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Stem Cell Therapy Perspectives in Treating Motor Neuron Diseases: ALS and SMA

BioPolaris, April 2010, Pages: 31


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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 Gehrig's 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, the publisher 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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