Preclinical toxicity evaluation of erythrocyte-encapsulated thymidine phosphorylase in BALB/c mice and beagle dogs: An enzyme-replacement therapy for mitochondrial neurogastrointestinal encephalomyopathy

Toxicological Sciences

Volumn 131, Issue 1, 2013, Pages 311-324

  Levene, Michelle ^a   Coleman, David G ^b   Kilpatrick, Hugh C ^b   Fairbanks, Lynette D ^c   Gangadharan, Babunilayam ^b   Gasson, Charlotte ^b   Bax, Bridget E ^a  

^a ST GEORGE'S UNIVERSITY OF LONDON   (United Kingdom)

^b HUNTINGDON LIFE SCIENCES   (United Kingdom)

^c GSTS Pathology   (United Kingdom)

Author keywords

Enzyme replacement; Erythrocyte carriers; Mitochondrial neurogastrointestinal encephalomyopathy; Thymidine phosphorylase; Toxicity

Indexed keywords

RECOMBINANT THYMIDINE PHOSPHORYLASE; THYMIDINE PHOSPHORYLASE; UNCLASSIFIED DRUG;

ADVERSE OUTCOME; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CLINICAL FEATURE; CONTROLLED STUDY; DOSE RESPONSE; DRUG SAFETY; DRUG SCREENING; ENZYME REPLACEMENT; ERYTHROCYTE; FEMALE; IMMUNE RESPONSE; IMMUNITY; LUNG EMBOLISM; MALE; MNGIE SYNDROME; MOUSE; NONHUMAN; ORGAN WEIGHT; SPLENOMEGALY;

ANIMALS; BLOOD TRANSFUSION, AUTOLOGOUS; DOGS; DRUG CARRIERS; DRUG EVALUATION, PRECLINICAL; ENZYME REPLACEMENT THERAPY; ERYTHROCYTE TRANSFUSION; ERYTHROCYTES; INTESTINAL PSEUDO-OBSTRUCTION; MICE; MICE, INBRED BALB C; MITOCHONDRIAL ENCEPHALOMYOPATHIES; THYMIDINE PHOSPHORYLASE; TOXICITY TESTS;

ANIMALIA; CANIS FAMILIARIS; MUS;

EID: 84872367581     PISSN: 10966080     EISSN: 10960929     Source Type: Journal    
DOI: 10.1093/toxsci/kfs278     Document Type: Article

References (25)

