Hematopoietic stem cell transplantation for the treatment of autoimmunity in type 1 diabetes

Current Stem Cell Research and Therapy

Volumn 6, Issue 1, 2011, Pages 29-37

  Locascio, Samuel A ^a,b   Spinelli, Joia ^a   Kurtz, Josef ^a,b  

^a EMMANUEL COLLEGE   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

Author keywords

Allogeneic; Chimerism; Diabetes; Hematopoietic; Tolerance

Indexed keywords

ANTIGEN PRESENTING CELL; ARTICLE; AUTOIMMUNE DISEASE; AUTOIMMUNITY; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CD4+ CD25+ T LYMPHOCYTE; CHIMERA; DENDRITIC CELL; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IMMUNE SYSTEM; INSULIN DEPENDENT DIABETES MELLITUS; MAJOR HISTOCOMPATIBILITY COMPLEX; NATURAL KILLER T CELL; NONHUMAN; PANCREAS ISLET TRANSPLANTATION; PERIPHERAL T CELL LYMPHOMA; PRIORITY JOURNAL; REGULATORY T LYMPHOCYTE; TH1 CELL; TH2 CELL; TH3 CELL;

ANIMALS; AUTOANTIGENS; AUTOIMMUNITY; DIABETES MELLITUS, EXPERIMENTAL; DIABETES MELLITUS, TYPE 1; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; T-LYMPHOCYTES; TRANSPLANTATION CONDITIONING; TRANSPLANTATION IMMUNOLOGY; TRANSPLANTATION, AUTOLOGOUS; TRANSPLANTATION, HETEROLOGOUS;

EID: 79551655241     PISSN: 1574888X     EISSN: None     Source Type: Journal    
DOI: 10.2174/157488811794480681     Document Type: Article

References (76)

