Introduction to toxicologic pathology

Toxicologic Pathology: Nonclinical Safety Assessment

Volumn , Issue , 2013, Pages 77-96

  Markovits, Judit E ^a   Bouchard, Page R ^a   Clarke, Christopher J ^b   McMartin, Donald N ^c  

^a NOVARTIS   (United States)

^b AMGEN INC   (United States)

^c PathTox Consulting LLC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 85046847972     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1201/b13783     Document Type: Chapter

References (105)

1. Black, H. E. 1994. Design and writing of the preclinical safety report. Toxicol Pathol 22:202-5.
2. Black, H. E. 1997. The pebble in the pond. Toxicol Pathol 25:80-1.
3. Bolon, B. 2011. The world weighs in on optimal toxicologic pathology training practices: it’s unanimous! Toxicol Pathol 39:294.
4. Bolon, B., Barale-Thomas, E., Bradley, A. et al. 2010. International recommendations for future toxicologic pathologists participating in regulatory-type, non-clinical toxicity studies. Toxicol Pathol 38:984-92.
5. Bolon, B. and Galbreath, E. 2002. Use of genetically engineered mice in drug discovery and development: wielding Occam’s razor to prune the product portfolio. Intern J Toxicol 21:55-64.
6. Boorman, G. A., Anderson, S. P., Casey, W. M. et al. 2002. Toxicogenomics, drug discovery and the pathologist. Toxicol Pathol 30:15-27.
7. Boverhof, D. R., Chamberlain, M. P., Elcombe, C. R. et al. 2011. Transgenic animal models in toxicology: historical perspectives and future outlook. Toxicol Sci 121:207-33.
8. Boyce, J. T., Boyce, R. W., and Gundersen, J. 2010. Choice of morphometric methods and consequences in the regulatory environment. Toxicol Pathol 38:1128-33.
9. Boyce, R. W., Dorph-Petersen, K. A., Lyck, L. et al. 2010. Design-based stereology: introduction to basic concepts and practical approaches for estimation of cell number. Toxicol Pathol 38:1011-25.
10. Bregman, C. L., Adler, R. R., Morton, D. G. et al. 2003. Recommended tissue list for histopathological examination in repeat-dose toxicity and carcinogenicity studies: a proposal of the Society of Toxicologic Pathology (STP). Toxicol Pathol 31:252-3.
11. Brott, D. A., Gold, S., Jones, H. 2005a. Biomarkers of drug-induced vascular injury. Toxicol Appl Pharmacol 207:S441-5.
12. Brott, D. A., Jones H. B., and Gould, S. 2005b. Current status and future directions for diagnostic markers of drug-induced vascular injury. Cancer Biomarkers 1:15-28.
13. Burkhardt, J. E., Pandher, K., Solter, P. F. et al. 2011. Recommendations for the evaluation of pathology data in nonclinical safety biomarker qualification studies. Toxicol Pathol 39:1129-37.
14. Bussiere, J., Leach, M. W., Price, K. D. et al. 2011. Survey results on the use of the tissue cross-reactivity immunohistochemistry assay. Reg Toxicol Pharmacol 59:493-502.
15. Caponigro, G. and Sellers, W. R. 2011. Advances in the preclinical testing of cancer therapeutic hypothesis. Nature Rev Drug Discov 10:179-87.
16. Chamanza, R., Marxfeld, H. A., Blanco, A. I. et al. 2010. Incidences and range of spontaneous findings in control cynomolgus monkeys (Macaca fascicularis) used in toxicity studies. Toxicol Pathol 38:642-57.
17. Chen, X. M. 2009. MicroRNA signatures in liver disease. World J Gastroenterol 15:1665-72.
18. Cheng, H., Kari, G., Dicker, A. P. et al. 2011. A novel preclinical strategy for identifying cardiotoxic kinase inhibitors and mechanisms of cardiotoxicity. Circ Res 109:1401-9.
19. Crissman, J. W., Goodman, D. G., Hildebrandt, P. K. et al. 2004. Best practices guideline: toxicologic histopathology. Toxicol Pathol 32:126-31.
20. Davies, T. S. and Monro, A. 1995. Marketed human pharmaceuticals reported to be tumorigenic in rodents. Toxicol Pathol 14:90-107.
21. Dieterle, F., Sistare, F., Goodsaid, F. et al. 2010. Renal biomarker qualification submission: a dialog between the FDA-EMEA and Predictive Safety Testing Consortium. Nat Biotechnol 28:455-62.
22. Dorato, M. A., McMillan, C. L., and Vodicnik, M. J. 2008. The toxicologic assessment of pharmaceuticals and biotechnology products. In: Principles and Methods in Toxicology (A. Wallace Hayes, editor). Fifth Edition. CRC Press, Boca Raton, FL, pp. 325-68.
23. Dunstan, R. W., Wharton, K. A., Quigley, C. et al. 2011. The use of immunohistochemistry for biomarker assessment-can it compete with other technologies? Toxicol Pathol 39:988-1002.
24. Elmore, S. A. and Peddada, S. D. 2009. Points to consider on the statistical analysis of rodent cancer bioassay data when incorporating historical control data. Toxicol Pathol 37:672-6.
25. EMEA. 2000. The European Agency for the Evaluation of Medicinal Products Committee for Proprietary Medicinal Products. Note for guidance on repeated dose toxicity. http://www.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003102.pdf. (accessed March 2012).
