron disease processes that may benefit from targeted stem cell therapies. Clinical trials throughout the United States are engaged in Phase I and II clinical testing in the use of autologous stem cell transplants. Autologous stem cell transplants utilize a patient's own bone marrow as a source of adult stem cells, while other studies are utilizing fetal spinal cord tissues or embryonic stem cells. To date, researchers have seen positive benefits of utilizing motor neuron cell based transplants and implantation therapies for the treatment of both SMA and ALS.
Stem cell therapy to treat SMA focuses on completely replacing motor neurons that have been damaged or destroyed due to the disease process. Not only will such treatments slow down the disease progression, but may also be used to treat a variety of disorders including spinal cord injuries or other neuromuscular damage or disease processes that interrupts the flow of information signals controlling nerve and muscular movement.
Stem cell treatments and technologies offer hope in the future for enhancing quality of life, restoring mobility and muscular function as well as range of motion and stability for individuals currently diagnosed with SMA.
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