497
Views
3
CrossRef citations to date
0
Altmetric
Drug Evaluation

GAD65: a prospective vaccine for treating Type 1 diabetes?

Pages 1033-1043 | Received 16 Jun 2016, Accepted 05 May 2017, Published online: 25 May 2017

References

  • 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.
  • 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.
  • Lind M, Svensson AM, Rosengren A. Glycemic control and excess mortality in type 1 diabetes. N Engl J Med. 2015 Feb 26;372(9):880–881.
  • Keenan HA 1, Sun JK, Levine J, et al. Residual insulin production and pancreatic ß-cell turnover after 50 years of diabetes: Joslin Medalist Study. Diabetes. 2010 Nov;59(11):2846–2853.
  • 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.
  • Steffes MW, Sibley S, Jackson M, et al. Beta-cell function and the development of diabetes-related complications in the diabetes control and complications trial. Diabetes Care. 2003;26:832–836.
  • Wahren J, Ekberg K, Jörnvall H. C-peptide is a bioactive peptide. Diabetologia. 2007 Mar;50(3):503–509.
  • Roep BO, Peakman M. Diabetogenic T lymphocytes in human Type 1 diabetes. Curr Opin Immunol. 2011 Dec;23(6):746–753.
  • Eizirik DL, Colli ML, Ortis F. The role of inflammation in insulitis and beta-cell loss in type 1 diabetes. Nat Rev Endocrinol. 2009 Apr;5(4):219–226.
  • Eizirik DL, Grieco FA. On the immense variety and complexity of circumstances conditioning pancreatic β-cell apoptosis in type 1 diabetes. Diabetes. 2012 Jul;61(7):1661–1663.
  • Rewers M, Ludvigsson J. Environmental risk factors for type 1 diabetes. Lancet. 2016;387:2340–2348.
  • Ludvigsson J, Heding L, Liedén G, et al. Plasmapheresis in the initial treatment of insulin-dependent diabetes mellitus in children. Br Med J (Clin Res Ed). 1983;286(6360):176–178.
  • Stiller CR, Laupacis A, Dupre J, et al. Cyclosporine for treatment of early type I diabetes: preliminaryresults. N Engl J Med. 1983;308(20):1226–1227.
  • Eisenbarth GS, Srikanta S, Jackson R, et al. Anti-thymocyte globulin and prednisone immunotherapy of recent onset type 1 diabetes mellitus. Diabetes Res. 1985;2:271–276.
  • Pocecco M, De Campo C, Cantoni L, et al. Effect of high doses intravenous IgG in newly diagnosed diabetic children. Helv Paediatr Acta. 1987;42(4):289–295.
  • 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(10):599–604.
  • Ludvigsson J. Immunological aspects on IDDM in children. Indian J Pediatr. 1989;56(Suppl 1):S7–14.
  • 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 Sep;41(9):1040–1046.
  • Allen HF, Klingensmith GJ, Jensen P, et al. Effect of bacillus Calmette–Guerin vaccination on new-onset type 1 diabetes. A randomized clinical study. Diabetes Care. 1999;22(10):1703–1707.
  • 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(2):149–154.
  • Ludvigsson J, Samuelsson U, Johansson C, et al. Treatment with antioxidants at onset of type 1 diabetes in children: a randomized, double-blind placebo-controlled study. Diabetes Metab Res Rev. 2001 Mar-Apr;17(2):131–136.
  • Herold KC, Hagopian W, Auger JA, et al. Anti-CD3 monoclonal antibody in new-onset type 1 diabetes mellitus. N Engl J Med. 2002;346:1692–1698.
  • 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(25):2598–2608.
  • Sherry N, Hagopian W, Ludvigsson J, et al. Teplizumab for treatment of type 1 diabetes (Protege study): 1-year results from a randomised, placebo-controlled trial. Lancet. 2011;378(9790):487–497.
