Toward clinical islet xenotransplantation - Are revisions to the IXA guidelines warranted?

Xenotransplantation

Volumn 20, Issue 2, 2013, Pages 68-74

  Cooper, David K C ^a   Bottino, Rita ^a,b   Satyananda, Vikas ^a   Wijkstrom, Martin ^a   Trucco, Massimo ^b  

^a UNIVERSITY OF PITTSBURGH MEDICAL CENTER   (United States)

^b CHILDREN S HOSPITAL OF PITTSBURGH   (United States)

Author keywords

diabetes mellitus; IBMIR; islets; non human primates; pig; porcine endogenous retroviruses; xenotransplantation

Indexed keywords

INSULIN;

ALLOTRANSPLANTATION; AUTOTRANSPLANTATION; CHRONIC PANCREATITIS; DIABETES MELLITUS; ENDOGENOUS RETROVIRUS; GLUCOSE BLOOD LEVEL; GLUCOSE METABOLISM; GOOD MANUFACTURING PRACTICE; HEALTH CARE COST; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; INSULIN DEPENDENT DIABETES MELLITUS; INSULIN PUMP; INSULIN TREATMENT; KIDNEY TRANSPLANTATION; NONHUMAN; NOTE; PANCREAS ISLET TRANSPLANTATION; PANCREAS RESECTION; PRIORITY JOURNAL; XENOTRANSPLANTATION;

ANIMALS; DIABETES MELLITUS, TYPE 1; DIABETES MELLITUS, TYPE 2; ENDOGENOUS RETROVIRUSES; HUMANS; INCIDENCE; ISLETS OF LANGERHANS TRANSPLANTATION; POSTOPERATIVE COMPLICATIONS; PRACTICE GUIDELINES AS TOPIC; PREVALENCE; PRIMATES; SWINE; TRANSPLANTATION, HETEROLOGOUS; WORLD HEALTH;

EID: 84873445708     PISSN: 0908665X     EISSN: 13993089     Source Type: Journal    
DOI: 10.1111/xen.12015     Document Type: Note

References (65)

