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Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report

Received: 9 June 2015     Accepted: 10 June 2015     Published: 30 June 2015
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Abstract

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.

Published in American Journal of Bioscience and Bioengineering (Volume 3, Issue 4-1)

This article belongs to the Special Issue Stem Cells for Neuro-Regeneration: Where Do We Stand

DOI 10.11648/j.bio.s.2015030401.15
Page(s) 30-33
Creative Commons

This is an Open Access article, distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium or format, provided the original work is properly cited.

Copyright

Copyright © The Author(s), 2015. Published by Science Publishing Group

Keywords

Neuroregeneration, Spinal Muscle Atrophy, Mesenchymal Stem Cells

References
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[4] 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.
[5] 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.
[6] Arnold WD, Kassar D, Kissel JT.(2015): Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle Nerve.;51(2):157-67.
[7] 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.
[8] 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.
[9] 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.
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[12] Lunn JS, Sakowski SA, Hur J, Feldman E (2011): Stem Cell Technology for Neurodegenerative diseases. Ann Neurol. 70(3):353-361.
[13] 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.
[14] 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.
[15] Norrgard K (2008): Medical Ethics: Genetic Testing and Spinal Muscular Atrophy. Nature Education 1(1):88.
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  • APA Style

    Abo Elkheir W., Gabr H., Salah Y. (2015). Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report. American Journal of Bioscience and Bioengineering, 3(4-1), 30-33. https://doi.org/10.11648/j.bio.s.2015030401.15

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    ACS Style

    Abo Elkheir W.; Gabr H.; Salah Y. Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report. Am. J. BioSci. Bioeng. 2015, 3(4-1), 30-33. doi: 10.11648/j.bio.s.2015030401.15

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    AMA Style

    Abo Elkheir W., Gabr H., Salah Y. Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report. Am J BioSci Bioeng. 2015;3(4-1):30-33. doi: 10.11648/j.bio.s.2015030401.15

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  • @article{10.11648/j.bio.s.2015030401.15,
      author = {Abo Elkheir W. and Gabr H. and Salah Y.},
      title = {Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report},
      journal = {American Journal of Bioscience and Bioengineering},
      volume = {3},
      number = {4-1},
      pages = {30-33},
      doi = {10.11648/j.bio.s.2015030401.15},
      url = {https://doi.org/10.11648/j.bio.s.2015030401.15},
      eprint = {https://article.sciencepublishinggroup.com/pdf/10.11648.j.bio.s.2015030401.15},
      abstract = {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.},
     year = {2015}
    }
    

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  • TY  - JOUR
    T1  - Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report
    AU  - Abo Elkheir W.
    AU  - Gabr H.
    AU  - Salah Y.
    Y1  - 2015/06/30
    PY  - 2015
    N1  - https://doi.org/10.11648/j.bio.s.2015030401.15
    DO  - 10.11648/j.bio.s.2015030401.15
    T2  - American Journal of Bioscience and Bioengineering
    JF  - American Journal of Bioscience and Bioengineering
    JO  - American Journal of Bioscience and Bioengineering
    SP  - 30
    EP  - 33
    PB  - Science Publishing Group
    SN  - 2328-5893
    UR  - https://doi.org/10.11648/j.bio.s.2015030401.15
    AB  - 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.
    VL  - 3
    IS  - 4-1
    ER  - 

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Author Information
  • Immunology Department, Military Medical Academy, Cairo, Egypt

  • Clinical Pathology Department, Faculty of Medicine, Cairo University, Cairo Egypt

  • Anaesthesia Department, Military Medical Academy, Cairo, Egypt

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