  • Hagopian W, Ferry RJ Jr, Sherry N, et al. Teplizumab preservesC-peptide in recent-onset type 1 diabetes: two-year results from the randomized, placebo-controlled Prote´ge´ trial. Diabetes. 2013;62(11):3901–3908.
  • Ortqvist E, Björk E, Wallensteen M, et al. Temporary preservation of beta-cell function by diazoxide treatment in childhood type 1 diabetes. Diabetes Care. 2004 Sep;27(9):2191–2197.
  • Voltarelli JC, Couri CE, Stracieri A, et al. Autologous nonmyeloablative hematopoietic stem cell transplantation in newly diagnosed type 1 diabetes mellitus. JAMA. 2007;297(14):1568–1576.
  • Mastrandrea L, Yu J, Behrens T, et al. Etanercept treatment in children with new-onset type 1 diabetes: pilot randomized, placebo-controlled, double-blind study. Diabetes Care. 2009;32(7):1244–1249.
  • Pescovitz MD, Greenbaum CJ, Krause-Steinrauf H, et al. Type 1 Diabetes TrialNet anti-CD20 study group. Rituximab, B-lymphocyte depletion,and preservation of beta-cell function. N Engl J Med. 2009;361(22):2143–2152.
  • Haller MJ, Wasserfall CH, McGrail KM, et al. Autologous umbilical cord blood transfusion in very young children with type 1 diabetes. Diabetes Care. 2009 Nov;32(11):2041–2046.
  • Haller MJ, Wasserfall CH, Hulme MA, et al. Autologous umbilical cord blood transfusion in young children with type 1 diabetes fails to preserve C-peptide. Diabetes Care. 2011 Dec;34(12):2567–2569.
  • Haller MJ, Wasserfall CH, Hulme MA, et al. Autologous umbilical cord blood infusion followed by oral docosahexaenoic acid and vitamin D supplementation for C-peptide preservation in children with type 1 diabetes. Biol Blood Marrow Transplant. 2013 Jul;19(7):1126–1129.
  • Thrower SL, James L, Hall W, et al. Proinsulin peptideimmunotherapy in type 1 diabetes: report of a first-in-man phase I safety study. Clin Exp Immunol. 2009;155(2):156–165.
  • Gottlieb PA 1, Quinlan S, Krause-Steinrauf H, et al. Failure to preserve beta-cell function with mycophenolate mofetil and daclizumab combined therapy in patients with new- onset type 1 diabetes. Diabetes Care. 2010 Apr;33(4):826–832. DOI:10.2337/dc09-1349
  • Orban T, Bundy B, Becker DJ, et al. Co-stimulation modulation with abatacept in patients with recent-onset type 1 diabetes: a randomized double-masked controlled trial. Lancet. 2011;378(9789):412–419.
  • Moran A, Bundy B, Becker DJ, et al. Interleukin- 1 antagonism in type 1 diabetes of recent onset: two multicentre, randomised, double-blind, placebo-controlled trials. Lancet. 2013;381(9881):1905–1915.
  • Rigby MR, Harris KM, Pinckney A, et al. Alefacept provides sustained clinical and immunological effects in new-onset type 1 diabetes patients. J Clin Invest. 2015;125(8):3285–3296. DOI:10.1172/JCI81722
  • Haller MJ, Gitelman SE, Gottlieb PA, et al. Anti-thymocyte globulin/G-CSF treatment preserves b cell function in patients with established type 1 diabetes. J Clin Invest. 2015;125(1):448–455.
  • Schatz D, Gale EA, Atkinson MA. Why can’t we prevent type 1 diabetes?: maybe it’s time to try a different combination. Diabetes Care. 2003 Dec;26(12):3326–3328.
  • Ludvigsson J. Combination therapy for preservation of beta cell function in type 1 diabetes: new attitudes and strategies are needed! Immunol Lett. 2014 May-Jun;159(1–2):30–35.
  • Senti G, Prinz Vavricka BM, Erdmann I, et al. Intralymphatic allergen administration renders specific immunotherapy faster and safer: a randomized controlled trial. PNAS. 2008;105(46):17908–17912.
