Allogeneic mesenchymal stem cell therapy outcomes for three patients with spinal muscular atrophy type 1

American Journal of Physical Medicine and Rehabilitation

Volumn 94, Issue 5, 2015, Pages 410-415

  Villanova, Marcello ^a   Bach, John Robert ^b  

^a Ospedale Privato Nigrisoli Dermatology   (Italy)

^b RUTGERS NEW JERSEY MEDICAL SCHOOL   (United States)

Author keywords

Allogenic Mesenchymal Stem Cells; Spinal Muscular Atrophy Type 1; Treatment Outcomes

Indexed keywords

ALLOTRANSPLANTATION; CASE REPORT; DISEASE COURSE; FEMALE; HUMAN; MALE; MESENCHYMAL STEM CELL TRANSPLANTATION; NEWBORN; PROCEDURES; SPINAL MUSCULAR ATROPHIES OF CHILDHOOD; TREATMENT OUTCOME;

DISEASE PROGRESSION; FEMALE; HUMANS; INFANT, NEWBORN; MALE; MESENCHYMAL STEM CELL TRANSPLANTATION; SPINAL MUSCULAR ATROPHIES OF CHILDHOOD; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME;

EID: 84928121510     PISSN: 08949115     EISSN: 15377385     Source Type: Journal    
DOI: 10.1097/PHM.0000000000000309     Document Type: Article

References (25)

1. Ogino S, Wilson RB: Spinal muscular atrophy: Molecular genetics and diagnostics. Expert Rev Mol Diagn 2004;4:15-29
2. Swoboda KJ, Prior TW, Scott CB, et al: Natural history of denervation in SMA: Relation to age, SMN2 copy number, and function. Ann Neurol 2005; 57:704-12
3. Bach JR, Gupta K, Reyna M, et al: Spinal muscular atrophy type 1: Prolongation of survival by noninvasive respiratory aids. Pediat Asthma, Allergy Immunol 2009;22:151-62
4. Maldonado-Soto AR, Oakley DH, Wichterle H, et al: Stem cells in the nervous system. Am J Phys Med Rehabil 2014;93:S132-44
5. Tanna T, Sachan V: Mesenchymal stem cells: Potential in treatment of neurodegenerative diseases. Curr Stem Cell Res Ther 2014;9:513-21
6. Minguell JJ, Allers C, Lasala GP: Mesenchymal stem cells and the treatment of conditions and diseases: The less glittering side of a conspicuous stem cell for basic research. Stem Cells Dev 2013;22:193-203
7. Bach JR, Niranjan V, Weaver B: Spinal muscular atrophy type 1: A noninvasive respiratory management approach. Chest 2000;117:1100-5
8. Bach JR, Baird JS, Plosky D, et al: Spinal muscular atrophy type 1: Management and outcomes. Pediatr Pulmonol 2002;34:16-22
9. Glanzman AM, McDermott MP, Montes J, et al; Pediatric Neuromuscular Clinical Research Network for Spinal Muscular Atrophy (PNCR); Muscle Study Group (MSG): Validation of the Children-s Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND). Pediatr Phys Ther 2011;23:322-6
10. Bach JR: Update and perspectives on noninvasive respiratory muscle aids: Part 2VThe expiratory muscle aids. Chest 1994;105:1538-44
11. Carrozzi M, Amaddeo A, Biondi A, et al: Stem cells in severe infantile spinal muscular atrophy (SMA1). Neuromuscul Disord 2012;22:1032-4
12. Yamout B, Hourani R, Salti H, et al: Bone marrow mesenchymal stem cell transplantation in patients with multiple sclerosis: A pilot study. J Neuroimmunol 2010;227:185-9
13. Karussis D: Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis. Arch Neurol 2010;67:1187-94
14. Connick P, Kolappan M, Crawley C, et al: Autologous mesenchymal stem cells for the treatment of secondary progressive multiple sclerosis: An open-label phase 2a proof-of-concept study. Lancet Neurol 2012;11:150-6
15. Kumar SKV, Balaraju S, Radhakrishnan RC, et al: Open-labeled study of unilateral autologous boneumbilicalmarrow-derived mesenchymal stem cell transplantation in Parkinson-s disease. Transl Res 2010;155:62-70
16. Venkataramana NK, Pal R, Rao SAV, et al: Bilateral transplantation of allogenic adult human bone marrow-derived mesenchymal stem cells into the subventricular zone of Parkinson-s disease: A pilot clinical study. Stem Cells Int 2012;2012:931902 doi: 10.1155/2012/931902
17. Nakajima H, Uchida K, Guerrero AR, et al: Transplantation of mesenchymal stem cells promotes an alternative pathway of macrophage activation and functional recovery after spinal cord injury. J Neurotrauma 2012;29:1614-25
18. Murphy MB, Moncivais K, Caplan AI: Mesenchymal stem cells: Environmentally responsive therapeutics for regenerative medicine. Exp Mol Med 2013; 45:e54
19. Paradisi M, Alviano F, Pirondi S, et al: Human mesenchymal stem cells produce bioactive neurotrophic factors: Source, individual variability and differentiation issues. Int J Immunopathol Pharmacol 2014;27: 391-402
20. Andolina M: Treatment of spinal muscular atrophy with intrathecal mesenchymal cells. Int J Stem Cells 2012;5:73-5
21. Liu J, Han D, Wang Z, et al: Clinical analysis of the treatment of spinal cord injury with umbilical cord mesenchymal stem cells. Cytotherapy 2013; 15:185-91
22. Mercuri E, Bertini E: Stem cells in severe infantile spinal muscular atrophy. Neuromuscul Disord 2012; 22:1105-7
23. Finkel RS: Stem cells in severe infantile spinal muscular atrophy (SMA1). Neuromuscul Disord 2012; 22:1105-6
24. Cohn RD: Stem cells in severe infantile spinal muscular atrophy (SMA1). Neuromuscul Disord 2012; 22:1106-7
25. Ankrum JA, Faii Ong J, Karp JM: Mesenchymal stem cells: Immune evasive, not immune privileged. Nat Biotechnol 2014;32:252-60