GAD-alum (Diamyd) - A new concept for preservation of residual insulin secretion

Expert Opinion on Biological Therapy

Volumn 10, Issue 5, 2010, Pages 787-799

  Ludvigsson, Johnny ^a  

^a LINKÖPING UNIVERSITY   (Sweden)

Author keywords

Diamyd; GAD; GADA; Immune intervention; Prevention; Type 1 diabetes

Indexed keywords

DIAMYD; GLUTAMATE DECARBOXYLASE 65; PLACEBO; UNCLASSIFIED DRUG;

CLINICAL TRIAL; DRUG EFFICACY; DRUG SAFETY; HUMAN; IMMUNE RESPONSE; INJECTION SITE REACTION; INSULIN DEPENDENT DIABETES MELLITUS; INSULIN RELEASE; REVIEW; RHINOPHARYNGITIS;

ADJUVANTS, IMMUNOLOGIC; ADOLESCENT; ADULT; AGED; ALUM COMPOUNDS; ANIMALS; CHILD; CHILD, PRESCHOOL; DIABETES MELLITUS, TYPE 1; EVIDENCE-BASED MEDICINE; GLUTAMATE DECARBOXYLASE; HUMANS; IMMUNE TOLERANCE; IMMUNOTHERAPY; INSULIN; INSULIN-SECRETING CELLS; MIDDLE AGED; RECOMBINANT PROTEINS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 77951102828     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712591003742920     Document Type: Review

References (68)

