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Stem Cell Therapy Perspectives in Treating Motor Neuron Diseases: ALS and SMA |
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| Published Date : April 2010 |
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Pages : 31 |
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Summary Motor neuron diseases (MNDs) are characterized by gradual and progressive degeneration and death of motor neurons. Normally, messages from nerve cells in the brain, or upper motor neurons, are transmitted to nerve cells in the brain stem and spinal cord, known as lower motor neurons, and from there to skeletal muscles. Upper motor neurons direct the lower motor neurons to produce movements such as walking or chewing. Lower motor neurons control movement in the arms, legs, chest, face, throat, and tongue. Currently, there is no cure for MNDs. Motor neuron diseases include: amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease), post-polio syndrome (PPS), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), pseudobulbar palsy (spastic), progressive bulbar palsy (spastic and flaccid), and spinal muscular atrophy (SMA). However, currently only amyotrophic lateral sclerosis (ALS, Lou Gehrigs disease) and spinal muscular atrophy (SMA Type I) attract attention of various companies as potential targets for stem cell therapy. Stem Cell Therapy Perspectives in Treating Motor Neuron Diseases: ALS and SMA pipeline contains 7 R & D products undergoing development by 6 companies, all from the USA. Out of 7 products one product is in Phase I/II, three are in Phase I clinical trials, and three products are in preclinical stage of development. Six products are undergoing development for ALS, and one for ALS and SMA. The majority of adult stem cells used for the treatment of ALS and SMA are autologous, only one stem cells-based product is allotransplant. Patients own bone marrow was source of adult stem cells in four products, patients own skin in one product, fetal spinal cord tissue in one product, and embryonic stem cells in one product. Motor neurons were differentiated for use in two products. If there are no major setbacks, including alarming adverse effects, Expects that this pipeline will progress relatively efficiently. Possible positive therapeutic effects of motor neuron cell-based products for the treatment of both ALS and SMA may be expected. When evaluating results of products in this pipeline, it is important to remember that alternative for patients is death, and any positive result will have enormous significance. |
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Table of Contents : |
1. Amyotrophic Lateral Sclerosis (ALS, Lou Gehrigs Disease) 2. Spinal Muscular Atrophy (SMA Type I) 3. Brain, Spinal Cord and Neural Cells 3.1. Neurons 3.2. Oligodendrocytes, Astrocytes and Ependymal cells. 4. Stem Cells 4. 1. Adult Stem Cells 4.1.1. Origin of Adult Stem Cells 4.1.2. Sources of Adult Stem Cells 4.1.3. Neural Stem Cells 4.1.4. Neural Stem-Like Cells 4.1.5. Mesenchymal Stem Cells (MSCs) 4.1.5.1. Criteria for Definition of Mesenchymal Stem Cells 4.1.6. Mesenchymal-Like Stem Cells 4.1.7. Advantages and Disadvantages of Adult Stem Cells for Therapeutic Applications 4.1.8. Manipulation of Adult Stem Cells Ex Vivo 4.1.8.1. Isolation and Purification of Adult Stem Cells 4.1.8.1.1. Isolation of Neural Stem Cells 4.1.8.2. Expansion of Adult Stem Cells in Vitro (Bioreactors) 4.1.8.2.1. Expansion of Adult Neural Stem Cells 4.1.8.3. Adult Stem Cell Differentiation in Vitro (Biochips) 4.1.8.3.1. Adult Neural Stem Cells Differentiation in Vitro 4.1.8.4. Stimulation of Adult Neural Stem Cells Differentiation in Vitro 4.1.8.5. Encapsulation of Adult Stem Cells 4.1.8.6. Cryopreservation of Adult Stem Cells 4.1.9. Manipulation of Adult Stem Cells in Vivo 4.1.9.1. Activation of Dormant Adult Stem Cells in Vivo 4.1.9.2. Stimulation of Proliferation and Differentiation of Adult Stem Cells in Vivo 4.1.9.3. Mobilization of Adult Stem Cells in Vivo Motor neuron diseases include: amyotrophic lateral sclerosis (ALS, Lou Gehrig’s disease), post-polio syndrome (PPS), primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), pseudobulbar palsy (spastic), progressive bulbar palsy (spastic and flaccid), and spinal muscular atrophy (SMA). However, currently only amyotrophic lateral sclerosis (ALS, Lou Gehrigs disease) and spinal muscular atrophy (SMA Type I) attract attention of various companies as potential targets for stem cell therapy. Stem Cell Therapy Perspectives in Treating Motor Neuron Diseases: ALS and SMA pipeline contains 7 R & D products undergoing development by 6 companies, all from the USA. Out of 7 products one product is in Phase I/II, three are in Phase I clinical trials, and three products are in preclinical stage of development. Six products are undergoing development for ALS, and one for ALS and SMA. The majority of adult stem cells used for the treatment of ALS and SMA are autologous, only one stem cells-based product is allotransplant. Patients own bone marrow was source of adult stem cells in four products, patients own skin in one product, fetal spinal cord tissue in one product, and embryonic stem cells in one product. Motor neurons were differentiated for use in two products. If there are no major setbacks, including alarming adverse effects, BioPolaris expects that this pipeline will progress relatively efficiently. Possible positive therapeutic effects of motor neuron cell-based products for the treatment of both ALS and SMA may be expected. When evaluating results of products in this pipeline, it is important to remember that alternative for patients is death, and any positive result will have enormous significance. Content: 5. Embryonic Stem Cells 5.1. Advantages and Disadvantages of Embryonic Stem Cells for Therapeutic Use 5.2. Growth of Embryonic Stem Cells 5.3. Differentiation of Embryonic Stem Cells 5.3.1. Differentiation of Embryonic Stem Cells into Neurons, Including Motor Neurons and Glial Cells 6. Engineered Stem Cells. 6.1. Induced pluripotent stem cells (iPSCs) 7. Effects of ALS and SMA on Blood-Brain Barrier and Adult Stem Cells 8. Stem Cell Therapy for Motor Neuron Diseases 8.1. Types of Adult Stem Cell Transplantations for Motor Neuron Diseases 8.2. Stem Cell Therapy for ALS 8.2.1. Adult Stem Cells for ALS 8.2.1.1. Mesenchymal Stem Cells for ALS 8.2.1.1.1. Engineered Mesenchymal Stem Cells (MSCs) for ALS 8.2.1.2. Mesenchymal-Like Stem Cells for ALS 8.2.1.3. Neural Stem Cells for ALS 8.2.1.4. Engineered Neural Stem Cells for ALS 8.2.1.5. Combination of Adult Stem Cell Therapy for ALS 8.2.2. Embryonic Stem Cells-Derived Motor Neurons for ALS 8.2.3. Induced Pluripotent Stem Cells (iPSCs) for ALS 8.2.4. Results of Completed Stem Cell Therapy Clinical Studies and Trials in ALS Patients 8.3. Stem Cell Therapy for SMA 8.3.1. Adult Neural Stem Cells for SMA 8.3.2. Mesenchymal Stem Cells for SMA 8.3.3. Embryonic Stem Cell-Derived Motor Neurons for SMA 8.3.4. iPSCs for SMA 9. Pipeline Analysis and Expectations 10. Products Profiles 11. Companies and Investors 11.1. Company Profiles 12. Conclusion A. Tables Table 1. Adult Stem Cells-Derived Mature Neural Cells Table 2. Stem Cell R & D Therapeutic Products for Motor Neuron Diseases Table 3. Type and Sources of Stem Cells used for the Treatment of MNDs Table 4. Companies and Investors B. Illustrations Illustration 1. Basic Neuron Design Illustration 2. Diffusion of Neurotransmitters Across the Synaptic Cleft Illustration 3. DPN® renders precise nanopatterns capable of producing a homogeneous population of differentiated adult cells C. Photos Photo 1. Oligodendrocyte. Transfected with GFP (Green Fluorescent Protein). Three days in vitro mouse Photo 2. This is an astrocyte, labeled with GFAP (red), Focal Adhesion Kinase (FAK) green, and nuclear stain To-Pro (blue) Photo 3. Two neurospheres, compact masses of neuron precursor cells, derived from human embryonic stem cells, as captured by a fluorescent microscope. Differentiated neurons, whose nuclei are shown in red, have begun to extend neuronal processes (green) toward one another, forming connections |
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Published By :BioPolaris |
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