Novel strategies for stimulating erythropoiesis and potential new treatments for anaemia

Lancet

Volumn 368, Issue 9539, 2006, Pages 947-953

  Macdougall, Iain C ^a   Eckardt, Kai Uwe ^b  

^a KING S COLLEGE HOSPITAL   (United Kingdom)

^b UNIVERSITY OF ERLANGEN NUREMBERG   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

CONTINUOUS ERYTHROPOIETIN RECEPTOR ACTIVATOR; ERYTHROPOIETIN; ERYTHROPOIETIN FUSION PROTEIN; ERYTHROPOIETIN MIMETIC PEPTIDE; HEMOPOIETIC CELL PHOSPHATASE INHIBITOR; HYPOXIA INDUCIBLE FACTOR 1ALPHA; HYPOXIA INDUCIBLE FACTOR ALPHA STABILIZER; NOVEL ERYTHROPOIESIS STIMULATING PROTEIN; PEPTIDE DERIVATIVE; RECOMBINANT ERYTHROPOIETIN; SYNTHETIC ERYTHROPOIESIS PROTEIN; UNCLASSIFIED DRUG;

ANEMIA; CHRONIC KIDNEY FAILURE; CLINICAL TRIAL; DRUG EFFECT; DRUG MECHANISM; ERYTHROPOIESIS; GENE TRANSFER; GENETIC RECOMBINATION; HUMAN; NONVIRAL GENE THERAPY; PRIORITY JOURNAL; RECOMBINANT DNA TECHNOLOGY; REVIEW; SITE DIRECTED MUTAGENESIS;

EID: 33748307721     PISSN: 01406736     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0140-6736(06)69120-4     Document Type: Review

References (61)

