Emerging therapies for polycystic kidney disease

Current Opinion in Pharmacology

Volumn 5, Issue 5 SPEC.ISS., 2005, Pages 535-542

  Gattone II, Vincent H ^a  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANGIOTENSIN RECEPTOR ANTAGONIST; ANTILIPEMIC AGENT; ANTISENSE OLIGONUCLEOTIDE; AVI 4126; CHLOROTRIANISENE; CYCLIC AMP; CYCLOOXYGENASE 2 INHIBITOR; DIPEPTIDYL CARBOXYPEPTIDASE INHIBITOR; EPIDERMAL GROWTH FACTOR RECEPTOR KINASE INHIBITOR; MATRIX METALLOPROTEINASE INHIBITOR; METHYLPREDNISOLONE; MOZAVAPTAN; MYC PROTEIN; OCTREOTIDE; PACLITAXEL; PIOGLITAZONE; RAPAMYCIN; SMALL INTERFERING RNA; TOLVAPTAN; UNCLASSIFIED DRUG; VASOPRESSIN RECEPTOR ANTAGONIST; VASOPRESSIN V2 RECEPTOR;

CLINICAL TRIAL; DISEASE CONTROL; DISEASE COURSE; DRUG MECHANISM; DRUG SCREENING; GENE LOCUS; GENE THERAPY; GENETIC ASSOCIATION; HUMAN; KIDNEY CYST; KIDNEY POLYCYSTIC DISEASE; MONOGENIC DISORDER; NONHUMAN; PATHOLOGY; PHENOTYPE; PRIORITY JOURNAL; REVIEW;

EID: 24344502280     PISSN: 14714892     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.coph.2005.04.016     Document Type: Review

References (85)

1. G.J. Pazour Intraflagellar transport and cilia-dependent renal disease: the ciliary hypothesis of polycystic kidney disease J Am Soc Nephrol 15 2004 2528-2536
2. Q. Zhang P.D. Taulman B.K. Yoder Cystic kidney diseases: All roads lead to the cilium Physiology (Bethesda) 19 2004 225-230
3. V.H. Gattone J.P. Calvet B.D. Cowley A.P. Evan T.S. Shaver K. Helmstadter J.J. Grantham Autosomal recessive polycystic kidney disease in a murine model. A gross and microscopic description Lab Invest 59 1988 231-238
4. J.L. Ricker V.H. Gattone J.P. Calvet C.A. Rankin Development of autosomal recessive polycystic kidney disease in BALB/c-cpk/cpk mice J Am Soc Nephrol 11 2000 1837-1847
5. J.H. Moyer M.J. Lee-Tischler H.Y. Kwon J.J. Schrick E.D. Avner W.E. Sweeney V.L. Godfrey N.L. Cacheiro J.E. Wilkinson R.P. Woychik Candidate gene associated with a mutation causing recessive polycystic kidney disease in mice Science 264 1994 1329-1333
6. P.D. Taulman C.J. Haycraft D.F. Balkovetz B.K. Yoder Polaris, a protein involved in left-right axis patterning, localizes to basal bodies and cilia Mol Biol Cell 12 2001 589-599
7. J. Nauta Y. Ozawa W.E. Sweeney J.C. Rutledge E.D. Avner Renal and biliary abnormalities in a new murine model of autosomal recessive polycystic kidney disease Pediatr Nephrol 7 1993 163-172
8. C.J. Ward M.C. Hogan S. Rossetti D. Walker T. Sneddon X. Wang V. Kubly J.M. Cunningham R. Bacallao M. Ishibashi et al. The gene mutated in autosomal recessive polycystic kidney disease encodes a large, receptor-like protein Nat Genet 30 2002 259-269
9. E.A. Otto B. Schermer T. Obara J.F. O'Toole K.S. Hiller A.M. Mueller R.G. Ruf J. Hoefele F. Beekmann D. Landau et al. Mutations in INVS encoding inversin cause nephronophthisis type 2, linking renal cystic disease to the function of primary cilia and left-right axis determination Nat Genet 34 2003 413-420
10. H. Takahashi J.P. Calvet D. Dittemore-Hoover K. Yoshida J.J. Grantham V.H. Gattone A hereditary model of slowly progressive polycystic kidney disease in the mouse J Am Soc Nephrol 1 1991 980-989
11. H. Omran K. Haffner S. Burth C. Fernandez B. Fargier A. Villaquiran H.G. Nothwang S. Schnittger H. Lehrach D. Woo et al. Human adolescent nephronophthisis: Gene locus synteny with polycystic kidney disease in pcy mice J Am Soc Nephrol 12 2001 107-113
