Enzyme bioreactors as drugs

Drugs of the Future

Volumn 37, Issue 4, 2012, Pages 263-272

  Godfrin, Yann ^a   Bax, B E ^b  

^a ERYTECH PHARMA   (France)

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

Author keywords

Asparaginase; Cancer therapy; Enzyme replacement therapy; Erythrocytes; MNGIE; Thymidine phosphorylase

Indexed keywords

AGALSIDASE BETA; ASPARAGINASE; GALSULFASE; IDURONATE 2 SULFATASE; IMIGLUCERASE; LARONIDASE; RECOMBINANT GLUCAN 1,4 ALPHA GLUCOSIDASE; THYMIDINE PHOSPHORYLASE;

BLOOD CLOTTING DISORDER; CANCER THERAPY; CHEST TIGHTNESS; DRUG FORMULATION; DRUG HYPERSENSITIVITY; DRUG MANUFACTURE; DRUG METABOLISM; ENCAPSULATION; ENZYME REPLACEMENT; ERYTHEMA; ERYTHROCYTE; GENE MUTATION; HUMAN; HYPOALBUMINEMIA; IMMUNOGENICITY; LIVER DISEASE; LIVER TOXICITY; LYMPHATIC LEUKEMIA; MNGIE SYNDROME; NONHUMAN; PANCREATITIS; PROTEIN BLOOD LEVEL; REVIEW; SIDE EFFECT;

EID: 84952989026     PISSN: 03778282     EISSN: None     Source Type: Journal    
DOI: 10.1358/dof.2012.037.04.1779020     Document Type: Review

References (41)