1. Harbo HF, Lie BA, Sawcer S, et al. Genes in the HLA class I re gion may contribute to the HLA class II-associated genetic suscep tibility to multiple sclerosis. Tissue Antigens 2004; 63(3): 237-47.
2. Fronek Z, Timmerman LA, Alper CA, et al. Major histocompatibility complex genes and susceptibility to systemic lupus erythematosus. Arthritis Rheum 1990; 33(10): 1542-53.
3. Deighton CM, Walker DJ, Griffiths ID, Roberts DF. The contribution of HLA to rheumatoid arthritis. Clin Genet 1989; 36(3): 178-82.
4. Todd JA. Genetic control of autoimmunity in type 1 diabetes. Immunol Today 1990; 11(4): 122-9.
5. Davies JL, Kawaguchi Y, Bennett ST, et al. A genome-wide search for human type 1 diabetes susceptibility genes. Nature 1994; 371(6493): 130-6.
6. Qiao Q, Osterholm AM, He B, et al. A genome-wide scan for type 1 diabetes susceptibility genes in nuclear families with multiple affected siblings in Finland. BMC Genet 2007; 8: 84.
7. Anderson MS, Bluestone JA. The NOD mouse: a model of immune dysregulation. Annu Rev Immunol 2005; 23: 447-85.
8. Wicker LS, Clark J, Fraser HI, et al. Type 1 diabetes genes and pathways shared by humans and NOD mice. J Autoimmun 2005; 25 (Suppl): 29-33 [Epub ahead].
9. Ueda H, Howson JM, Esposito L, et al. Association of the T-cell regulatory gene CTLA4 with susceptibility to autoimmune disease. Nature 2003; 423(6939): 506-11.
10. Bergman ML, Cilio CM, Penha-Goncalves C, et al. CTLA-4-/-mice display T cell-apoptosis resistance resembling that ascribed to autoimmune-prone non-obese diabetic (NOD) mice. J Autoimmun 2001; 16(2): 105-13.
11. Colucci F, Bergman ML, Penha-Goncalves C, Cilio CM, Holmberg D. Apoptosis resistance of nonobese diabetic peripheral lymphocytes linked to the Idd5 diabetes susceptibility region. Proc Natl Acad Sci USA 1997; 94(16): 8670-4.
12. Mathis D, Benoist C. Back to central tolerance. Immunity 2004; 20(5): 509-16.
13. Vafiadis P, Bennett ST, Todd JA, et al. Insulin expression in human thymus is modulated by INS VNTR alleles at the IDDM2 locus. Nat Genet 1997; 15(3): 289-92.
14. Wicker LS, Todd JA, Peterson LB. Genetic control of autoimmune diabetes in the NOD mouse. Annu Rev Immunol 1995; 13: 179-200.
15. Bach JF. Infections and autoimmune diseases. J Autoimmun 2005; 25 Suppl: 74-80.
16. Jun HS, Yoon JW. A new look at viruses in type 1 diabetes. Diabetes Metab Res Rev 2003; 19(1): 8-31.
17. Ercolini AM, Miller SD. The role of infections in autoimmune disease. Clin Exp Immunol 2009; 155(1): 1-15.
18. Zhao ZS, Granucci F, Yeh L, Schaffer PA, Cantor H. Molecular mimicry by herpes simplex virus-type 1: autoimmune disease after viral infection. Science 1998; 279(5355): 1344-7.
19. Horwitz MS, Bradley LM, Harbertson J, Krahl T, Lee J, Sarvetnick N. Diabetes induced by Coxsackie virus: initiation by bystander damage and not molecular mimicry. Nat Med 1998; 4(7): 781-5.
20. Horwitz MS, Ilic A, Fine C, Rodriguez E, Sarvetnick N. Presented antigen from damaged pancreatic beta cells activates autoreactive T cells in virus-mediated autoimmune diabetes. J Clin Invest 2002; 109(1): 79-87.
21. Romagnani S. The increased prevalence of allergy and the hygiene hypothesis: missing immune deviation, reduced immune suppression, or both? Immunology 2004; 112(3): 352-63.
22. Singh B. Stimulation of the developing immune system can prevent autoimmunity. J Autoimmun 2000; 14(1): 15-22.
23. Gale EA. A missing link in the hygiene hypothesis? Diabetologia 2002; 45(4): 588-94.
24. Cooke A. Review series on helminths, immune modulation and the hygiene hypothesis: how might infection modulate the onset of type 1 diabetes? Immunology 2009; 126(1): 12-7.
25. Pozzilli P, Signore A, Williams AJ, Beales PE. NOD mouse colonies around the world--recent facts and figures. Immunol Today 1993; 14(5): 193-6.
26. Cooke A, Tonks P, Jones FM, et al. Infection with schistosoma mansoni prevents insulin dependent diabetes mellitus in non-obese diabetic mice. Parasite Immunol 1999; 21(4): 169-76.
27. Gottlieb PA, Hayward AR. Cytokine and immunosuppressive therapies of type 1 diabetes mellitus. Endocrinol Metab Clin North Am 2002; 31(2): 477-95.
28. Gibbons C, Sykes M. Manipulating the immune system for anti-tumor responses and transplant tolerance via mixed hematopoietic chimerism. Immunol Rev 2008; 223: 334-60.
29. Kawai T, Cosimi AB, Spitzer TR, et al. HLA-mismatched renal transplantation without maintenance immunosuppression. N Engl J Med 2008; 358(4): 353-61.
30. Sykes M, Nikolic B. Treatment of severe autoimmune disease by stem-cell transplantation. Nature 2005; 435(7042): 620-7.
31. Sykes M. Mixed chimerism and transplant tolerance. Immunity 2001; 14(4): 417-24.