12. G. Wu V. D'Agati Y. Cai G. Markowitz J.H. Park D.M. Reynolds Y. Maeda T.C. Le H. Hou R. Kucherlapati et al. Somatic inactivation of Pkd2 results in polycystic kidney disease Cell 93 1998 177-188
13. B.D. Cowley S. Gudapaty A.L. Kraybill B.D. Barash M.A. Harding J.P. Calvet V.H. Gattone Autosomal-dominant polycystic kidney disease in the rat Kidney Int 43 1993 522-534
14. W. Lu B. Peissel H. Babakhanlou A. Pavlova L. Geng X. Fan C. Larson G. Brent J. Zhou Perinatal lethality with kidney and pancreas defects in mice with a targetted Pkd1 mutation Nat Genet 17 1997 179-181
15. K. Kim I. Drummond O. Ibraghimov-Beskrovnaya K. Klinger M.A. Arnaout Polycystin 1 is required for the structural integrity of blood vessels Proc Natl Acad Sci USA 97 2000 1731-1736
16. C. Boulter S. Mulroy S. Webb S. Fleming K. Brindle R. Sandford Cardiovascular, skeletal, and renal defects in mice with a targeted disruption of the Pkd1 gene Proc Natl Acad Sci USA 98 2001 12174-12179
17. K.B. Piontek D.L. Huso A. Grinberg L. Liu D. Bedja H. Zhao K. Gabrielson F. Qian C. Mei H. Westphal G.G. Germino A functional floxed allele of Pkd1 that can be conditionally inactivated in vivo J Am Soc Nephrol 15 2004 3035-3043
18. I.S. Lantinga-van Leeuwen J.G. Dauwerse H.J. Baelde W.N. Leonhard A. van de Wal C.J. Ward S. Verbeek M.C. Deruiter M.H. Breuning E. de Heer D.J. Peters Lowering of Pkd1 expression is sufficient to cause polycystic kidney disease Hum Mol Genet 13 2004 3069-3077
19. P.A. Gabow W.D. Kaehny A.M. Johnson I.T. Duley M. Manco-Johnson D.C. Lezotte R.W. Schrier The clinical utility of renal concentrating capacity in polycystic kidney disease Kidney Int 35 1989 675-680
20. K. MacRea-Dell R.A. McDonald S.L. Watkins E.D. Avner Polycystic Kidney Disease Edited by E.D. Avner W.E. Harmon P. Niaudet In Pediatric Nephrology edn 5 2004 Lippincott Williams & Wilkins 676-699
21. F. Hildebrandt R. Waldherr R. Kutt M. Brandis The nephronophthisis complex: Clinical and genetic aspects Clin Investig 70 1992 802-808
22. V.H. Gattone R.L. Maser C. Tian J.M. Rosenberg M.G. Branden Developmental expression of urine concentration-associated genes and their altered expression in murine infantile-type polycystic kidney disease Dev Genet 24 1999 309-318
23. V.H. Gattone X. Wang P.C. Harris V.E. Torres Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist Nat Med 9 2003 1323-1326
24. V.E. Torres X. Wang Q. Qian S. Somlo P.C. Harris V.H. Gattone Effective treatment of an orthologous model of autosomal dominant polycystic kidney disease Nat Med 10 2004 363-364
25. T.K. Neufeld D. Douglass M. Grant M. Ye F. Silva T. Nadasdy J.J. Grantham In vitro formation and expansion of cysts derived from human renal cortex epithelial cells Kidney Int 41 1992 1222-1236
26. M. Thibonnier P. Coles A. Thibonnier M. Shoham The basic and clinical pharmacology of nonpeptide vasopressin receptor antagonists Annu Rev Pharmacol Toxicol 41 2001 175-202
27. Y. Yamamura H. Ogawa H. Yamashita T. Chihara H. Miyamoto S. Nakamura T. Onogawa T. Yamashita T. Hosokawa T. Mori et al. Characterization of a novel aquaretic agent, OPC-31260, as an orally effective, nonpeptide vasopressin V2 receptor antagonist Br J Pharmacol 105 1992 787-791
28. X. Wang V. Gattone P.C. Harris V.E. Torres Effectiveness of vasopressin V2 receptor antagonists OPC-31260 and OPC-41061 on polycystic kidney disease development in the PCK rat J Am Soc Nephrol 16 2005 846-851