1. Ludvigsson J, Heding L, Lieden G, et al. Plasmapheresis in the initial treatment of insulin-dependent diabetes mellitus in children. Br Med J (Clin Res Ed) 1983;286:176-178
2. Baekkeskov S, Nielsen JH, Marner B, et al. Autoantibodies in newly diagnosed diabetic children immunoprecipitate human pancreatic islet cell proteins. Nature 1982;10:167-169
3. Baekkeskov S, Aanstoot HJ, Christgau S, et al. Identification of the 64K autoantigen in insulin-dependent diabetes as the GABA-synthesizing enzyme glutamic acid decarboxylase. Nature 1990;347:151-156
4. Christgau S, Schierbeck H, Aanstoot HJ, et al. Pancreatic beta cells express two autoantigenic forms of glutamic acid decarboxylase, a 65-kDa hydrophilic form and a 64-kDa amphiphilic form which can be both membrane-bound and soluble. J Biol Chem 1991;266:21257-21264
5. Mehta AK, Ticku MK. An update on GABAA receptors. Brain Res Brain Res Rev 1999;29:196-217
6. Barker JF, Behar T, Li YX, et al. GABA-ergic cells and signals in CNS development. Perspect Dev Neurobiol 1998;5:305-322
7. Barker JF. GABA as a neurotransmitter and neurogenic signal. Encyclopedia of life sciences. John Wiley & Sons, Chichester, West Sussex; 2004. p. 1-9: published online 03 May 2005, doi: 10.1038/npg.els.0000121
8. Murinson BB. Stiff-person syndrome. Neurologist 2004;10:131-137
9. Levy LM, Dalakas MC, Floeter MK. The stiff-person syndrome: an autoimmune disorder affecting neurotransmission of gamma-aminobutyric acid. Ann Intern Med 1999;131:522-530
10. Lohmann T, Hawa M, Leslie RD, et al. Immune reactivity to glutamic acid decarboxylase 65 in stiffman syndrome and type 1 diabetes mellitus. Lancet 2000;356:31-35
11. Liu J, Wolfe D, Hao S, et al. Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain. Mol Ther 2004;10:57-66
12. Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 2007;369:2097-2105
13. Okada Y, Taniguchi H, Schimada C. High concentration of GABA and high glutamate decarboxylase activity in rat pancreatic islets and human insulinoma. Science 1976;194:620-622
14. Bjork E, Kampe O, Andersson A, Karlsson FA. Expression of the 64 kDa/glutamic acid decarboxylase rat islet cell autoantigen is influenced by the rate of insulin secretion. Diabetologia 1992;35:490-493
15. Bjork E, Kämpe O, Karlsson FA, et al. Glucose regulation of the autoantigen GAD65 in human pancreatic islets. J Clin Endocrinol Metab 1992;75:1574-1576
16. Kampe O, Andersson A, Bjork E, et al. High-glucose stimulation of 64,000-Mr islet cell autoantigen expression. Diabetes 1989;38:1326-1328
17. Rorsman P, Berggren PO, Bokvist K, et al. Glucose-inhibition of glucagon secretion involves activation of GABAA-receptor chloride channels. Nature 1989;341:233-236
18. Gu XH, Kurose T, Kato S, et al. Suppressive effect of GABA on insulin secretion from the pancreatic beta-cells in the rat. Life Sci 1993;52:687-694
19. Shi Y, Kanaani J, Menard-Rose V, et al. Increased expression of GAD65 and GABA in pancreatic beta-cells impairs first-phase insulin secretion. Am J Physiol Endocrinol Metab 2000;279:E684-694
20. Fenalti G, Rowley MJ. GAD65 as a prototypic autoantigen. J Autoimmun 2008;31:228-232
21. Schloot NC, Roep BO, Wegmann DR, et al. T-cell reactivity to GAD65 peptide sequences shared with coxsackie virus protein in recent-onset IDDM, post-onset IDDM patients and control subjects. Diabetologia 1997;40:332-338
22. Karlsson MG, Ludvigsson J. Peptide from glutamic acid decarboxylase similar to coxsackie B virus stimulates IFN-gamma mRNA expression in Th1-like lymphocytes from children with recent-onset insulin-dependent diabetes mellitus. Acta Diabetol 1998;35:137-144
23. Skarsvik S, Puranen J, Honkanen J, et al. Decreased in vitro type 1 immune response against coxsackie virus B4 in children with type 1 diabetes. Diabetes 2006;55:996-1003
24. Kaufman DL, Erlander MG, Clare-Salzler M, et al. Autoimmunity to two forms of glutamate decarboxylase in insulin-dependent diabetes mellitus. J Clin Invest 1992;89:283-292
25. Atkinson MA, Kaufman DL, Campbell L, et al. Response of peripheral-blood mononuclear cells to glutamate decarboxylase in insulin-dependent diabetes. Lancet 1992;339:458-459
26. Zimmet P. Antibodies to glutamic acid decarboxylase in the prediction of insulin dependency. Diabetes Res Clin Pract 1996;34 (Suppl):S125-131
27. Bingley PJ, Bonifacio E, Williams AJ, et al. Prediction of IDDM in the general population: strategies based on combinations of autoantibody markers. Diabetes 1997;46:1701-1710
28. Lernmark A. Controlling the controls: GAD65 autoreactive T cells in type 1 diabetes. J Clin Invest 2002;109:869-870
29. Bach JF. Autoimmunity and type I diabetes. Trends Endocrinol Metab 1997;8:71-74
30. Bach JF. New concepts of the etiopathogenesis and treatment of insulin-dependent diabetes mellitus. Clin Rev Allergy Immunol 2000;19:217-225
31. Atkinson MA, Eisenbarth GS. Type 1 diabetes: new perspectives on disease pathogenesis and treatment. Lancet 2001;358:221-229
32. Roep BO. The role of T-cells in the pathogenesis of Type 1 diabetes: from cause to cure. Diabetologia 2003;46:305-321
33. The Diabetes Control and Complications Trial Research Group. The effect of intensive treatment of diabetes on the development and progression of long-term complications in insulin-dependent diabetes mellitus. N Engl J Med 1993;329:977-986
34. Bojestig M, Arnqvist HJ, Hermansson G, et al. Declining incidence of nephropathy in insulin-dependent diabetes mellitus. N Engl J Med 1994;330:15-18
35. Nordfeldt S, Ludvigsson J. Severe hypoglycemia in children with IDDM. A prospective population study, 1992-1994. Diabetes Care 1997;20:497-503
36. Steffes MW, Sibley S, Jackson M, Thomas W. beta-cell function and the development of diabetes-related complications in the diabetes control and complications trial. Diabetes Care 2003;26:832-836
37. Ludvigsson J, Samuelsson U, Ernerudh J, et al. Photopheresis at onset of type 1 diabetes: a randomised, double blind, placebo controlled trial. Arch Dis Child 2001;85:149-154