32. Ildstad ST, Wren SM, Bluestone JA, Barbieri SA, Sachs DH. Characterization of mixed allogeneic chimeras. Immunocompetence, in vitro reactivity, and genetic specificity of tolerance. J Exp Med 1985; 162(1): 231-44.
33. Bala KK, Moudgil KD. Induction and maintenance of self tolerance: the role of CD4+CD25+ regulatory T cells. Arch Immunol Ther Exp (Warsz) 2006; 54(5): 307-21.
34. Lohmann T, Leslie RD, Londei M. T cell clones to epitopes of glutamic acid decarboxylase 65 raised from normal subjects and patients with insulin-dependent diabetes. J Autoimmun 1996; 9(3): 385-9.
35. Tisch R, Wang B. Dysrulation of T cell peripheral tolerance in type 1 diabetes. Adv Immunol 2008; 100: 125-49.
36. Khan A, Tomita Y, Sykes M. Thymic dependence of loss of tolerance in mixed allogeneic bone marrow chimeras after depletion of donor antigen. Peripheral mechanisms do not contribute to maintenance of tolerance. Transplantation 1996; 62(3): 380-7.
37. Tomita Y, Khan A, Sykes M. Role of intrathymic clonal deletion and peripheral anergy in transplantation tolerance induced by bone marrow transplantation in mice conditioned with a nonmyeloabla-tive regimen. J Immunol 1994; 153(3): 1087-98.
38. Manilay JO, Pearson DA, Sergio JJ, Swenson KG, Sykes M. Intra-thymic deletion of alloreactive T cells in mixed bone marrow chimeras prepared with a nonmyeloablative conditioning regimen. Transplantation 1998; 66(1): 96-102.
39. Wekerle T, Sayegh MH, Hill J, et al. Extrathymic T cell deletion and allogeneic stem cell engraftment induced with costimulatory blockade is followed by central T cell tolerance. J Exp Med 1998; 187(12): 2037-44.
40. Kurtz J, Shaffer J, Lie A, Anosova N, Benichou G, Sykes M. Mechanisms of early peripheral CD4 T cell tolerance induction by anti-CD154 monoclonal antibody and allogeneic bone marrow transplantation: evidence for anergy and deletion, but not regulatory cells. Blood 2004; 103(11): 4336-43.
41. Fehr T, Takeuchi Y, Kurtz J, Wekerle T, Sykes M. Early regulation of CD8 T cell alloreactivity by CD4+CD25- T cells in recipients of anti-CD154 antibody and allogeneic BMT is followed by rapid peripheral deletion of donor-reactive CD8+ T cells, precluding a role for sustained regulation. Eur J Immunol 2005; 35(9): 2679-90.
42. Kurtz J, Wekerle T, Sykes M. Tolerance in mixed chimerism - a role for regulatory cells? Trends Immunol 2004; 25(10): 518-23.
43. Kurtz J, Ito H, Wekerle T, Shaffer J, Sykes M. Mechanisms involved in the establishment of tolerance through costimulatory blockade and BMT: lack of requirement for CD40L-mediated signaling for tolerance or deletion of donor-reactive CD4+ cells. Am J Transplant 2001; 1(4): 339-49.
44. Kurtz J, Raval F, Vallot C, Der J, Sykes M. CTLA-4 on alloreac-tive CD4 T cells interacts with recipient CD80/86 to promote tolerance. Blood 2009; 113(15): 3475-84.
45. Schwartz RH. T cell anergy. Annu Rev Immunol 2003; 21: 305-34.
46. Wells AD, Walsh MC, Bluestone JA, Turka LA. Signaling through CD28 and CTLA-4 controls two distinct forms of T cell anergy. J Clin Invest 2001; 108(6): 895-903.
47. Sakaguchi S, Sakaguchi N, Asano M, Itoh M, Toda M. Immu-nologic self-tolerance maintained by activated T cells expressing IL-2 receptor alpha-chains (CD25). Breakdown of a single mechanism of self-tolerance causes various autoimmune diseases. J Immunol 1995; 155(3): 1151-64.
48. Takahashi T, Kuniyasu Y, Toda M, et al. Immunologic self-tolerance maintained by CD25+CD4+ naturally anergic and sup-pressive T cells: induction of autoimmune disease by breaking their anergic/suppressive state. Int Immunol 1998; 10(12): 1969-80.
49. Shevach EM. Regulatory T cells in autoimmunity. Annu Rev Immunol 2000; 18: 423-49.
50. Bayer AL, Jones M, Chirinos J, et al. Host CD4+CD25+ T cells can expand and comprise a major component of the Treg compartment after experimental HCT. Blood 2009; 113(3): 733-43.
51. Barao I, Hanash AM, Hallett W, et al. Suppression of natural killer cell-mediated bone marrow cell rejection by CD4+CD25+ regulatory T cells. Proc Natl Acad Sci USA 2006; 103(14): 5460-5.
52. Taylor PA, Noelle RJ, Blazar BR. CD4(+)CD25(+) immune regulatory cells are required for induction of tolerance to alloantigen via costimulatory blockade. J Exp Med 2001; 193(11): 1311-8.
53. Taylor PA, Lees CJ, Waldmann H, Noelle RJ, Blazar BR. Requirements for the promotion of allogeneic engraftment by anti-CD154 (anti-CD40L) monoclonal antibody under nonmyeloabla-tive conditions. Blood 2001; 98(2): 467-74.
54. van Bekkum DW. Stem cell transplantation for autoimmune disorders. Preclinical experiments. Best Pract Res Clin Haematol 2004; 17(2): 201-22.