  • Boettler T, Pagni PP, Jaffe R, et al. The clinical and immunological significance of GAD-specific autoantibody and T-cell responses in type 1 diabetes. Autoimmun. 2013;44:40–48.
  • Shoda LK, Young DL, Ramanujan S, et al. A comprehensive review of interventions in the NOD mouse and implications for translation. Immunity. 2005;23(2):115–126.
  • Boettler T, von Herrath M. Type 1 diabetes vaccine development: animal models vs. humans. Hum Vaccin. 2011;7(1):19–26.
  • Staeva-Vieira T, Peakman M, von Herrath M. Translational mini-review series on type 1 diabetes: immune-based therapeutic approaches for type 1 diabetes. Clin Exp Immunol. 2007;148(1):17–31.
  • Quinn A, McInerney B, Reich EP, et al. Regulatory and effector CD4 T cells in nonobese diabetic mice recognize overlapping determinants on glutamic acid decarboxylase and use distinct V beta genes. J Immunol. 2001;166(5):2982–2991.
  • Homann D, Holz A, Bot A, et al. Autoreactive CD4+ T cells protect from autoimmune diabetes via bystander suppression using the IL-4/Stat6 pathway. Immunity. 1999;11(4):463–472.
  • Peakman M, von Herrath M. Antigen-specific immunotherapy for type 1 diabetes: maximizing the potential. Diabetes. 2010;59(9):2087–2093.
  • Tisch R, Wang B, Serreze DV. Induction of glutamic acid decarboxylase 65-specific Th2 cells and suppression of autoimmune diabetes at late stages of disease is epitope dependent. J Immunol. 1999;163(3):1178–1187.
  • Ludvigsson J. Adequate doses of autoantigen administered using the appropriate route may create tolerance and stop autoimmunity. Diabetologia. 2009;52(1):175–176.
  • Martens H, Goxe B, Geenen V. The thynmic repertoire of neuroendocrine-related self-antigens: physiological implications in T-cell life and death. Immunol Today. 1996;17:312–317.
  • Kyewski B, Klein L. A central role for central tolerance. Annu Rev Immunol. 2006;24:571–606.
  • Baekkeskov S, Nielsen JH, Marner B, et al. Autoantibodies in newly diagnosed diabetic children immunoprecipitate human pancreatic islet cell proteins. Nature. 1982;298(5870):167–169.
  • Tian J, Dang H, Nguyen AV, et al. Combined therapy with GABA and proinsulin/alum acts synergistically to restore long-term normoglycemia by modulating T-cell autoimmunity and promoting β-cell replication in newly diabetic NOD mice. Diabetes. 2014;63(9):3128–3134.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • Ludvigsson J, Faresjo M, Hjorth M, et al. GAD treatment and insulin secretion in recent-onset type 1 diabetes. N Engl J Med. 2008;359(18):1909–1920.
  • Ludvigsson J, Hjorth M, Chéramy M, et al. Extended evaluation of the safety and efficacy of GAD treatment of children and adolescents with recent-onset type 1 diabetes: a randomised controlled trial. Diabetologia. 2011 Mar;54(3):634–640.
  • Wherrett DK, Bundy B, Becker DJ, et al. Antigen-based therapy with glutamicacid decarboxylase (GAD) vaccine in patients with recent-onset type 1 diabetes: a randomised double-blind trial. Lancet. 2011 Jul 23;378(9788):319–327.
  • Ludvigsson J, Krisky D, Casas R, et al. GAD65 antigen therapy in recently diagnosed type 1 diabetes mellitus. N Engl J Med. 2012 Feb 2;366(5):433–442.
  • Ludvigsson J, Chéramy M, Axelsson S, et al. GAD-treatment of children and adolescents with recent-onset type 1 diabetes preserves residual insulin secretion after 30 months. For the Clinical GAD-Study Group in Sweden. Diabetes Metab Res Rev.2014 Jul;30(5):405–414. doi: 10.1002/dmrr.2503.