1. Jelkmann W. Erythropoietin: structure, control of production, and function. Physiol Rev 72 (1992) 449-489
2. Tan C., Eckardt K.-U., and Ratcliffe P. Feedback modulation of renal and hepatic erythropoietin messenger RNA in response to graded anemia and hypoxia. Am J Physiol 263 (1992) F474-F481
3. Alexanian R., Vaughn W., and Ruchelman M. Erythropoietin excretion in man following androgens. J Lab Clin Med 70 (1967) 777-785
4. Caro J., Brown S., Miller O., Murray T., and Erslev A. Erythropoietin levels in uremic nephric and anephric patients. J Lab Clin Med 93 (1979) 449-458
5. Sarnak M., Tighiouart H., Manjunath G., et al. Anemia as a risk factor for cardiovascular disease in The Atherosclerosis Risk in Communities (ARIC) study. J Am Coll Cardiol 40 (2002) 27-33
6. Rossert J., and Eckardt K.-U. Erythropoietin receptors: their role beyond erythropoiesis. Nephrol Dial Transplant 20 (2005) 1025-1028
7. Miyake T., Kung C., and Goldwasser E. Purification of human erythropoietin. J Biol Chem 252 (1977) 5558-5564
8. Lin F., Suggs S., Lin C., et al. Cloning and expression of the human erythropoietin gene. Proc Natl Acad Sci USA 82 (1985) 7580-7584
9. Winearls C., Oliver D., Pippard M., Reid C., Downing M., and Cotes P. Effect of human erythropoietin derived from recombinant DNA on the anaemia of patients maintained by chronic haemodialysis. Lancet 328 (1986) 1175-1178
10. Eschbach J., Egrie J., Downing M., Browne J., and Adamson J. Correction of the anemia of end-stage renal disease with recombinant human erythropoietin. N Engl J Med 316 (1987) 73-78
11. Lacombe C., and Mayeux P. The molecular biology of erythropoietin. Nephrol Dial Transplant 14 suppl 2 (1999) 22-28
12. Koury M., and Bondurant M. Erythropoietin retards DNA breakdown and prevents programmed death in erythroid progenitor cells. Science 248 (1990) 378-381
13. Casadevall N., Nataf J., Viron B., et al. Pure red-cell aplasia and anti-erythropoietin antibodies in patients treated with recombinant erythropoietin. N Engl J Med 346 (2002) 469-475
14. Bennett C., Luminari S., Nissenson A., et al. Pure red-cell aplasia and epoetin therapy. N Engl J Med 351 (2004) 1403-1408
15. Sharma B., Bader F., Templeman T., Lisi P., Ryan M., and Heavner G. Technical investigations into the cause of the increased incidence of antibody-mediated pure red cell aplasia associated with Eprex. Eur J Hosp Pharm 5 (2004) 86-91
16. Boven K., Stryker S., Knight J., et al. The increased incidence of pure red cell aplasia with an Eprex formulation in uncoated rubber stopper syringes. Kidney Int 67 (2005) 2346-2353
17. Schellekens H. Follow-on biologics: challenges of the 'next generation'. Nephrol Dial Transplant 20 suppl 4 (2005) iv31-iv36
18. Committee for Proprietary Medicinal Products. Guideline on comparability of medicinal products containing biotechnology-derived proteins as active substance. Quality issues. Evaluation of medicines for human use (2003), European Agency for the Evaluation of Medicinal Products, London
19. Egrie J., and Browne J. Development and characterization of novel erythropoiesis stimulating protein (NESP). Br J Cancer 84 suppl 1 (2001) 3-10
20. Macdougall I., Gray S., Elston O., et al. Pharmacokinetics of novel erythropoiesis stimulating protein compared with epoetin alfa in dialysis patients. J Am Soc Nephrol 10 (1999) 2392-2395
21. Vanrenterghem Y., Barany P., Mann J., et al., for the European/Australian NESP 970200 study group. Randomized trial of darbepoetin alfa for treatment of renal anemia at a reduced dose frequency compared with rHuEPO in dialysis patients. Kidney Int 62 (2002) 2167-2175
22. Nissenson A., Swan S., Lindberg J., et al. Randomized, controlled trial of darbepoetin alfa for the treatment of anemia in hemodialysis patients. Am J Kidney Dis 40 (2002) 110-118
23. Jadoul M., Vanrenterghem Y., Foret M., Walker R., and Gray S. Darbepoetin alfa administered once monthly maintains haemoglobin levels in stable dialysis patients. Nephrol Dial Transplant 19 (2004) 898-903
24. Haselbeck A., Bailon P., Pahlke W., et al. The discovery and characterization of CERA, an innovative agent for the treatment of anemia. Blood 100 (2002) 227A (abstr 857)
25. Macdougall I. CERA (continuous erythropoietin receptor activator): a new erythropoiesis-stimulating agent for the treatment of anemia. Curr Hematol Rep 4 (2005) 436-440
26. Provenzano R., Besarab A., Macdougall I., et al., for the BA16528 study group. CERA (continuous erythropoietin receptor activator) administered up to once every 3 weeks corrects anemia in patients with chronic kidney disease not on dialysis. J Am Soc Nephrol 15 (2004) 544A
27. Locatelli F., Villa G., Arias M., et al., for the BA16286 study group. CERA (continuous erythropoietin receptor activator) maintains hemoglobin levels in dialysis patients when administered subcutaneously up to once every 4 weeks. J Am Soc Nephrol 15 (2004) 543A
28. Dmoszynska A., Kloczko J., Rokicka M., et al. CERA (continuous erythropoietin receptor activator) in patients with multiple myeloma: an exploratory phase I-II dose escalation study. J Clin Oncol 22 suppl 15 (2004) 14S
29. Kochendoerfer G., Chen S., Mao F., et al. Design and chemical synthesis of a homogeneous polymer-modified erythropoiesis protein. Science 299 (2003) 884-887
30. Sytkowski A., Lunn E., Risinger M., and Davis K. An erythropoietin fusion protein comprised of identical repeating domains exhibits enhanced biological properties. J Biol Chem 274 (1999) 24773-24778
