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Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report
American Journal of Bioscience and Bioengineering
Volume 3, Issue 4-1, July 2015, Pages: 30-33
Received: Jun. 9, 2015; Accepted: Jun. 10, 2015; Published: Jun. 30, 2015
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Abo Elkheir W., Immunology Department, Military Medical Academy, Cairo, Egypt
Gabr H., Clinical Pathology Department, Faculty of Medicine, Cairo University, Cairo Egypt
Salah Y., Anaesthesia Department, Military Medical Academy, Cairo, Egypt
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Rationale : Spinal Muscular Atrophy (SMA) is the most common genetic disorder and presents the most common cause of infant mortality. To date, patient management is symptomatic and focuses on improvement of independence and treatment of complications. Stem cell therapy represents a novel therapeutic option for many neurological diseases. Presenting concerns of the patient: This patient with type III SMA presented with generalized hypotonia and muscle weakness with inability to raise hands and legs, support back or neck, in addition to respiratory distress. Diagnosis: Clinical examination showed hypotonia and loss of reflexes. Creatine kinase levelxxx, electrophysiologyxx. Interventions: Allogenic mesenchymal stem cells (MSCs) were injected in a dose of xxx intrathecally and a dose of xxx injected systemically. Outcomes: The patient showed improvement of GFM score and upgrading of the GFMC grade from Grade V to Grade III in 3 months. Improved quality of life was reflected in improvement of the PEDI scores. Improvement was noticed in respiration. No complications were encountered. Improvement was maintained until date. Conclusions: Allogenic MSC therapy may present a new therapeutic strategy for SMA patients. Controlled clinical trials are recommended to document the safety and efficacy of the procedure.
Neuroregeneration, Spinal Muscle Atrophy, Mesenchymal Stem Cells
To cite this article
Abo Elkheir W., Gabr H., Salah Y., Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report, American Journal of Bioscience and Bioengineering. Special Issue: Stem Cells for Neuro-Regeneration: Where Do We Stand. Vol. 3, No. 4-1, 2015, pp. 30-33. doi: 10.11648/
Lascone DM, Henderson CE, Lee JC (2015): Spinal muscular atrophy: from tissue specificity to therapeutic strategies. F1000Prime Rep 5(7):04.
Cohen S, Nathan JA, Goldberg AL (2015): Muscle wasting in disease: molecular mechanisms and promising therapies. Nat Rev Drug Discov 14(1):58-74.
Yoshida M, Kitaoka S, Egawa N, Yamane M, Ikeda R, Tsukita K, Amano N, Watanabe A, Morimoto M, Takahashi J, Hosoi H, Nakahata T, Inoue H, Saito MK (2015): Modeling the Early Phenotype at the Neuromuscular Junction of Spinal Muscular Atrophy using patient-derived iPSCs. Stem Cell Reports. S2213-6711(15)00066-1.
Hua Y, Liu YH, Sahashi K, Rigo F, Bennett CF, Krainer AR (2015): Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models. Genes Dev.;29(3):288-97.
Harding BN, Kariya S, Monani UR, Chung WK, Benton M, Yum SW, Tennekoon G, Finkel RS. (2015): Spectrum of neuropathophysiology in spinal muscular atrophy type I. J Neuropathol Exp Neurol.;74(1):15-24.
Arnold WD, Kassar D, Kissel JT.(2015): Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle Nerve.;51(2):157-67.
Porro F, Rinchetti P, Magri F, Riboldi G, Nizzardo M, Simone C, Zanetta C, Faravelli I, Corti S.(2014): The wide spectrum of clinical phenotypes of spinal muscular atrophy with respiratory distress type 1: a systematic review. J Neurol Sci. ;346(1-2):35-42.
Cottler-Fox MH1, Lapidot T, Petit I, Kollet O, DiPersio JF, Link D, Devine S. (2003): Stem cell mobilization. Am Soc Hematol Educ Program 419-37.
Dominici M, Le Blanc K, Mueller I, Slaper-Cortenback I, Marini F, Krause D, Deans R, Keating A, Prockop Dj, Horwitz E (2006): Minimal criteria for defining multipotent mesenchymal stromal cells. The International Society for Cellular Therapy position statement. Cytotherapy 8(4):315-317.
Berard C, Payan C, Hodgkinson I, Fermanian J (2005): A motor function measure scale for neuromuscular diseases. construction and validation study. Neuromusular Disorders 463-470.
Wassenberg-Severijnen J, Custers J, Hox J, Vermeer A and Helders P (2003): Reliability of the Dutch Pediatric Evaluation of Disability Inventory (PEDI) . Clinical Rehabilitation 17: 457–462.
Lunn JS, Sakowski SA, Hur J, Feldman E (2011): Stem Cell Technology for Neurodegenerative diseases. Ann Neurol. 70(3):353-361.
Dantuma E, Merchant S and Sugaya K (2010): .Stem cells for the treatment of neurodegenerative diseases Stem Cell Research & Therapy , 1:37 doi:10.1186/scrt37.
Walker PA, Harting MT, Shah SK, Day MC, El-Khoury R, Savitz SI, Baumgartner J and Cox CS (2010): Progenitor Cell Therapy for the Treatment of Central Nervous System Injury: A Review of the State of Current Clinical Trials. Stem Cells International, volume 2010 .Article ID 369578, 8 pages.
Norrgard K (2008): Medical Ethics: Genetic Testing and Spinal Muscular Atrophy. Nature Education 1(1):88.
Gabr H, Abd El-Fattah R, Ahmed D, Farhan M, Mousa S (2011): Mesenchymal stem cells derived from bone marrow and leukapheresis show different putative subpopulations. Stem Cell Studies vol 1 (1).
Abo El-Kheir W, Gabr H, Awad MR, von Wild K, and Ramadan M (2010): Autologous Mesenchymal Stem Cells for Neuro-Regeneration After Traumatic Spinal Cord Injury: A Comparison Between Bone Marrow and Peripheral Blood Populations. Am J Neuroprot & Neuroreg Vol. 2, 1–8.
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