Transplantation of human umbilical cord blood cells in the repair of CNS diseases

Expert Opinion on Biological Therapy

Volumn 4, Issue 2, 2004, Pages 121-130

  Newman, Mary B ^a   Davis, Cyndy D ^b   Borlongan, Cesar V ^c,d,e   Emerich, Dwaine ^a   Sanberg, Paul R ^a  

^a UNIVERSITY OF SOUTH FLORIDA   (United States)

^b Saneron CCEL Therapeutics Inc   (United States)

^c MEDICAL COLLEGE OF GEORGIA   (United States)

^d School of Graduate Studies   (United States)

^e VETERANS AFFAIRS MEDICAL CENTER   (United States)

Author keywords

Amyotropic lateral sclerosis; Blood brain barrier; Cell transplantation; Developing brain; Gene therapy; Graft versus host disease; Haematopoietic cells; Human umbilical cord blood; Phenotype; Spinal cord injury; Stem cells; Stroke; Traumatic brain injury

Indexed keywords

AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL MODEL; BRAIN CELL; BRAIN DEVELOPMENT; BRAIN INJURY; CELL BASED GENE THERAPY; CELL REGENERATION; CELL TRANSPLANTATION; CENTRAL NERVOUS SYSTEM DISEASE; CENTRAL NERVOUS SYSTEM FUNCTION; CORD BLOOD STEM CELL TRANSPLANTATION; DEGENERATIVE DISEASE; FETAL TISSUE TRANSPLANTATION; GRAFT VERSUS HOST REACTION; HUMAN; HUMAN CELL; HUMAN EXPERIMENT; HUNTINGTON CHOREA; IN VITRO STUDY; MEDICAL ETHICS; PARKINSON DISEASE; PHENOTYPE; REVIEW; SPINAL CORD LESION; STANDARDIZATION; STEM CELL; STROKE; UMBILICAL CORD BLOOD;

ANIMALS; CELL TRANSPLANTATION; CENTRAL NERVOUS SYSTEM DISEASES; CEREBROVASCULAR ACCIDENT; FETAL BLOOD; GENE THERAPY; HUMANS; STEM CELL TRANSPLANTATION;

ANIMALIA;

EID: 1042288378     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712598.4.2.121     Document Type: Review

References (71)

