Amyotrophic lateral sclerosis (ALS), also known as Lou-Gehrig’s disease, is a neurodegenerative condition characterized by progressive degeneration of the motor nerves. According to statistics, the worldwide incidence of the disease is 2.5 per 100,000 persons. The complexity of the ALS makes it difficult to develop effective therapies. Stem cell therapy is a promising treatment option because these cells have the ability to transform into multiple neuronal lineages.
Animal Studies involving Stem Cell Therapy
Neural stem cells have been studied for beneficial effects in rats with ALS-like illness. The transplanted cells were noted to change into neurons (nerve cells), form synaptic connections with body tissues, slow disease progression, and prolong survival. In another clinical study, grafted neural stem cells protected nearby motor neurons and achieved transient functional improvement. In addition, the disease symptoms improve, and mice subjections had enhanced motor function. ALS mice treated with stem cells were found to live three times longer than non-grafted mice.
Several studies with animal models have investigated injections of stem cells peripherally or directly into the spinal cord. The systemic administration of adipose-derived mesenchymal stem cells into mice with motor deterioration. At the 6 weeks evaluation, researchers noted delayed motor deterioration and up-regulated levels of growth factor. These findings indicate that stem cells promote neuroprotection and prolonged life. In another clinical study, stem cells were genetically modified to release growth factors, and then injected into mice subjects. Results proved stem cells extended survival, alleviate lost motor function, and slowed disease progression.
Human Studies involving Stem Cell Therapy
In 2009, the FDA approved a clinical trial in human subjects to investigate the tolerability and safety of surgically implanted stem cells. In this study, 18 patients with ALS received intraspinal transplanted stem cells. After monitoring the study subjects for 18 months, researchers found that all patients tolerated the procedure without complications, and their conditions did not worsen. An expansion of this study was conducted a year later. The researchers noted safety and tolerability of stem cells in all patients.
Another group of researchers evaluated autologous MSC transplantation. The researchers noted that there were no signs of toxicity or abnormal cell growth, and four patients had improvement from the treatment. The cells were found to differentiate into specialized neuron-supporting cells that secreted neurotrophic factors. Because ALS does not influence MSC expansion and transformation, these cells can be extracted from the patient’s own body. This is used to bypass the immune rejection response.
In 2008, a group of researchers developed a strain of human stem cells from an 82-year-old woman with ALS. The cells were derived from the patient’s own somatic cells, and expressed stem cell markers. Called IPSCs, these cells can give rise to all three germ layers, so they are useful for treating ALS. The researchers found that the stem cell-derived neural progenitors could have a beneficial effect in replacing lost motor neurons, as well as offering potent neurotrophic factors. These effects are postulated to reduce macro- and microgliosis, which means increased resistance to motor neuron degeneration and death.
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Sun H, Hou Z, Yang H, Meng M, Li P, Zou Q, Yang L. et al. Multiple systemic transplantations of human amniotic mesenchymal stem cells exert therapeutic effects in an ALS mouse model. Cell Tissue Res. 2014;357(3):571–582. doi: 10.1007/s00441-014-1903-z.
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The most revolutionary regenerative medicine treatments now being offered include amniotic and umbilical stem cell treatments. These are FDA regulated and contain growth factors, hyaluronic acid, cytokines and stem cells.