1. DCCT. The effect of intensive treatment of diabetes on the development and progression of long-term complications in insulin-dependent diabetes mellitus. The Diabetes Control and Complications Trial Research Group. N Engl J Med. 1993; 329: 977-986.
2. NDIC. National Diabetes Information Claringhouse. National Diabetes Statistics. Availble at: http://diabetes.niddk.nih.gov/dm/pubs/statistics/DM- Statistics.pdf, 2011 [accessed on 29 July 2011].
3. Bassi R, Fiorina P,. Impact of islet transplantation on diabetes complications and quality of life. Curr Diab Rep 2011; 11: 355-363.
4. CITR. Collaborative Islet Transplant Registry. Sixth Annual Report. November 1, 2009. Available on http://www.citregistry.com/, 2009 [accessed 29 July 2011].
5. Bellin MD, Barton FB, Heitman A, et al. Potent induction immunotherapy promotes long-term insulin independence after islet transplantation in type 1 diabetes. Am J Transplant 2012; 12: 1576-1583.
6. Barton FB, Rickels MR, Alejandro R, et al. Improvement in outcomes of clinical islet transplantation 1999/x962010. Diabetes Care 2012; 35: 1436-1445.
7. Walsh RM, Saavedra JR, Lentz G, et al. Improved quality of life following after total pancreatectomy and autoislet transplantation for chronic pancreatitis. J Gastrointest Surg 2012; 16: 1469-1477.
8. Hering BJ, Cooper DKC, Cozzi E, et al. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes. executive summary. Xenotransplantation 2009; 16: 196-202.
9. van derWindt DJ, Bottino R, Kumar G, et al. Clinical islet xenotransplantation-how close are we? Diabetes 2012; 61: 3046-3055
10. Hering BJ, Wijkstrom M, Graham ML, et al. Prolonged diabetes reversal after intraportal xenotransplantation of wild-type porcine islets in immunosuppressed nonhuman primates. Nat Med 2006; 12: 301-303.
11. Cardona K, Korbutt GS, Milas Z, et al. Long-term survival of neonatal porcine islets in nonhuman primates by targeting costimulation pathways. Nat Med 2006; 12: 304-306.
12. Cardona K, Milas Z, Strobert E, et al. Engraftment of adult porcine islet xenografts in diabetic nonhuman primates through targeting of costimulation pathways. Am J Transplant 2007; 7: 2260-2268.
13. van derWindt DJ, Bottino R, Casu A, et al. Long-term controlled normoglycemia in diabetic non-human primates after transplantation with hCD46 transgenic porcine islets. Am J Transplant 2009; 9: 2716-2726.
14. Hecht G, Eventov-Friedman S, Rosen C, et al. Embryonic pig pancreatic tissue for the treatment of diabetes in a nonhuman primate model. Proc Natl Acad Sci U S A 2009; 106: 8659-8664.
15. Dufrane D, Goebbels RM, Gianello P,. Alginate macroencapsulation of pig islets allows correction of streptozotocin-induced diabetes in primates up to 6 months without immunosuppression. Transplantation 2010; 90: 1054-1062.
16. Thompson P, Cardona K, Russell M, et al. CD40-specific costimulation blockade enhances neonatal porcine islet survival in nonhuman primates. Am J Transplant 2011; 11: 947-957.
17. Thompson P, Badell IR, Lowe M, etal. islet xenotransplantation using Gal-deficient neonatal donors improves engraftment. Am J Transplant 2011; 11: 2593-2602.
18. van der Windt DJ, Bottino R, Casu A, Campanile N, Cooper DKC,. Rapid loss of intraportally-transplanted islets: an overview of pathophysiology and preventive strategies. Xenotransplantation 2007; 14: 288-297.
19. van derWindt DJ, Marigliano M, He J, et al. Early islet damage after direct exposure of pig islets to blood: has humoral immunity been underestimated? Cell Transplant 2012; 21: 1791-1802.
20. Casu A, Bottino R, Balamurugan AN, et al. Metabolic aspects of pig-to-monkey (Macaca fascicularis) islet transplantation: implications for translation into clinical practice. Diabetologia 2008; 51: 120-129.
21. Casu A, Echeverri GJ, Bottino R, et al. Insulin secretion and glucose metabolism in alpha 1,3-galactosyltransferase knock-out pigs compared to wild-type pigs. Xenotransplantation 2010; 17: 131-139.