1. BROXMEYER HE: Primitive hematopoietic stem and progenitor cells in human umbilical cord blood: an alternative source of transplantable cells. Cancer Treat. Res. (1996) 84:139-148.
2. MAYANI H, LANSDORP PM: Biology of human umbilical cord blood-derived hematopoietic stem/progenitor cells. Stem Cells (1998) 16(3):153-165.
3. NATIONAL CENTER FOR FARMWORKERS: National Vital Statistics Reports. Centers for Disease Control and Prevention, USA (2002).
4. HOWS JM, MARSH JC, BRADLEY BA et al.: Human cord blood: a source of transplantable stem cells? Bone Marrow Transplant. (1992) 9(Suppl. 1):105-108.
5. CARDOSO AA, LI ML, BATARD P et al.: Release from quiescence of CD34+ CD38- human umbilical cord blood cells reveals their potentiality to engraft adults. Proc. Natl. Acad. Sci. USA (1993) 90(18):8707-8711.
6. METCALF D: Clonal Culture of Hemopoietic Cells: Techniques and Applications. Elsevier Science Publishers B.V., The Netherlands (1984).
7. BROXMEYER HE, HANGOC G, COOPER S et al.: Growth characteristics and expansion of human umbilical cord blood and estimation of its potential for transplantation in adults. Proc. Natl. Acad. Sci. USA (1992) 89(9):4109-4113.
8. MIRAGLIA S, GODFREY W, YIN AH et al.: A novel five-transmembrane hematopoietic stem cell antigen: isolation, characterization, and molecular cloning. Blood (1997) 90(12):5013-5021.
9. YIN AH, MIRAGLIA S, ZANJANI ED et al.: AC133, a novel marker for human hematopoietic stem and progenitor cells. Blood (1997) 90(12):5002-5012.
10. POTGENS AJ, BOLTE M, HUPPERTZ B, KAUFMANN P, FRANK HG: Human trophoblast contains an intracellular protein reactive with an antibody against CD133-a novel marker for trophoblast. Placenta (2001) 22(7):639-645.
11. UCHIDA N, BUCK DW, HE D et al.: Direct isolation of human central nervous system stem cells. Proc. Natl. Acad. Sci. USA (2000) 97(26):14720-14725.
12. TAMAKI S, ECKERT K, HE D et al.: Engraftment of sorted/expanded human central nervous system stem cells from fetal brain. J. Neurosci. Res. (2002) 69(6):976-986.
13. BROXMEYER HE, SROUR EF, HANGOC G, COOPER S, ANDERSON SA, BODINE DM: High-efficiency recovery of functional hematopoietic progenitor and stem cells from human cord blood cryopreserved for 15 years. Proc. Natl. Acad. Sci. USA (2003) 100(2):645-650.
14. BROXMEYER HA: Cellular Characteristics of Cord Blood and Cord Blood Transplantation. Broxmeyer HA (Ed.), AABB Press, Bethesda, MA, USA (1998).
15. ORLIC D, GIRARD LJ, JORDAN CT, ANDERSON SM, CLINE AP, BODINE DM: The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction. Proc. Natl. Acad. Sci. USA (1996) 93(20):11097-11102.
16. ORLIC D, GIRARD LJ, ANDERSON SM et al.: Transduction efficiency of cell lines and hematopoietic stem cells correlates with retrovirus receptor mRNA levels. Stem Cells (1997) 15(Suppl. 1):23-28; discussion 28-29.
17. GLUCKMAN E, ROCHA V, BOYER-CHAMMARD A et al.: Outcome of cord-blood transplantation from related and unrelated donors. EurocordTransplant Group and the European Blood and Marrow Transplantation Group. N. Engl. J. Med. (1997) 337(6):373-381.
18. WAGNER JE, BROXMEYER HE, BYRD RL et al.: Transplantation of umbilical cord blood after myeloablative therapy: analysis of engraftment. Blood (1992) 79(7):1874-1881.
19. WAGNER JE, KERNAN NA, STEINBUCH M, BROXMEYER HE, GLUCKMAN E: Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet (1995) 346(8969):214-219.
20. VAZIRI H, DRAGOWSKA W, ALLSOPP RC, THOMAS TE, HARLEY CB, LANSDORP PM: Evidence for a mitotic clock in human hematopoietic stem cells: loss of telomeric DNA with age. Proc. Natl. Acad. Sci. USA (1994) 91(21):9857-9860.
21. MIYAWAKI T, MORIYA N, NAGAOKI T, TANIGUCHI N: Maturation of B-cell differentiation ability and T-cell regulatory function in infancy and childhood. Immunol. Rev. (1981) 57:61-87.
22. NAGAOKI T, MIYAWAKI T, CIORBARU R et al.: Maturation of B cell differentiation ability and T cell regulatory function during child growth assessed in a Nocardia water soluble mitogen-driven system. J. Immunol. (1981) 126(5):2015-2019.
23. UENO Y, MIYAWAKI T, SEKI H et al.: Differential effects of recombinant human interferon-gamma and interleukin 2 on natural killer cell activity of peripheral blood in early human development. J. Immunol. (1985) 135(1):180-184.
24. WILSON CB, WESTALL J, JOHNSTON L, LEWIS DB, DOWER SK, ALPERT AR: Decreased production of interferon-gamma by human neonatal cells. Intrinsic and regulatory deficiencies. J. Clin. Invest. (1986) 77(3):860-867.
25. COHEN RI, MCKAY R, ALMAZAN G: Cyclic AMP regulates PDGF-stimulated signal transduction and differentiation of an immortalized optic-nerve-derived cell line. J. Exp. Biol. (1999) 202(Pt 4):461-473.