1. Angervall, L., and Carlström, E. (1963). Theoretical criteria for the use of relative organ weights and similar ratios in biology. J. Theor. Biol. 4, 254-259.
2. Bartlett, M. S. (1937). Properties of sufficiency and statistical tests. P. Roy. Soc. Lond. A Mat. 160, 268-282.
3. Barton, G. J., Ponting, C. P., Spraggon, G., Finnis, C., and Sleep, D. (1992). Human platelet-derived endothelial cell growth factor is homologous to Escherichia coli thymidine phosphorylase. Protein Sci. 1, 688-690.
4. Bax, B. E., Bain, M. D., Fairbanks, L. D., Webster, A. D., and Chalmers, R. A. (2000). In vitro and in vivo studies with human carrier erythrocytes loaded with polyethylene glycol-conjugated and native adenosine deaminase. Br. J. Haematol. 109, 549-554.
5. Bax, B. E., Bain, M. D., Fairbanks, L. D., Webster, A. D., Ind, P. W., Hershfield, M. S., and Chalmers, R. A. (2007). A 9-yr evaluation of carrier erythrocyte encapsulated adenosine deaminase (ADA) therapy in a patient with adulttype ADA deficiency. Eur. J. Haematol. 79, 338-348.
6. Bax, B. E., Bain, M. D., Talbot, P. J., Parker-Williams, E. J., and Chalmers, R. A. (1999). Survival of human carrier erythrocytes in vivo. Clin. Sci. 96, 171-178.
7. Brito, L. A., and Singh, M. (2011). Acceptable levels of endotoxin in vaccine formulations during preclinical research. J. Pharm. Sci. 100, 34-37.
8. Chalmers, R. A. (1985). Comparison and potential of hypo-osmotic and iso-osmotic erythrocyte ghosts and carrier erythrocytes as drug and enzyme carriers. Bibl. Haematol. 51, 15-24.
9. Fisher, R. A. (1973). Statistical Methods for Research Workers, 14th ed., p. 96. Hafner Publishing Company, New York, NY.
10. Garone, C., Tadesse, S., and Hirano, M. (2011). Clinical and genetic spectrum of mitochondrial neurogastrointestinal encephalomyopathy. Brain 134(Pt 11), 3326-3332.
11. Godfrin, Y., and Bax, B. E. (2012). Enzyme bioreactors as drugs. Drugs of the Future. 37, 263-272.
12. Godfrin, Y., Horand, F., Franco, R., Dufour, E., Kosenko, E., Bax, B. E., Banz, A., Skorokhod, O. A., Lanao, J. M., Vitvitsky, V., et al. (2012). International seminar on the red blood cells as vehicles for drugs. Expert Opin. Biol. Ther. 12, 127-133.
13. Halter, J., Schüpbach, W. M., Casali, C., Elhasid, R., Fay, K., Hammans, S., Illa, I., Kappeler, L., Krähenbühl, S., Lehmann, T., et al. (2011). Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach. Bone Marrow Transplant. 46, 330-337.
14. Harris, I. M., Mcalister, J. M., and Prankerd, T. A. (1957). The relationship of abnormal red cells to the normal spleen. Clin. Sci. 16, 223-230.
15. Hirano, M., Silvestri, G., Blake, D. M., Lombes, A., Minetti, C., Bonilla, E., Hays, A. P., Lovelace, R. E., Butler, I., and Bertorini, T. E. (1994). Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): Clinical, biochemical, and genetic features of an autosomal recessive mitochondrial disorder. Neurology 44, 721-727.
16. Martí, R., Nishigaki, Y., and Hirano, M. (2003). Elevated plasma deoxyuridine in patients with thymidine phosphorylase deficiency. Biochem. Biophys. Res. Commun. 303, 14-18.
17. Moran, N. F., Bain, M. D., Muqit, M. M., and Bax, B. E. (2008). Carrier erythrocyte entrapped thymidine phosphorylase therapy for MNGIE. Neurology 71, 686-688.
18. Murray, A. M., Pearson, I. F., Fairbanks, L. D., Chalmers, R. A., Bain, M. D., and Bax, B. E. (2006). The mouse immune response to carrier erythrocyte entrapped antigens. Vaccine 24, 6129-6139.
19. Pearse, G. (2006). Histopathology of the thymus. Toxicol. Pathol. 34, 515-547.
20. Schellekens, H. (2002). Immunogenicity of therapeutic proteins: Clinical implications and future prospects. Clin. Ther. 24, 1720-40; discussion 1719.
21. Shaw, T., Smillie, R. H., Miller, A. E., and MacPhee, D. G. (1988). The role of blood platelets in nucleoside metabolism: Regulation of platelet thymidine phosphorylase. Mutat. Res. 200, 117-131.
22. Spinazzola, A., Marti, R., Nishino, I., Andreu, A. L., Naini, A., Tadesse, S., Pela, I., Zammarchi, E., Donati, M. A., Oliver, J. A., et al. (2002). Altered thymidine metabolism due to defects of thymidine phosphorylase. J. Biol. Chem. 277, 4128-4133.
23. Sprandel, U., Hubbard, A. R., and Chalmers, R. A. (1981) In vivo-Überlebeszeit von erythrozytenschatten als Trägersysteme für Pharmaka. Verh. Dt. Ges. Inn. Med. 87, 1172-1174.
24. Valentino, M. L., Martí, R., Tadesse, S., López, L. C., Manes, J. L., Lyzak, J., Hahn, A., Carelli, V., and Hirano, M. (2007). Thymidine and deoxyuridine accumulate in tissues of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). FEBS Lett. 581, 3410-3414.
25. Wilcoxon, F. (1945). Individual comparisons by ranking methods. Biom. Bull. 1, 80-83.