29. A.B. Chapman L.M. Guay-Woodford J.J. Grantham V.E. Torres K.T. Bae D.A. Baumgarten P.J. Kenney B.F. King J.F. Glockner L.H. Wetzel et al. Consortium for radiologic imaging studies of polycystic kidney disease cohort. Renal structure in early autosomal-dominant polycystic kidney disease (ADPKD): The Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP) cohort Kidney Int 64 2003 1035-1045
30. B.F. King V.E. Torres M.E. Brummer A.B. Chapman K.T. Bae J.F. Glockner K. Arya J.P. Felmlee J.J. Grantham L.M. Guay-Woodford et al. Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP). Magnetic resonance measurements of renal blood flow as a marker of disease severity in autosomal-dominant polycystic kidney disease Kidney Int 64 2003 2214-2221
31. B.D. Cowley F.L. Smardo J.J. Grantham J.P. Calvet Elevated c-myc protooncogene expression in autosomal recessive polycystic kidney disease Proc Natl Acad Sci USA 85 1988 2578
32. V.H. Gattone K.A. Kuenstler G.W. Lindemann X. Lu B.D. Cowley C.A. Rankin J.P. Calvet Renal expression of a transforming growth factor-alpha transgene accelerates the progression of inherited, slowly progressive polycystic kidney disease in the mouse J Lab Clin Med 127 1996 214-222
33. M.A. Harding V.H. Gattone J.J. Grantham J.P. Calvet Localization of overexpressed c-myc mRNA in polycystic kidneys of the cpk mouse Kidney Int 41 1992 317-325
34. H. Husson P. Manavalan V.R. Akmaev R.J. Russo B. Cook B. Richards D. Barberio D. Liu X. Cao G.M. Landes et al. New insights into ADPKD molecular pathways using combination of SAGE and microarray technologies Genomics 84 2004 497-510
35. M. Trudel V. D'Agati F. Costantini C-myc as an inducer of polycystic kidney disease in transgenic mice Kidney Int 39 1991 665-671
36. M. Trudel N. Chretien V. D'Agati Disappearance of polycystic kidney disease in revertant c-myc transgenic mice Mamm Genome 5 1994 149-152
37. J.L. Ricker J.E. Mata P.L. Iversen V.H. Gattone C-myc antisense oligonucleotide treatment ameliorates murine ARPKD Kidney Int 61 Suppl 1 2002 125-131
38. V.H. Gattone J.R. Ricker Interventions in polycystic kidney disease using antisense oligonucleotides [abstract] FASEB J 16 2002 A1097
39. V.H. Gattone J.L. Ricker C.M. Trambaugh R.M. Klein Multiorgan mRNA misexpression in murine autosomal recessive polycystic kidney disease Kidney Int 62 2002 1560-1569
40. V.H. Gattone G.K. Andrews F.W. Niu L.J. Chadwick R.M. Klein J.P. Calvet Defective epidermal growth factor gene expression in mice with polycystic kidney disease Dev Biol 138 1990 225-230
41. B.D. Cowley J.C. Rupp Abnormal expression of epidermal growth factor and sulfated glycoprotein SGP-2 messenger RNA in a rat model of autosomal dominant polycystic kidney disease J Am Soc Nephrol 6 1995 1679-1681
42. T. Weinstein D. Hwang A. Lev-Ran Y. Ori A. Korzets J. Levi Excretion of epidermal growth factor in human adult polycystic kidney disease Isr J Med Sci 33 1997 641-642
43. S.A. Orellana W.E. Sweeney C.D. Neff E.D. Avner Epidermal growth factor receptor expression is abnormal in murine polycystic kidney Kidney Int 47 1995 490-499
44. W.E. Sweeney E.D. Avner Functional activity of epidermal growth factor receptors in autosomal recessive polycystic kidney disease Am J Physiol 275 1998 F387-F394
45. J. Nauta W.E. Sweeney J.C. Rutledge E.D. Avner Biliary epithelial cells from mice with congenital polycystic kidney disease are hyperresponsive to epidermal growth factor Pediatr Res 37 1995 755-763
46. K.M. Dell R. Nemo W.E. Sweeney J.I. Levin P. Frost E.D. Avner A novel inhibitor of tumor necrosis factor-alpha converting enzyme ameliorates polycystic kidney disease Kidney Int 60 2001 1240-1248