  • Ludvigsson J, Wahlberg J, Casas R. Intralymphatic injection of autoantigen in type 1 diabetes. N Engl J Med. 2017 Feb 16; 376(7):697–699.
  • Greenbaum CJ 1, Mandrup-Poulsen T, McGee PF, et al. Mixed-meal tolerance test versus glucagon stimulation test for the assessment of beta-cell function in therapeutic trials in type diabetes. Diabetes Care. 2008 Oct;31(10):1966–1971.
  • Tavira B, Cheramy M, Axelsson S, et al. Effect of simultaneous vaccination with H1N1 and GAD-alum on GAD65-induced immune response. Diabetologia. 2017 Mar 29. doi: 10.1007/s00125-017-4263-x.
  • Ludvigsson J. The clinical potential of low-level C-peptide secretion. Expert Rev Mol Diagn. 2016 Sep;16(9):933–940.
  • Beam CA, MacCallum C, Herold KC et al. GAD vaccine reduces insulin loss in recently diagnosed type 1 diabetes: findings from a Bayesian meta-analysis. Diabetologia. 2017 Jan;60(1):43–49. Epub 2016 Oct 4.
  • Axelsson S, Hjorth M, Åkerman L, et al. Early induction of Th2 responses in type 1 diabetic children treated with alum-formulated GAD65. Diabetes Met Res Rev. 2010 Oct;26(7):559–568.
  • Skoglund C, Chéramy M, Casas R, et al. GAD65Ab epitope pattern and anti-idiotype antibodies after immune modulation with alum formulated GAD65 in children with type 1 diabetes. Pediatr Diabetes. 2012 May;13(3):244–250.
  • Chéramy M, Skoglund C, Johansson I, et al. GAD-alum treatment in patients with type 1 diabetes and the subsequent effect on GADA IgG subclass distribution, GAD65 enzyme activity and humoral response. Clinical Immunol, Clin Immunol. 2010 Oct;137(1):31–40.
  • Axelsson S, Chéramy M, Hjorth M, et al. Long-lasting immune responses 4 years after GAD-alum treatment in children with type 1 diabetes. PLoS One. 2011;6(12):e29008.
  • Hjorth M, Axelsson S, Rydén A, et al. GAD-alum treatment induces GAD65-specific CD4+CD25highFOXP3+ cells in type 1 diabetic patients. Clin Immunol. 2011 Jan;138(1):117–126.
  • Pihl M, Barcenilla H, Axelsson S, et al. GAD-specific T cells are induced by GAD-alum treatment in type-1 diabetes patients. Clin Immunol. 2017;176:114–121.
  • Axelsson S, Chéramy M, Akerman L, et al. Cellular and humoral immune responses in type 1 diabetic patients participating in a phase III GAD-alum intervention trial. Diabetes Care. 2013;36(11):3418–3424.
  • Piemonti L, Monti P, Sironi M, et al. Vitamin D3 affects differentiation, maturation, and function of human monocyte-derived dendritic cells. J Immunol. 2000;164:4443–4451.
  • Mathieu C, Gysemans C, Giulietti A, et al. Vitamin D and diabetes. Diabetologia. 2005;48:1247–1257.
  • Teegarden D, Donkin SS. Vitamin D: emerging new roles in insulin sensitivity. Nutr Res Rev. 2009;22:82–92.
  • Mandrup-Poulsen T, Zumsteg U, Reimers J, et al. Involvement of interleukin 1 and interleukin 1 antagonist in pancreatic β-cell destruction in insulin-dependent diabetes mellitus. Cytokine. 1993;5:185–191.
  • Argiles JM, Lopez-Soriano J. Lopez-Soriano FJ. Cytokines and diabetes: the final step? Involvement of TNF-α in both type I and II diabetes mellitus. Horm Metab Res. 1994;26:447–449.
  • Von Beust BR, Johansen P, Smith KA, et al. Improving the therapeutic index for CpG oligodeoxynucleotides by intralymphatic administration. Eur J Immunol. 2005;35:1869–1876.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.