55. Asano M, Toda M, Sakaguchi N, Sakaguchi S. Autoimmune disease as a consequence of developmental abnormality of a T cell subpopulation. J Exp Med 1996; 184(2): 387-96.
56. Roord ST, de Jager W, Boon L, et al. Autologous bone marrow transplantation in autoimmune arthritis restores immune homeosta-sis through CD4+CD25+Foxp3+ regulatory T cells. Blood 2008; 111(10): 5233-41.
57. de Kleer I, Vastert B, Klein M, et al. Autologous stem cell transplantation for autoimmunity induces immunologic self-tolerance by reprogramming autoreactive T cells and restoring the CD4+CD25+ immune regulatory network. Blood 2006; 107(4): 1696-702.
58. Van Wijmeersch B, Sprangers B, Rutgeerts O, et al. Allogeneic bone marrow transplantation in models of experimental autoimmune encephalomyelitis: evidence for a graft-versus-autoimmunity effect. Biol Blood Marrow Transplant 2007; 13(6): 627-37.
59. Wen Y, Ouyang J, Yang R, et al. Reversal of new-onset type 1 diabetes in mice by syngeneic bone marrow transplantation. Bio-chem Biophys Res Commun 2008; 374(2): 282-7.
60. Fernando MM, Stevens CR, Walsh EC, et al. Defining the role of the MHC in autoimmunity: a review and pooled analysis. PLoS Genet 2008; 4(4): e1000024.
61. Beilhack GF, Scheffold YC, Weissman IL, et al. Purified alloge-neic hematopoietic stem cell transplantation blocks diabetes patho-genesis in NOD mice. Diabetes 2003; 52(1): 59-68.
62. Ikehara S, Ohtsuki H, Good RA, et al. Prevention of type I diabetes in nonobese diabetic mice by allogenic bone marrow transplantation. Proc Natl Acad Sci USA 1985; 82(22): 7743-7.
63. Yasumizu R, Sugiura K, Iwai H, et al. Treatment of type 1 diabetes mellitus in non-obese diabetic mice by transplantation of allogeneic bone marrow and pancreatic tissue. Proc Natl Acad Sci USA 1987; 84(18): 6555-7.
64. Seung E, Iwakoshi N, Woda BA, et al. Allogeneic hematopoietic chimerism in mice treated with sublethal myeloablation and anti-CD154 antibody: absence of graft-versus-host disease, induction of skin allograft tolerance, and prevention of recurrent autoimmunity in islet-allografted NOD/Lt mice. Blood 2000; 95(6): 2175-82.
65. Kang EM, Zickler PP, Burns S, et al. Hematopoietic stem cell transplantation prevents diabetes in NOD mice but does not contribute to significant islet cell regeneration once disease is established. Exp Hematol 2005; 33(6): 699-705.
66. Serreze DV, Osborne MA, Chen YG, et al. Partial versus full allo-geneic hemopoietic chimerization is a preferential means to inhibit type 1 diabetes as the latter induces generalized immunosuppres-sion. J Immunol 2006; 177(10): 6675-84.
67. Kaufman CL, Li H, Ildstad ST. Patterns of hemopoietic reconstitu-tion in nonobese diabetic mice: dichotomy of allogeneic resistance versus competitive advantage of disease-resistant marrow. J Immunol 1997; 158(5): 2435-42.
68. Markees TG, Serreze DV, Phillips NE, et al. NOD mice have a generalized defect in their response to transplantation tolerance induction. Diabetes 1999; 48(5): 967-74.
69. Li H, Kaufman CL, Boggs SS, Johnson PC, Patrene KD, Ildstad ST. Mixed allogeneic chimerism induced by a sublethal approach prevents autoimmune diabetes and reverses insulitis in nonobese diabetic (NOD) mice. J Immunol 1996; 156(1): 380-8.
70. Nikolic B, Takeuchi Y, Leykin I, Fudaba Y, Smith RN, Sykes M. Mixed hematopoietic chimerism allows cure of autoimmune diabetes through allogeneic tolerance and reversal of autoimmunity. Diabetes 2004; 53(2): 376-83.
71. Verda L, Kim DA, Ikehara S, et al. Hematopoietic mixed chimer-ism derived from allogeneic embryonic stem cells prevents autoimmune diabetes mellitus in NOD mice. Stem Cells 2008; 26(2): 381-6.
72. Jang MH, Seth NP, Wucherpfennig KW. Ex vivo analysis of thymic CD4 T cells in nonobese diabetic mice with tetramers generated from I-A(g7)/class II-associated invariant chain peptide precursors. J Immunol 2003; 171(8): 4175-86.
73. Yang Y, Santamaria P. T-cell receptor-transgenic NOD mice: a reductionist approach to understand autoimmune diabetes. J Auto-immun 2004; 22(2): 121-9.
74. Haskins K. Pathogenic T-cell clones in autoimmune diabetes: more lessons from the NOD mouse. Adv Immunol 2005; 87: 123-62.
75. Schmidt D, Verdaguer J, Averill N, Santamaria P. A mechanism for the major histocompatibility complex-linked resistance to auto-immunity. J Exp Med 1997; 186(7): 1059-75.
76. Gonzalez A, Andre-Schmutz I, Carnaud C, Mathis D, Benoist C. Damage control, rather than unresponsiveness, effected by protective DX5+ T cells in autoimmune diabetes. Nat Immunol 2001; 2(12): 1117-25.