38. Heinze E. Immunoglobulins in children with autoimmune diabetes mellitus. Clin Exp Rheumatol 1996;14 (Suppl 15):S99-102
39. Dupré J, Stiller CR, Gent M, et al. Clinical trials of cyclosporin in IDDM. Diabetes Care 1988;11 (Suppl 1):37-44
40. Silverstein J, Maclaren N, Riley W, et al. Immunosuppression with azathioprine and prednisone in recent-onset insulin-dependent diabetes mellitus. N Engl J Med 1988;319:599-604
41. Coutant R, Landais P, Rosilio M, et al. Low dose linomide in Type I juvenile diabetes of recent onset: a randomised placebo-controlled double blind trial. Diabetologia 1998;41:1040-1046
42. Rother KI, Brown RJ, Morales MM, et al. Effect of ingested interferon-alpha on beta-cell function in children with new-onset type 1 diabetes. Diabetes Care 2009;32(7):1250-1255
43. Pescovitz MD, Greenbaum CJ, Krause-Steinrauf H, et al. Rituximab, B-lymphocyte depletion, and preservation of beta-cell function. N Engl J Med 2009;361(22):2143-2152
44. Herold KC, Gitelman SE, Masharani U, et al. A single course of anti-CD3 monoclonal antibody hOKT3gamma1 (Ala-Ala) results in improvement in C-peptide responses and clinical parameters for at least 2 years after onset of type 1 diabetes. Diabetes 2005;54:1763-1769
45. Keymeulen B, Vandemeulebroucke E, Ziegler AG, et al. Insulin needs after CD3-antibody therapy in new-onset type 1 diabetes. N Engl J Med 2005;352:2598-2608 (Pubitemid 41007846)
46. Skyler JS, Krischer JP, Wolfsdorf J, et al. Effects of oral insulin in relatives of patients with type 1 diabetes: the Diabetes Prevention Trial--Type 1. Diabetes Care 2005;28:1068-1076
47. Nänto-Salonen K, Kupila A, Simell S, et al. Nasal insulin to prevent type 1 diabetes in children with HLA genotypes and autoantibodies conferring increased risk of disease: a double-blind, randomised controlled trial. Lancet 2008;372(9651):1746-1755
48. Raz I, Elias D, Avron A, et al. beta-cell function in new-onset type 1 diabetes and immunomodulation with a heat-shock protein peptide (DiaPep277): a randomised, double-blind, phase II trial. Lancet 2001;358:1749-1753
49. Schloot NC, Meierhoff G, Lengyel C, et al. Effect of heat shock protein peptide DiaPep277 on beta-cell function in paediatric and adult patients with recent-onset diabetes mellitus type 1: two prospective, randomized, double-blind phase II trials. Diabetes Metab Res Rev 2007;23:276-285
50. Ludvigsson J. Adequate doses of autoantigen administered using the appropriate route may create tolerance and stop autoimmunity. Diabetologia 2009;52(1):175-176
51. Kaufman DL, Clare-Salzler M, Tian J, et al. Spontaneous loss of T-cell tolerance to glutamic acid decarboxylase in murine insulin-dependent diabetes. Nature 1993;366:69-72
52. Tisch R, Yang XD, Singer SM, et al. Immune response to glutamic acid decarboxylase correlates with insulitis in non-obese diabetic mice. Nature 1993;366:72-75
53. Petersen JS, Karlsen AE, Markholst H, et al. Neonatal tolerization with glutamic acid decarboxylase but not with bovine serum albumin delays the onset of diabetes in NOD mice. Diabetes 1994;43:1478-1484
54. Pleau JM, Fernandez-Saravia F, Esling A, et al. Prevention of autoimmune diabetes in nonobese diabetic female mice by treatment with recombinant glutamic acid decarboxylase (GAD 65). Clin Immunol Immunopathol 1995;76:90-95
55. Tian J, Atkinson MA, Clare-Salzler M, et al. Nasal administration of glutamate decarboxylase (GAD65) peptides induces Th2 responses and prevents murine insulin-dependent diabetes. J Exp Med 1996;183:1561-1567
56. Tian J, Clare-Salzler M, Herschenfeld A, et al. Modulating autoimmune responses to GAD inhibits disease progression and prolongs islet graft survival in diabetes-prone mice. Nat Med 1996;2:1348-1353
57. Tisch R, Liblau RS, Yang XD, et al. Induction of GAD65-specific regulatory T-cells inhibits ongoing autoimmune diabetes in nonobese diabetic mice. Diabetes 1998;47:894-899
58. Plesner A, Worsaae A, Dyrberg T, et al. Immunization of diabetes-prone or non-diabetes-prone mice with GAD65 does not induce diabetes or islet cell pathology. J Autoimmun 1998;11:335-341
59. Yoon JW, Yoon CS, Lim HW, et al. Control of autoimmune diabetes in NOD mice by GAD expression or suppression in beta cells. Science 1999;284:1183-1187
60. Tisch R, Wang B, Weaver DJ, et al. Antigen-specific mediated suppression of beta cell autoimmunity by plasmid DNA vaccination. J Immunol 2001;166:2122-2132
61. Jun HS, Chung YH, Han J, et al. Prevention of autoimmune diabetes by immunogene therapy using recombinant vaccinia virus expressing glutamic acid decarboxylase. Diabetologia 2002;45:668-676
62. Agardh CD, Cilio CM, Lethagen A, et al. Clinical evidence for the safety of GAD65 immunomodulation in adult-onset autoimmune diabetes. J Diabetes Complications 2005;19:238-246
63. Agardh C-D, Lynch K, Palmér M, et al. GAD65 vaccination significantly reduces insulin dependence at five years follow-up in a dose escalating study in adult-onset autoimmune diabetes patients. Diabetologia 2008;51 (Suppl 1):S230
64. Ludvigsson J, Faresjo M, Hedman M, et al. GAD65 treatment and residual insulin secretion in recent onset type 1 diabetes. N Engl J Med 2008;30:1909-1920
65. Greenbaum CJ, Mandrup-Poulsen T, McGee PF, et al. Mixed meal tolerance test versus the glucagon stimulation test for the assessment of beta-cell function in therapeutic trials in type 1 diabetes. Diabetes Care 2008;31:1966-1971
66. Madsbad S, Alberti KG, Binder C, et al. Role of residual insulin secretion in protecting against ketoacidosis in insulin-dependent diabetes. Br Med J 1979;2:1257-1259
67. The Diabetes Control and Complications Trial Research Group. Effect of intensive therapy on residual beta-cell function in patients with type 1 diabetes in the diabetes control and complications trial. A randomized, controlled trial. Ann Intern Med 1998;128:517-523
68. Wahren J, Ekberg K, Jornvall H. C-peptide is a bioactive peptide. Diabetologia 2007;50:503-509