26. LU L, SHEN RN, BROXMEYER HE: Stem cells from bone marrow, umbilical cord blood and peripheral blood for clinical application: current status and future application. Crit. Rev. Oncol. Hematol. (1996) 22(2):61-78.
27. GLUCKMAN E, BROXMEYER HA, AUERBACH AD et al.: Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. N. Engl. J. Med. (1989) 321(17):1174-1178.
28. BROXMEYER HE, GLUCKMAN E, AUERBACH A et al.: Human umbilical cord blood: a clinically useful source of transplantable hematopoietic stem/progenitor cells. Int. J. Cell Cloning (1990) 8(Suppl. 1):76-89; discussion 89-91.
29. LAUGHLIN MJ, BARKER J, BAMBACH B et al.: Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N. Engl. J. Med. (2001) 344(24):1815-1822.
30. BROXMEYER HE: Growth factors and cord blood stem and progenitor cells. Immunol. Invest. (1995) 24(1-2):391-402.
31. CARDOSO AA, LI ML, BATARD P et al.: Human umbilical cord blood CD34+ cell purification with high yield of early progenitors. J. Hematother. (1993) 2(2):275-279.
32. LU L, XIAO M, SHEN RN, GRIGSBY S, BROXMEYER HE: Enrichment, characterization, and responsiveness of single primitive CD34 human umbilical cord blood hematopoietic progenitors with high proliferative and replicating potential. Blood (1993) 81(1):41-48.
33. RAPPOLD I, ZIEGLER BL, KOHLER I et al.: Functional and phenotypic characterization of cord blood and bone marrow subsets expressing FLT3 (CD135) receptor tyrosine kinase. Blood (1997) 90(1):111-125.
34. TRAYCOFF CM, ABBOUD MR, LAVER J et al.: Evaluation of the in vitro behavior of phenotypically defined populations of umbilical cord blood hematopoietic progenitor cells. Exp. Hematol. (1994) 22(2):215-222.
35. MUJTABA T, PIPER DR, KALYANI A, GROVES AK, LUCERO MT, RAO MS: Lineage-restricted neural precursors can be isolated from both the mouse neural tube and cultured ES cells. Dev. Biol. (1999) 214(1):113-127.
36. CARPENTER MK, INOKUMA MS, DENHAM J, MUJTABA T, CHIU CP, RAO MS: Enrichment of neurons and neural precursors from human embryonic stem cells. Exp. Neurol. (2001) 172(2):383-397.
37. DIETRICH J, NOBLE M, MAYER-PROSCHEL M: Characterization of A2B5+ glial precursor cells from cryopreserved human fetal brain progenitor cells. Glia (2002) 40(1):65-77.
38. LENDAHL U, ZIMMERMAN LB, MCKAY RD: CNS stem cells express a new class of intermediate filament protein. Cell (1990) 60(4):585-595.
39. REYNOLDS BA, WEISS S: Generation of neurons and astrocytes from isolated cells of the adult mammalian central nervous system. Science (1992) 255(5052):1707-1710.
40. PALMER TD, MARKAKIS EA, WILLHOITE AR, SAFAR F, GAGE FH: Fibroblast growth factor-2 activates a latent neurogenic program in neural stem cells from diverse regions of the adult CNS. J. Neurosci. (1999) 19(19):8487-8497.
41. JOHANSSON CB, SVENSSON M, WALLSTEDT L, JANSON AM, FRISEN J: Neural stem cells in the adult human brain. Exp. Cell Res. (1999) 253(2):733-736.
42. SHIH CC, WENG Y, MAMELAK A, LEBON T, HU MC, FORMAN SJ: Identification of a candidate human neurohematopoietic stem-cell population. Blood (2001) 98(8):2412-2422.
43. POLTAVTSEVA RA, MAREY MV, ALEKSANDROVA MA, REVISHCHIN AV, KOROCHKIN LI, SUKHIKH GT: Evaluation of progenitor cell cultures from human embryos for neurotransplantation. Brain Res. Dev. Brain Res. (2002) 134(1-2):149-154.
44. KUKEKOV VG, LAYWELL ED, SUSLOV O et al.: Multipotent stem/progenitor cells with similar properties arise from two neurogenic regions of adult human brain. Exp. Neurol. (1999) 156(2):333-344.
45. SANCHEZ-RAMOS JR, SONG S, KAMATH SG et al.: Expression of neural markers in human umbilical cord blood. Exp. Neurol. (2001) 171(1):109-115.
46. HA Y, CHOI JU, YOON DH et al.: Neural phenotype expression of cultured human cord blood cells in vitro. Neuroreport (2001) 12(16):3523-3527.
47. BICKNESE AR, GOODWIN HS, QUINN CO, VERNEAKE CD: Human umbilical cord blood cells can be induced to express markers for neurons and glia. Cell Transplant. (2002) 11(3):261-264. This is a key paper demonstrating evidence that umbilical cord blood-derived stem cells have the capacity to differentiate into neural cells.
48. BUZANSKA L, MACHAJ EK, ZABLOCKA B, POJDA Z, DOMANSKA-JANIK K: Human cord blood-derived cells attain neuronal and glial features in vitro. J. Cell Sci. (2002) 115(Pt 10):2131-2138.
49. ZIGOVA T, SONG S, WILLING AE et al.: Human umbilical cord blood cells express neural antigens after transplantation into the developing rat brain. Cell Transplant. (2002) 11(3):265-274. This paper describes the differentiating properties of HUCB cells in a neonatal animal model.
