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First update of 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.

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Cooper, David K C Bottino, Rita Gianello, Pierre [UCL] Graham, Melanie Hawthorne, Wayne J Weber, Collin

In 2009, the International Xenotransplantation Association (IXA) published a consensus document that provided guidelines and "recommendations" (not regulations) for those contemplating clinical trials of porcine islet transplantation. These guidelines included the IXA's opinion on what constituted "rigorous pre-clinical studies using the most relevant animal models" and were based on "non-human primate testing." We now report our discussion following a careful review of the 2009 guidelines as they relate to pre-clinical testing. In summary, we do not believe there is a need to greatly modify the conclusions and recommendations of the original consensus document. Pre-clinical studies should be sufficiently rigorous to provide optimism that a clinical trial is likely to be safe and has a realistic chance of success, but need not be so demanding that success might only be achieved by very prolonged experimentation, as this would not be in the interests of patients whose quality of life might benefit immensely from a successful islet xenotransplant. We believe these guidelines will be of benefit to both investigators planning a clinical trial and to institutions and regulatory authorities considering a proposal for a clinical trial. In addition, we suggest consideration should be given to establishing an IXA Clinical Trial Advisory Committee that would be available to advise (but not regulate) researchers considering initiating a clinical trial of xenotransplantation.

Document type Article de périodique (Journal article) – Article de recherche
Access type Accès restreint
Publication date 2016
Language Anglais
Journal information "Xenotransplantation" - Vol. 23, no. 1, p. 46-52 (2016)
Peer reviewed yes
Publisher Wiley-Blackwell Publishing, Inc. ((United States) Malden, MA)
issn 0908-665X
e-issn 1399-3089
Publication status Publié
Affiliations UCL - SSS/IREC/CHEX - Pôle de chirgurgie expérimentale et transplantation
UCL - (SLuc) Service de chirurgie et transplantation abdominale
Keywords Diabetes melitus ; Islets ; non-human primates ; Pig ; Xenotransplantation
Links
  1. World Health Organization The Changsha Communique First WHO Global Consultation on Regulatory Requirements for Xenotransplantation Clinical Trials Changsha, China, November 19-21 2008
  2. Cooper David K. C., Global Consultation on Regulatory Requirements for Xenotransplantation in Clinical Trials, 10.1111/j.1399-3089.2009.00518.x
  3. Hering Bernhard J., Cooper David K. C., Cozzi Emanuele, Schuurman Henk-Jan, Korbutt Gregory S., Denner Joachim, O’Connell Philip J., Vanderpool Harold Y., Pierson III Richard N., Executive summary, 10.1111/j.1399-3089.2009.00547.x
  4. Cooper David K. C., Casu Anna, Chapter 4: Pre-clinical efficacy and complication data required to justify a clinical trial, 10.1111/j.1399-3089.2009.00543.x
  5. Cooper David K.C., Bottino Rita, Satyananda Vikas, Wijkstrom Martin, Trucco Massimo, Toward clinical islet xenotransplantation - are revisions to the IXA guidelines warranted?, 10.1111/xen.12015
  6. Samy Kannan P., Martin Benjamin M., Turgeon Nicole A., Kirk Allan D., Islet cell xenotransplantation: a serious look toward the clinic, 10.1111/xen.12095
  7. van der Windt D. J., Bottino R., Kumar G., Wijkstrom M., Hara H., Ezzelarab M., Ekser B., Phelps C., Murase N., Casu A., Ayares D., Lakkis F. G., Trucco M., Cooper D. K. C., Clinical Islet Xenotransplantation: How Close Are We?, 10.2337/db12-0033
  8. Hawthorne W. J., Salvaris E. J., Phillips P., Hawkes J., Liuwantara D., Burns H., Barlow H., Stewart A. B., Peirce S. B., Hu M., Lew A. M., Robson S. C., Nottle M. B., D'Apice A. J. F., O'Connell P. J., Cowan P. J., Control of IBMIR in Neonatal Porcine Islet Xenotransplantation in Baboons : Genetically Manipulated NICC Stop Thrombosis, 10.1111/ajt.12722
  9. Casu A., Bottino R., Balamurugan A. N., Hara H., van der Windt D. J., Campanile N., Smetanka C., Cooper D. K. C., Trucco M., Metabolic aspects of pig-to-monkey (Macaca fascicularis) islet transplantation: implications for translation into clinical practice, 10.1007/s00125-007-0844-4
  10. Casu Anna, Echeverri Gabriel J., Bottino Rita, van der Windt Dirk J., He Jing, Ekser Burcin, Ball Suyapa, Ayares David, Cooper David K. C., Insulin secretion and glucose metabolism in alpha 1,3-galactosyltransferase knock-out pigs compared to wild-type pigs, 10.1111/j.1399-3089.2010.00572.x