22. Graham ML, Bellin MD, Papas KK, Hering BJ, Schuurman HJ,. Species incompatibilities in the pig-to-macaque islet xenotransplant model affect transplant outcome: a comparison with allotransplantation. Xenotransplantation 2011; 18: 328-342.
23. Cooper DK, Casu A,. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes-chapter 4: Pre-clinical efficacy and complication data required to justify a clinical trial. Xenotransplantation 2009b; 16: 229-238.
24. Gaber AO, Fraga D, Kotb M, Lo A, Sabek O, Latif K,. Human islet graft function in NOD-SCID mice predicts clinical response in islet transplanted recipients. Transplant Proc 2004; 36: 1108-1110.
25. Sabek OM, Fraga DW, Minoru O, McClaren JL, Gaber AO,. Assessment of human islet viability using various mouse models. Transplant Proc 2005; 37: 3415-3416.
26. Xu BY, Yu Y, Al-Abdullah IH, Kandeel F, Hering B, Wright JR Jr,. Long-term survival and function of intraportal porcine and human islet xenografts in nondiabetic nude mice. Transplant Proc 2008; 40: 584-586.
27. O'Sullivan ES, Vegas A, Anderson DG, Weir GC,. Islets transplanted in immunoisolatrion devices: a review of the progress and the challenges that remain. Endocr Rev 2011; 32: 827-844.
28. Grose S,. Critics slam Russian trial to test pig pancreas for diabetics. Nat Med 2007; 13: 390-391.
29. Sykes M, Cozzi E, D'Apice AJ, et al. Response to Valdes-Gonzalez "Clinical trial of islet xenotransplantation in Mexico". Xenotransplantation 2007; 14: 90-91.
30. Sykes M, Pierson RN 3rd, O'Connell P, et al. Reply to 'Critics slam Russian trial to test pig pancreas for diabetes'. Nat Med 2007; 13: 662-663.
31. Elliott RB,. Towards xenotransplantation of pig islets in the clinic. Curr Opin Organ Transplant 2011; 16: 195-200.
32. Sgroi A, Buhler LH, Morel P, Sykes M, Noel L,. International human xenotransplantation inventory. Transplantation 2010; 90: 597-603.
33. Bellin MD, Blondet JJ, Beilman GJ, et al. Predicting islet yield in pediatric patients undergoing pancreatectomy and autoislet transplantation for chronic pancreatitis. Pediatr Diabetes 2010; 11: 227-234.
34. Bottino R, Bertera S, Grupillo M, et al. Isolation of human islets for autologous islet transplantation in children and adolescents with chronic pancreatitis. J Transplant 2012; 642787. doi: 10.1155/2012/642787.
35. Bottino R, Balamurugan AN, Smetanka C, et al. Isolation outcome and functional characteristics of young and adult pig pancreatic islets for transplantation studies. Xenotransplantation 2007; 14: 74-82.
36. Korbutt GS,. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 Diabetes. Chapter 3: Pig islet product manufacturing and release testing. Xenotransplantation 2009; 16: 223-228.
37. Fishman JA,. Xenosis and xenotransplantation: addressing the infectious risks posed by an emerging technology. Kidney Int Suppl 1997; 58: S41-S51.
38. Fishman JA,. Infection and xenotransplantation: developing strategies to minimize risk. Ann NY Acad Sci 1998; 862: 52-66.
39. Onions D, Cooper DK, Alexander TJ, et al. An approach to the control of disease transmission in pig-to-human xenotransplantation. Xenotransplantation 2000; 7: 143-155.
40. Schuurman H-J,. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 Diabetes. Chapter 2: source pigs. Xenotransplantation 2009; 16: 215-222.
41. Bucher P, Oberholzer J, Bosco D, et al. Microbial surveillance during human pancreatic islet Isolation. Transpl Int 2005; 18: 584-589.
42. Abrahante JE, Martins K, Papas KK, Hering BJ, Schuurman HJ, Murtaugh MP,. Microbiological safety of porcine islets: comparison with source pig. Xenotransplantation 2011; 18: 88-93.
43. Mueller NJ, Barth RN, Yamamoto S, et al. Activation of cytomegalovirus in pig-to-primate organ xenotransplantation. J Virol 2002; 76: 4734-4740.
44. Mueller NJ, Kuwaki K, Knosalla C, et al. Early weaning of piglets fails to exclude porcine lymphotropic herpesvirus. Xenotransplantation 2005; 12: 59-62.
45. Mueller NJ, Ezzelarab M, Buhler L, Haeberli L, Ayares D, Cooper DK,. Monitoring of porcine and baboon cytomegalovirus infection in xenotransplantation. Xenotransplantation 2009; 16: 535-536.