50. CHEN J, SANBERG PR, LI Y et al.: Intravenous administration of human umbilical cord blood reduces behavioral deficits after stroke in rats. Stroke (2001) 32(11):2682-2688. This paper shows that intravenous injections of HUCB migrate to the site of injury and improve functional recovery in stroke.
51. WILLING AE, LIXIAN J, MILLIKEN M et al.: Intravenous versus intrastriatal cord blood administration in a rodent model of stroke. J. Neurosci. Res. (2003) 73(3):296-307. This is a key paper comparing HUCB routes of administration for stroke in an animal model, which also demonstrates the therapeutic potential of cord blood stem cells for stroke.
52. BORLONGAN CV, HADMAN M, YU G, LIND JG, DAVIS CD, SANBERG PR: Upregulation of CNS trophic factors by human umbilical cord transplants is essential for neuroprotection against acute stroke. Soc. Neurosci. Abstract (2003) 789.17:
53. BORLONGAN CV, EMERICH DF: Facilitation of drug entry into the CNS via transient permeation of blood brain barrier: laboratory and preliminary clinical evidence from bradykinin receptor agonist, Cereport. Brain Res. Bull. (2003) 60(3):297-306.
54. LU D, SANBERG PR, MAHMOOD A et al.: Intravenous administration of human umbilical cord blood reduces neurological deficit in the rat after traumatic brain injury. Cell Transplant. (2002) 11(3):275-281.
55. SAPORTA S, KIM JJ, WILLING AE, FU ES, DAVIS CD, SANBERG PR: Human umbilical cord blood stem cells infusion in spinal cord injury: engraftment and beneficial influence on behavior. J. Hematother. Stem Cell Res. (2003) 12(3):271-278. This paper describes an excellent potential of umbilical cord blood for treatment of spinal cord injury.
56. GARBUZOVA-DAVIS S, WILLING AE, ZIGOVA T et al.: Intravenous administration of human umbilical cord blood cells in a mouse model of amyotrophic lateral sclerosis: distribution, migration, and differentiation. J. Hematother. Stem Cell Res. (2003) 12(3):255-270. This paper demonstrates that systemic administration of HUCB cells into presymptomatic ALS mice delayed disease progression, increased lifespan, and expressed neural markers in the brain, spinal cord, and parenchyma.
57. WATSON DJ, LONGHI L, LEE EB et al.: Genetically modified NT2N human neuronal cells mediate long-term gene expression as CNS grafts in vivo and improve functional cognitive outcome following experimental traumatic brain injury. J. Neuropathol. Exp. Neurol. (2003) 62(4):368-380.
58. MITTA B, RIMANN M, EHRENGRUBER MU et al.: Advanced modular self-inactivating lentiviral expression vectors for multigene interventions in mammalian cells and in vivo transduction. Nucleic Acids Res. (2002) 30(21):e113.
59. FALK A, HOLMSTROM N, CARLEN M, CASSIDY R, LUNDBERG C, FRISEN J: Gene delivery to adult neural stem cells. Exp. Cell Res. (2002) 279(1):34-39.
60. ROSENQVIST N, HARD AF SEGERSTAD C, SAMUELSSON C, JOHANSEN J, LUNDBERG C: Activation of silenced transgene expression in neural precursor cell lines by inhibitors of histone deacetylation. J. Gene Med. (2002) 4(3):248-257.
61. BUCHET D, SERGUERA C, ZENNOU V, CHARNEAU P, MALLET J: Long-term expression of beta-glucuronidase by genetically modified human neural progenitor cells grafted into the mouse central nervous system. Mol. Cell. Neurosci. (2002) 19(3):389-401.
62. HUGHES SM, MOUSSAVI-HARAMI F, SAUTER SL, DAVIDSON BL: Viral-mediated gene transfer to mouse primary neural progenitor cells. Mol. Ther. (2002) 5(1):16-24.
63. WOODS NB, MUESSIG A, SCHMIDT M et al.: Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis. Blood (2003) 101(4):1284-1289.
64. WOODS NB, MIKKOLA H, NILSSON E, OLSSON K, TRONO D, KARLSSON S: Lentiviral-mediated gene transfer into haematopoietic stem cells. J. Intern. Med. (2001) 249(4):339-343.
65. LUTHER-WYRSCH A, COSTELLO E, THALI M et al.: Stable transduction with lentiviral vectors and amplification of immature hematopoietic progenitors from cord blood of preterm human fetuses. Hum. Gene Ther. (2001) 12(4):377-389.
66. WEISS ML, MITCHELL KE, HIX JE et al.: Transplantation of porcine umbilical cord matrix cells into the rat brain. Exp. Neurol. (2003) 182(2):288-299.
67. ENDE N, WEINSTEIN F, CHEN R, ENDE M: Human umbilical cord blood effect on sod mice (amyotrophic lateral sclerosis). Life Sci. (2000) 67(1):53-59.
68. ENDE N, CHEN R, ENDE-HAARRIS D: Human umbilical cord blood cells ameliorate Alzheimer's disease in transgenic mice. J. Med. (2001) 32(3-4):241-247.
69. ENDE N, CHEN R: Human umbilical cord blood cells ameliorate Huntington's disease in transgenic mice. J. Med. (2001) 32(3-4):231-240.
70. ENDE N, CHEN R, MACK R: NOD/LtJ type I diabetes in mice and the effect of stemn cells (Berashis) derived from human umbilical cord blood. J. Med. (2002) 33:(1-4):181-187.
71. ENDE N, CHEN R: Parkinson's disease mice and human umbilical cord blood. J. Med. (2002) 33(1-4):173-180.