47. W.E. Sweeney Y. Chen K. Nakanishi P. Frost E.D. Avner Treatment of polycystic kidney disease with a novel tyrosine kinase inhibitor Kidney Int 57 2000 33-40
48. V.E. Torres W.E. Sweeney X. Wang Q. Qian P.C. Harris P. Frost E.D. Avner EGF receptor tyrosine kinase inhibition attenuates the development of PKD in Han:SPRD rats Kidney Int 64 2003 1573-1579
49. R.O. von Vigier W.E. Sweeney N.S. Murcia P. Frost E.D. Avner Receptor tyrosine kinase inhibition attenuates hepatobiliary abnormalities in a murine model of autosomal recessive polycystic kidney disease [abstract] J Am Soc Neprol 15 2004 57A
50. V.E. Torres W.E. Sweeney X. Wang Q. Qian P.C. Harris P. Frost E.D. Avner Epidermal growth factor receptor tyrosine kinase inhibition is not protective in PCK rats Kidney Int 66 2004 1766-1773
51. V.H. Gattone D.A. Lowden B.D. Cowley Epidermal growth factor ameliorates autosomal recessive polycystic kidney disease in mice Dev Biol 169 1995 504-510
52. J.L. Ricker V.H. Gattone J.P. Calvet C.A. Rankin Development of autosomal recessive polycystic kidney disease in BALB/c-cpk/cpk mice J Am Soc Nephrol 11 2000 1837-1847
53. K. Nakanishi V.H. Gattone W.E. Sweeney E.D. Avner Renal dysfunction but not cystic change is ameliorated by neonatal epidermal growth factor in bpk mice Pediatr Nephrol 16 2001 45-50
54. L.D. Truong Y.J. Choi S.S. Shen G. Ayala R. Amato B. Krishnan Renal cystic neoplasms and renal neoplasms associated with cystic renal diseases: Pathogenetic and molecular links Adv Anat Pathol 10 2003 135-159
55. D.D. Woo S.Y. Miao J.C. Pelayo A.S. Woolf Taxol inhibits progression of congenital polycystic kidney disease Nature 368 1994 750-753
56. C.S. Sommardahl R.P. Woychik W.E. Sweeney E.D. Avner J.E. Wilkinson Efficacy of taxol in the orpk mouse model of polycystic kidney disease Pediatr Nephrol 11 1997 728-733
57. J.R. Martinez B.D. Cowley V.H. Gattone S. Nagao T. Yamaguchi S. Kaneta H. Takahashi J.J. Grantham The effect of paclitaxel on the progression of polycystic kidney disease in rodents Am J Kidney Dis 29 1997 435-444
58. H.M. Aukema M.R. Ogborn K. Tomobe H. Takahashi T. Hibino B.J. Holub Effects of dietary protein restriction and oil type on the early progression of murine polycystic kidney disease Kidney Int 42 1992 837-842
59. K. Tomobe D. Philbrick H.M. Aukema W.F. Clark M.R. Ogborn A. Parbtani H. Takahashi B.J. Holub Early dietary protein restriction slows disease progression and lengthens survival in mice with polycystic kidney disease J Am Soc Nephrol 5 1994 1355-1360
60. B.D. Cowley J.J. Grantham M.J. Muessel A.L. Kraybill V.H. Gattone Modification of disease progression in rats with inherited polycystic kidney disease Am J Kidney Dis 27 1996 865-879
61. V.E. Torres K.A. Donovan G. Scicli K.E. Holley S.N. Thibodeau O.A. Carretero T. Inagami J.A. McAteer C.M. Johnson Synthesis of renin by tubulocystic epithelium in autosomal-dominant polycystic kidney disease Kidney Int 42 1992 364-373
62. M. Loghman-Adham C.E. Soto T. Inagami L. Cassis The intrarenal renin-angiotensin system in autosomal dominant polycystic kidney disease Am J Physiol Renal Physiol 287 2004 F775-F788
63. M.A. van Dijk M.H. Breuning R. Duiser L.A. van Es R.G. Westendorp No effect of enalapril on progression in autosomal dominant polycystic kidney disease Nephrol Dial Transplant 18 2003 2314-2320
64. D.S. Keith V.E. Torres C.M. Johnson K.E. Holley Effect of sodium chloride, enalapril, and losartan on the development of polycystic kidney disease in Han:SPRD rats Am J Kidney Dis 24 1994 491-498