1. Muller, H.J., Boos, J. Use of L-asparaginase in childhood ALL. Crit Rev Oncol Hematol 1998, 28(2): 97-113. (Pubitemid 28390409)
2. Leslie, M., Case, M.C., Hall, A.G. et al. Expression levels of asparagine synthetase in blasts from children and adults with acute lymphoblastic leukaemia. Br J Haematol 2006, 132(6): 740-2. (Pubitemid 43381635)
3. Lorenzi, P.L., Llamas, J., Gunsior, M. et al. Asparagine synthetase is a predictive biomarker of L-asparaginase activity in ovarian cancer cell lines. Mol Cancer Ther 2008, 7(10): 3123-8.
4. Raetz, E.A., Salzer, W.L. Tolerability and efficacy of L-asparaginase therapy in pediatric patients with acute lymphoblastic leukemia. J Pediatr Hematol Oncol 2010, 32(7): 554-63.
5. Asselin, B.L., Whitin, J.C., Coppola, D.J. et al. Comparative pharmacokinetic studies of three asparaginase preparations. J Clin Oncol 1993, 11(9): 1780-6. (Pubitemid 23277332)
6. Alpar, H.O., Lewis, D.A. Therapeutic efficacy of asparaginase encapsulated in intact erythrocytes. Biochem Pharmacol 1985, 34(2): 257-61. (Pubitemid 15153420)
7. Updike, S.J. Entrapment of L-asparaginase in red blood cells. A strategy to improve treatment of acute lymphoblastic leukemia. Bibl Haematol 1985, 51: 65-74.
8. Naqi, A., DeLoach, J.R., Andrews, K. et al. Determination of parameters for enzyme therapy using L-asparaginase entrapped in canine erythrocytes. Biotechnol Appl Biochem 1988, 10(4): 365-72.
9. Kravtzoff, R., Desbois, I., Lamagnere, J.P. et al. Improved pharmacodynamics of L-asparaginase-loaded in human red blood cells. Eur J Clin Pharmacol 1996, 49(6): 465-70.
10. Kwon, Y.M., Chung, H.S., Moon, C. et al. L-Asparaginase encapsulated intact erythrocytes for treatment of acute lymphoblastic leukemia (ALL). J Control Release 2009, 139(3): 182-9.
11. Domenech, C., Thomas, X., Chabaud, S. et al. L-Asparaginase loaded red blood cells in refractory or relapsing acute lymphoblastic leukaemia in children and adults: Results of the GRASPALL 2005-01 randomized trial. Br J Haematol 2011, 153(1): 58-65.
12. Capizzi, R.L., Davis, R., Powell, B. et al. Synergy between high-dose cytarabine and asparaginase in the treatment of adults with refractory and relapsed acute myelogenous leukemia - A Cancer and Leukemia Group B Study. J Clin Oncol 1988, 6(3): 499-508. (Pubitemid 18090699)
13. Lorenzi, P.L., Weinstein, J.N. Asparagine synthetase: A new potential biomarker in ovarian cancer. Drug News Perspect 2009, 22(1): 61-4.
14. Distasio, J.A., Durden, D.L., Paul, R.D. et al. Alteration in spleen lymphoid populations associated with specific amino acid depletion during L-asparaginase treatment. Cancer Res 1982, 42(1): 252-8. (Pubitemid 12207275)
15. Panosyan, E.H., Grigoryan, R.S., Avramis, I.A. et al. Deamination of glutamine is a prerequisite for optimal asparagine deamination by asparaginases in vivo (CCG-1961). Anticancer Res 2004, 24(2C): 1121-5. (Pubitemid 38624949)
16. Distasio, J.A., Salazar, A.M., Nadji, M. et al. Glutaminase-free asparaginase from vibrio succinogenes: An antilymphoma enzyme lacking hepatotoxicity. Int J Cancer 1982, 30(3): 343-7. (Pubitemid 12035865)
17. Kelo, E., Noronkoski, T., Stoineva, I.B. et al. Beta-aspartylpeptides as substrates of L-asparaginases from Escherichia coli and Erwinia chrysanthemi. FEBS Lett 2002, 528(1-3): 130-2. (Pubitemid 35258012)
18. Purcell, R.T., Lockey, R.F. Immunologic responses to therapeutic biologic agents. J Investig Allergol Clin Immunol 2008, 18(5): 335-42.
19. Hunley, T.E., Corzo, D., Dudek, M. et al. Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease. Pediatrics 2004, 114(4): e532-5. (Pubitemid 41529751)
20. Bax, B.E., Bain, M.D., Fairbanks, L.D. et al. In vitro and in vivo studies with human carrier erythrocytes loaded with polyethylene glycol-conjugated and native adenosine deaminase. Br J Haematol 2000, 109(3): 549-54. (Pubitemid 30415181)
21. Bax, B.E., Bain, M.D., Fairbanks, L.D. et al. A 9-yr evaluation of carrier erythrocyte encapsulated adenosine deaminase (ADA) therapy in a patient with adult-type ADA deficiency. Eur J Haematol 2007, 79(4): 338-48. (Pubitemid 47390137)
22. Godfrin, Y., Horand, F., Franco, R. et al. Meeting highlights: International seminar on the red blood cells as vehicles for drugs. Expert Opin Biol Ther 2012, 12(1): 127-33.
23. Hirano, M., Nishigaki, Y., Marti, R. Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A disease of two genomes. Neurologist 2004, 10(1): 8-17. (Pubitemid 38140835)
24. Hirano, M., Silvestri, G., Blake, D.M. et al. Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): Clinical, biochemical, and genetic features of an autosomal recessive mitochondrial disorder. Neurology 1994, 44(4): 721-7. (Pubitemid 24130006)
25. Nishino, I., Spinazzola, A., Hirano, M. Thymidine phosphorylase gene mutations in MNGIE, a human mitochondrial disorder. Science 1999, 283(5402): 689-92.
26. Marti, R., Nishigaki, Y., Hirano, M. Elevated plasma deoxyuridine in patients with thymidine phosphorylase deficiency. Biochem Biophys Res Commun 2003, 303(1): 14-8. (Pubitemid 36338200)
27. Nishino, I., Spinazzola, A., Papadimitriou, A. et al. Mitochondrial neurogastrointestinal encephalomyopathy: An autosomal recessive disorder due to thymidine phosphorylase mutations. Ann Neurol 2000, 47(6): 792- 800.
28. Spinazzola, A., Marti, R., Nishino, I. et al. Altered thymidine metabolism due to defects of thymidine phosphorylase. J Biol Chem 2002, 277(6): 4128-33. (Pubitemid 34968672)
29. Valentino, M.L., Martí, R., Tadesse, S. et al. Thymidine and deoxyuridine accumulate in tissues of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). FEBS Lett 2007, 581(18): 3410-4. (Pubitemid 47048186)
30. Nishigaki, Y., Marti, R., Copeland, W.C. et al. Site-specific somatic mitochondrial DNA point mutations in patients with thymidine phosphorylase deficiency. J Clin Invest 2003, 111(12): 1913-21. (Pubitemid 38057736)
31. Nishigaki, Y., Marti, R., Hirano, M. ND5 is a hot-spot for multiple atypical mitochondrial DNA deletions in mitochondrial neurogastrointestinal encephalomyopathy. Hum Mol Genet 2004, 13(1): 91-101. (Pubitemid 38072138)
32. Halter, J., Schupbach, W.M., Casali, C. et al. Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): A consensus conference proposal for a standardized approach. Bone Marrow Transplant 2011, 46(3): 330-7.
33. Bax, B.E., Bain, M.D., Talbot, P.J. et al. Survival of human carrier erythrocytes in vivo. Clin Sci (Lond) 1999, 96(2): 171-8. (Pubitemid 29094936)
34. Moran, N.F., Bain, M.D., Muqit, M.M. et al. Carrier erythrocyte entrapped thymidine phosphorylase therapy for MNGIE. Neurology 2008, 71(9): 686- 8.
35. Barton, G.J., Ponting, C.P., Spraggon, G. et al. Human platelet-derived endothelial cell growth factor is homologous to Escherichia coli thymidine phosphorylase. Protein Sci 1992, 1(5): 688-90. (Pubitemid 23009203)
36. Aldurazyme Prescribing Information (http://www.aldurazyme.com/pdf/az-us- hc-pi.pdf).
37. Fabrazyme Prescribing Information (http://www.fabrazyme.com/hcp/pi/fz-us- hc-pi.pdf).
38. Naglazyme Prescribing Information (http://www.naglazyme.com/en/documents/ Naglazyme-Prescribing-Information.pdf).
39. Cerezyme Prescribing Information (http://www.cerezyme.com/~/media/Files/ CerezymeUS/pdf/cerezyme-pi.pdf).
40. Myozyme Prescribing Information (http://www.genzyme.com/components/ highlights/mz-pi.pdf).
41. Elaprase Prescribing Information (http://www.elaprase.com/pdf/ ElaprasePI40-0120-REV-0-GT4.pdf).