46. Smith DM,. Endogenous retroviruses in xenografts. N Engl J Med 1993; 328: 142-143.
47. Patience C, Takeuchi Y, Weiss RA,. Infection of human cells by an endogenous retrovirus of pigs. Nat Med 1997; 3: 282-286.
48. Paradis K, Langford G, Long Z, et al. Search for cross-species transmission of porcine endogenous retrovirus in patients treated with living pig tissue. The XEN 111 Study Group. Science 1999; 285: 1236-1241.
49. Denner J, Schuurman HJ, Patience C,. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes-chapter 5: strategies to prevent transmission of porcine endogenous retroviruses. Xenotransplantation 2009; 16: 239-248.
50. Fishman JA, Patience C,. Xenotransplantation: infectious risk revisited. Am J Transplant 2004; 4: 1383-1390.
51. Hector RD, Meikle S, Grant L, Wilkinson RA, Fishman JA, Scobie L,. Pre-screening of miniature swine may reduce the risk of transmitting human tropic recombinant porcine endogenous retroviruses. Xenotransplantation 2007; 14: 222-226.
52. Dieckhoff B, Petersen B, Kues WA, Kurth R, Niemann H, Denner J,. Knockdown of porcine endogenous retrovirus (PERV) expression by PERV-specific shRNA in transgenic pigs. Xenotransplantation 2008; 15: 36-45.
53. Ramsoondar J, Vaught T, Ball S, et al. Production of transgenic pigs that express porcine endogenous retrovirus small interfering RNAs. Xenotransplantation 2009; 16: 164-180.
54. CDC. Centers for Disease Control and Prevention. U.S. Public Health Service Guideline on infectious disease issues in xenotransplantation. MMWR 2001; 50: 1-46. Available at: http://www.cdc.gov/mmwr/PDF/rr/rr5015.pdf, 2001 [accessed on 12 August 2011].
55. FDA. U.S. Food and Drug Administration. Guidance for industry: source animal, product, preclinical, and clinical issues concerning the use of xenotransplantation products in humans. 2003. Availble at: http://www.fda.gov/ BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/ Xenotransplantation/ucm074354.htm, 2003 [accessed on 12 August 2011].
56. Cryer PE,. The barrier of hypoglycemia in diabetes. Diabetes 2008; 57: 3169-3176.
57. Cryer PE,. Death during intensive glycemic therapy of diabetes: mechanisms and implications. Am J Med 2011; 124: 993-996.
58. O'Connell PJ,. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes. Chapter 6: patient selection for pilot clinical trials of islet xenotransplantation. Xenotransplantation 2009; 16: 249-254.
59. Zorzi D, Rastellini C, Freeman DH, Elias G, Duchini A, Cicalese L,. Increase in mortality rate of liver transplant candidates residing in specific geographic areas: analysis of UNOS Data. Am J Transplant 2012; 12: 2188-2197.
60. FDA. U.S. Food and Drug Administration. Guidance for Industry: Considerations for Allogeneic Pancreatic Islet Cell Products. Avilable at: http://www.fda.gov/BiologicsBloodVaccine/GuidanceComplianceRegulatory Information/default.htm, 2011 [accessed on 30 December 2012].
61. Tiwari JL, Schneider B, barton F, Anderson SA,. Islet cell transplantation in type 1 diabetes: an analysis of efficacy outcomes and considerations for trial designs. Am J Transplant 2012; 12: 1898-1907.
62. Cooper DK, Tseng YL, Saidman SL,. Alloantibody and xenoantibody cross-reactivity in transplantation. Transplantation 2004; 77: 1-5.
63. Echeverri GJ, McGrath K, Bottino R, et al. Endoscopic gastric submucosal transplantation of islets (ENDO-STI): technique and initial results in diabetic pigs. Am J Transplant 2009; 9: 2485-2496.
64. Cozzi E, Tallacchini M, Flanagan EB, Pierson RN 3rd, Sykes M, Vanderpool HY,. The International Xenotransplantation Association consensus statement on conditions for undertaking clinical trials of porcine islet products in type 1 diabetes-chapter 1: key ethical requirements and progress toward the definition of an international regulatory framework. Xenotransplantation 2009; 16: 203-214.
65. WHO. -WorldHealthOrganization. First WHO global consultation on regulatory requirements for xenotransplantation clinical trials: Changsha, China, 19-21 November 2008. The Changsha Communique. Xenotransplantation 2009; 16: 61-63.