65. M.R. Ogborn S. Sareen G. Pinette Cilazapril delays progression of hypertension and uremia in rat polycystic kidney disease Am J Kidney Dis 26 1995 942-946
66. V.H. Gattone B.D. Cowley B.D. Barash S. Nagao H. Takahashi T. Yamaguchi J.J. Grantham Methylprednisolone retards the progression of inherited polycystic kidney disease in rodents Am J Kidney Dis 25 1995 302-313
67. Y. Tao J. Kim R.W. Schrier C.L. Edelstein Rapamycin markedly slows disease progression in a rat model of polycystic kidney disease J Am Soc Nephrol 16 2005 46-51
68. B. McKee R. Perrone V.H. Gattone Rapamycin ameliorates murine polycystic kidney disease [abstract] Fed Proc 19 2005 A194
69. S. Nagao T. Yamaguchi M. Kasahara M. Kusaka J. Matsuda N. Ogiso H. Takahashi J.J. Grantham Effect of probucol in a murine model of slowly progressive polycystic kidney disease Am J Kidney Dis 35 2000 221-226
70. R.D. Gile B.D. Cowley V.H. Gattone M.P. O'Donnell S.K. Swan J.J. Grantham Effect of lovastatin on the development of polycystic kidney disease in the Han:SPRD rat Am J Kidney Dis 26 1995 501-507
71. S. Muto A. Aiba Y. Saito K. Nakao K. Nakamura K. Tomita T. Kitamura M. Kurabayashi R. Nagai E. Higashihara et al. Pioglitazone improves the phenotype and molecular defects of a targeted Pkd1 mutant Hum Mol Genet 11 2002 1731-1742
72. David Woo: Treatment of cystic disease with TNF-α. May 12, 1998. Patent Number 5,750,495.
73. David Woo: Inhibition of cyst formation by cytoskeletal specific drugs. March 16, 1999. Patent Number 5,882,881.
74. Vincent Gattone: Treatment of polycystic kidney disease using vasopressin V2 receptor antagonists. October 26, 1999. Patent Number 5,972,882.
75. David Woo: Treatment of cystic disease with compounds which stimulate TNF-α production in vivo. January 4, 2000. Patent Number 6,011,055.
76. Patricia Wilson and Chrostopher Burrows: Screening methods for compounds useful in the treatment of polycystic kidney disease. October 28, 2003. Patent Number 6,638,726.
77. Mathew Breyer: Cyclooxygenase-2 inhibition in the prevention and treatment of autosomal dominant polycystic kidney disease. February 5, 2004. Patent Application 20040024042.
78. Philip Frost and Jeremy Levin: Method for the treatment of polycystic kidney disease. April 1, 2004. Patent Application 20040063672.
79. Gattone VH, Kinne Q Torres VE: Efficacy of OPC41061 in the treatment of murine nephronophthisis. J Am Soc Nephrol 2005, in press.
80. WS25/- mice. J Am Soc Nephrol 2005, in press.
81. P. Ruggenenti A. Remuzzi P. Ondei G. Fasolini L. Antiga B. Ene-Iordache G. Remuzzi F.H. Epstein Safety and efficacy of long-acting somatostatin treatment in autosomal-dominant polycystic kidney disease Kidney Int 68 2005 206-216
82. Gambineri A, Patton L, De Iasio R, Cantelli B, Cognini GE, Filicori M, Barreca A, Diamanti-Kandarakis E, Pagotto U, Pasquali R: Efficacy of octreotide-LAR in dieting women with abdominal obesity and polycystic ovary syndrome. J Clin Endocrinol Metab 2005, in press.
83. Garvey DS, Letts LG, Lin C, Richardson SK, Wang T: Nitrosated and nitrosylated taxanes, compositions and methods of use. Patent Number 6,869,973.
84. Holub B, Collins FW, Bureau DP, Philbrick DJ: Triterpene saponins from soybeans for treating kidney disease. Patent Number 6,784,159.
85. Iversen PL, Mata JE, Gattone VH: Antisense to c-myc for treatment of polycystic kidney disease. Patent Number 6,875,747.