31. Wrighton N., Farrell F., Chang R., et al. Small peptides as potent mimetics of the protein hormone erythropoietin. Science 273 (1996) 458-464
32. Johnson D., and Jolliffe L. Erythropoietin mimetic peptides and the future. Nephrol Dial Transplant 15 (2000) 1274-1277
33. Woodburn K., Fan Q., Holmes C., et al. Preclinical evaluation of Hematide™, a novel erythropoietic receptor agonist for the treatment of anemia caused by kidney disease. Blood 104 (2004) 2904 (abstr)
34. Woodburn KW, Winslow S, Leuther KK, et al. Hematide™, a peptidic erythropoiesis stimulating agent that corrects anemia induced by partial nephrectomy and erythropoietin-specific antibodies in rats. Stockholm: European Haematology Association meeting, 2005.
35. Stead R, Lambert J, Wessels D, et al. Evaluation of the safety and pharmacodynamics of Hematide, a novel erythropoietic agent, in a phase 1, double-blind, placebo-controlled, dose escalation study in healthy volunteers. Blood (in press).
36. Qureshi S., Kim R., Konteatis Z., et al. Mimicry of erythropoietin by a nonpeptide molecule. Proc Natl Acad Sci USA 96 (1999) 12156-12161
37. Klingmuller U., Lorenz U., Cantley L., Neel B., and Lodish H. Specific recruitment of SH-PTP1 to the erythropoietin receptor causes inactivation of JAK2 and termination of proliferative signals. Cell 80 (1995) 729-738
38. Akagi S., Ichikawa H., Okada T., et al. The critical role of SRC homology domain 2-containing tyrosine phosphatase-1 in recombinant human erythropoietin hyporesponsive anemia in chronic hemodialysis patients. J Am Soc Nephrol 15 (2004) 3215-3224
39. Schofield C., and Ratcliffe P. Oxygen sensing by HIF hydroxylases. Nat Rev Mol Cell Biol 5 (2004) 343-354
40. Urquilla P., Fong A., Oksanen S., et al. Upregulation of endogenous EPO in healthy subjects by inhibition of HIF-PH. J Am Soc Nephrol 15 (2004) 546A
41. Wiecek A., Piecha G., Ignacy W., et al. Pharmacological stabilization of HIF increases hemoglobin concentration in anemic patients with chronic kidney disease. Nephrol Dial Transplant 20 suppl 5 (2005) v195
42. Maxwell P. HIF-1: an oxygen response system with special relevance to the kidney. J Am Soc Nephrol 14 (2003) 2712-2722
43. Gordeuk V., Sergueeva A., Miasnikova G., et al. Congenital disorder of oxygen sensing: association of the homozygous Chuvash polycythemia VHL mutation with thrombosis and vascular abnormalities but not tumors. Blood 103 (2004) 3924-3932
44. Fattori E., Cappelletti M., Zampaglione I., et al. Gene electro-transfer on an improved erythropoietin plasmid in mice and non-human primates. J Gene Med 7 (2005) 228-236
45. Rivera V., Gao G., Grant R., et al. Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer. Blood 105 (2005) 1424-1430
46. Kakeda M., Hiratsuka M., Nagata K., et al. Human artificial chromosome (HAC) vector provides long-term therapeutic transgene expression in normal human primary fibroblasts. Gene Ther 12 (2005) 852-856
47. Schwenter F., Schneider B., Pralong W., Deglon N., and Aebischer P. Survival of encapsulated human primary fibroblasts and erythropoietin expression under xenogeneic conditions. Hum Gene Ther 15 (2004) 669-680
48. Lippin Y., Dranitzki-Elhalel M., Brill-Almon E., et al. Human erythropoietin gene therapy for patients with chronic renal failure. Blood 106 (2005) 2280-2286
49. Takács K., Du Roure C., Nabarro S., et al. The regulated long-term delivery of therapeutic proteins by using antigen-specific B lymphocytes. PNAS 101 (2004) 16298-16303
50. Binley K., Askham Z., Iqball S., et al. Long-term reversal of chronic anemia using a hypoxia-regulated erythropoietin gene therapy. Blood 100 (2002) 2406-2413
51. Henke M., Laszig R., Rübe C., et al. Erythropoietin to treat head and neck cancer patients with anaemia undergoing radiotherapy: randomised, double-blind, placebo-controlled trial. Lancet 362 (2003) 1255-1260
52. Leyland-Jones B., Semiglazov V., Pawlicki M., et al. Maintaining normal hemoglobin levels with epoetin alfa in mainly nonanemic patients with metastatic breast cancer receiving first-line chemotherapy: a survival study. J Clin Oncol 23 (2005) 5960-5972
53. Besarab A., Bolton W., Browne J., et al. The effects of normal as compared with low hematocrit values in patients with cardiac disease who are receiving hemodialysis and epoetin. N Engl J Med 339 (1998) 584-590
54. Parfrey P., Foley R., Wittreich B., et al. Double-blind comparison of full and partial anemia correction in incident hemodialysis patients without symptomatic heart disease. J Am Soc Nephrol 16 (2005) 2180-2189
55. Mix T., Brenner R., Cooper M., et al. Rationale-Trial to Reduce Cardiovascular Events with Aranesp Therapy (TREAT): evolving the management of cardiovascular risk in patients with chronic kidney disease. Am Heart J 149 (2005) 408-413
56. Ehrenreich H., Hasselblatt M., Dembowski C., et al. Erythropoietin therapy for acute stroke is both safe and beneficial. Mol Med 8 (2002) 495-505
57. Lipton S. Erythropoietin for neurologic protection and diabetic neuropathy. N Engl J Med 350 (2004) 2516-2517
58. Parsa C., Matsumoto A., Kim J., et al. A novel protective effect of erythropoietin in the infarcted heart. J Clin Invest 112 (2003) 999-1007
59. Sharples E., Patel N., Brown P., et al. Erythropoietin protects the kidney against the injury and dysfunction caused by ischemia-reperfusion. J Am Soc Nephrol 15 (2004) 2115-2124
60. Chatterjee P. Pleiotropic renal actions of erythropoietin. Lancet 365 (2005) 1890-1892
61. Leist M., Ghezzi P., Grasso G., et al. Derivatives of erythropoietin that are tissue protective but not erythropoietic. Science 305 (2004) 239-242