  11. Graham Melanie L., Bellin Melena D., Papas Klearchos K., Hering Bernhard J., Schuurman Henk-Jan, Species incompatibilities in the pig-to-macaque islet xenotransplant model affect transplant outcome: a comparison with allotransplantation : Pig-to-NHP islet species incompatibilities, 10.1111/j.1399-3089.2011.00676.x
  12. Graham Melanie L., Schuurman Henk-Jan, The usefulness and limitations of the diabetic macaque model in evaluating long-term porcine islet xenograft survival : Modeling islet xenotransplantation in NHPs, 10.1111/xen.12012
  13. Wijkstrom Martin, Bottino Rita, Cooper David K.C., Limitations of the pig-to-non-human primate islet transplantation model, 10.1111/xen.12017
  14. Mueller Kate R, Balamurugan A N, Cline Gary W, Pongratz Rebecca L, Hooper Rebecca L, Weegman Bradley P, Kitzmann Jennifer P, Taylor Michael J, Graham Melanie L, Schuurman Henk-Jan, Papas Klearchos K, Differences in glucose-stimulated insulin secretionin vitroof islets from human, nonhuman primate, and porcine origin, 10.1111/xen.12022
  15. Nagaraju Santosh, Bertera Suzanne, Funair Amber, Wijkstrom Martin, Trucco Massimo, Cooper David KC, Bottino Rita, Streptozotocin-associated lymphopenia in cynomolgus monkeys, 10.4161/19382014.2014.944441
  16. Luo B., Chan W. F. N., Lord S. J., Nanji S. A., Rajotte R. V., Shapiro A. M. J., Anderson C. C., Diabetes Induces Rapid Suppression of Adaptive Immunity Followed by Homeostatic T-cell Proliferation, 10.1111/j.1365-3083.2006.01863.x
  17. Bennet W., Sundberg B., Groth C. G., Brendel M. D., Brandhorst D., Brandhorst H., Bretzel R. G., Elgue G., Larsson R., Nilsson B., Korsgren O., Incompatibility between human blood and isolated islets of Langerhans: a finding with implications for clinical intraportal islet transplantation?, 10.2337/diabetes.48.10.1907
  18. Bennet William, Sundberg Berit, Lundgren Torbj??rn, Tibell Annika, Groth Carl-Gustav, Richards Andrew, White David J., Elgue Graciela, Larsson Rolf, Nilsson Bo, Korsgren Olle, DAMAGE TO PORCINE ISLETS OF LANGERHANS AFTER EXPOSURE TO HUMAN BLOOD IN VITRO, OR AFTER INTRAPORTAL TRANSPLANTATION TO CYNOMOLOGUS MONKEYS : Protective Effects of sCR1 and Heparin1, 10.1097/00007890-200003150-00007
  19. van der Windt Dirk J., Bottino Rita, Casu Anna, Campanile Nathalie, Cooper David K.C., Rapid loss of intraportally transplanted islets: an overview of pathophysiology and preventive strategies, 10.1111/j.1399-3089.2007.00419.x
  20. Dufrane Denis, Goebbels Rose-Marie, Gianello Pierre, Alginate Macroencapsulation of Pig Islets Allows Correction of Streptozotocin-Induced Diabetes in Primates up to 6 Months Without Immunosuppression : , 10.1097/tp.0b013e3181f6e267
  21. Vériter Sophie, Gianello Pierre, Igarashi Yasuhiro, Beaurin Gwen, Ghyselinck Audrey, Aouassar Najima, Jordan Bénédicte, Gallez Bernard, Dufrane Denis, Improvement of Subcutaneous Bioartificial Pancreas Vascularization and Function by Coencapsulation of Pig Islets and Mesenchymal Stem Cells in Primates, 10.3727/096368913x663550
  22. Sgroi Antonino, Bühler Leo H., Morel Philippe, Sykes Megan, Noel Luc, International Human Xenotransplantation Inventory : , 10.1097/tp.0b013e3181eb2e8c
  23. Elliott Robert B, Towards xenotransplantation of pig islets in the clinic : , 10.1097/mot.0b013e3283449dec
  24. Wynyard Shaun, Nathu Divya, Garkavenko Olga, Denner Joachim, Elliott Robert, Microbiological safety of the first clinical pig islet xenotransplantation trial in New Zealand, 10.1111/xen.12102
  25. U.S. Department of Health and Human Services, Food and Drug Administration (CBER) Guidance for industry: source animal, product, preclinical and clinical issues concerning the use of xenotransplantation products in humans http://www.fda.gov/cber/gdins/clinxeno.htm 2003
  26. Cardona Kenneth, Korbutt Gregory S, Milas Zvonimir, Lyon James, Cano Jose, Jiang Wanhong, Bello-Laborn Hameeda, Hacquoil Brad, Strobert Elizabeth, Gangappa Shivaprakash, Weber Collin J, Pearson Thomas C, Rajotte Ray V, Larsen Christian P, Long-term survival of neonatal porcine islets in nonhuman primates by targeting costimulation pathways, 10.1038/nm1375
  27. Hecht G., Eventov-Friedman S., Rosen C., Shezen E., Tchorsh D., Aronovich A., Freud E., Golan H., El-Hasid R., Katchman H., Hering B. J., Zung A., Kra-Oz Z., Shaked-Mishan P., Yusim A., Shtabsky A., Idelevitch P., Tobar A., Harmelin A., Bachar-Lustig E., Reisner Y., Embryonic pig pancreatic tissue for the treatment of diabetes in a nonhuman primate model, 10.1073/pnas.0812253106
Bibliographic reference Cooper, David K C ; Bottino, Rita ; Gianello, Pierre ; Graham, Melanie ; Hawthorne, Wayne J ; et. al. First update of 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.. In: Xenotransplantation, Vol. 23, no. 1, p. 46-52 (2016)
Permanent URL http://hdl.handle.net/2078.1/172730