26. Ennulat, D., Magid-Slav, M., and Rehm, S. 2010a. Diagnostic performance of traditional hepatobiliary biomarkers of drug-induced liver injury in the rat. Toxicol Sci 116:397-412.
27. Ennulat, D., Walker, D., Cierno, F. et al. 2010b. Effects of hepatic drug-metabolizing enzyme induction on clinical pathology parameters in animals and man. Toxicol Pathol 38:810-28.
28. Ettlin, R. A., Bolon, B., Pyrah, I. et al. 2008. Global recognition of qualified toxicologic pathologists: where we are now and where we need to go. Toxicol Pathol 36:753-9.
29. FDA. 2011. Draft guidance. Guidance for industry: Use of histology in biomarker qualification studies. Washington, DC. USDHEW.
30. Finch, J. M. 2005. Recent developments in preclinical toxicological pathology. Toxicol Appl Pharmacol 207:S209-13.
31. Fishman, M. C. and Porter, J. A. 2005. A new grammar for drug discovery. Nature 437:491-3.
32. Frame, S. and Mann, P. 2008. Principles of pathology for toxicology studies In: Principles and Methods in Toxicology (A. Wallace Hayes, editor). Fifth Edition. CRC Press, Boca Raton, FL, pp. 591-610.
33. Hall, W. C. and Rojko, J. L. 1996. The use of immunohistochemistry for the evaluation the liver. Toxicol Pathol 24:4-12.
34. Hardisty, J. F., Anderson, D. C., Brodie, S. et al. 2008. Histopathology of the urinary bladders of cynomolgus monkeys treated with PPAR agonists. Toxicol Pathol 36:769-76.
35. Hardisty, J. F., Elwell, M. R., Ernst, H. et al. 2007. Histopathology of hemangiosarcomas in mice and hamsters and liposarcomas/fibrosarcomas in rats associated with PPAR agonists. Toxicol Pathol 35:928-41.
36. Harpur, E., Ennulat, D., Hoffman, D. et al. 2011. Biological qualification of biomarkers of chemical-induced renal toxicity in two strains of male rat. Toxicol Sci 122:235-52.
37. Hill, A., Mesens, N., Steeemans, M. et al. 2012. Comparisons between in vitro whole cell imaging and in vivo zebrafish-based approaches for identifying potential human hepatotoxicants earlier in pharmaceutical development. Drug Metab Rev 44:127-40.
38. Holland, T. and Holland, C. 2011a. Analysis of unbiased histopathology data from rodent toxicity studies (or, are these groups different enough to ascribe it to treatment?). Toxicol Pathol 39:569-75.
39. Holland, T. and Holland, C. 2011b. Unbiased histological examinations in toxicological experiments (or, the informed leading the blinded examination). Toxicol Pathol 39:711-4.
40. Iatropoulos, M. J. and Williams, G. M. 2011. Toxicologic pathology. Society of Toxicology Historical perspective. 11-2. http://www.toxicology.org/AI/MEET/AM2011/FAST_HistoricalPerspectives.pdf (accessed March 2012).
41. Jung, J., Park, M., Park, H. J. et al. 2011. 1H NMR-based metabolic profiling of naproxen-induced toxicity in rats. Toxicol Lett 200:1-7.
42. Kaufmann, W., Nolte, T., Rittinghausen, S. et al, 2005. INHAND International Harmonization of nomenclature and diagnostic criteria for lesions in rats and mice. http://www.toxpath.org/inhand_112105.pdf (accessed March 2012).
43. Keenan, C., Elmore, S., Francke-Carroll, S. et al. 2009. Best practices for use of historical control data of proliferative rodent lesions. Toxicol Pathol 37:679-93.
44. Kerns, W., Schwartz, L., Blanchard, K. et al. 2004. Drug-induced vascular injury-a quest for biomarkers. Toxicol Appl Pharmacol 203:62-87.
45. Kittel, B., Ruehl-Fehlert, C., Morawietz, G. et al. 2004. Revised guides for organ sampling and trimming in rats and mice-part 2. A joint publication of the RITA and NACAD groups. Exp Toxicol Pathol 55:413-31.
46. Kramer, J. A., Sagartz, J. E., and Morris, D. L. 2007. The application of discovery toxicology and pathology towards the design of safer pharmaceutical lead candidates. Nat Rev Drug Discov 6:636-49.
47. Lang, P., Yeow, K., and Nichols, A. 2006. Cellular imaging in drug discovery. Nat Rev Drug Discov 5:343-56.
48. Laterza, O. F., Lim, L., Garrett-Engele, P. W. et al. 2009. Plasma microRNAs as diagnostically sensitive and specific biomarkers of tissue injury. Clin Chem 55:1977-83.
49. Leach, M. W., Halpern, W. G., Johnson, C. W. et al. 2010. Use of tissue cross-reactivity studies in the development of antibody-based biopharmaceuticals: history, experience, methodology and future directions. Toxicol Pathol 38:1138-66.
50. Lewis, R. W., Billington, R., Debryune, E. et al. 2002. Recognition of adverse and nonadverse effects in toxicity studies. Toxicol Pathol 30:66-74.
51. Liebler, D. C. and Guengerich, F. P. 2005. Elucidating mechanisms of drug-induced toxicity. Nat Rev Drug Discov 4:410-20.
52. Louden, C., Brott, D., Katein, A. et al. 2006. Biomarkers and mechanisms of drug-induced vascular injury in non-rodents. Toxicol Pathol 34:19-26.
53. Maronpot, R. R., Yoshizawa, K., Nyska, A. et al. 2010. Hepatic enzyme induction: histopathology. Toxicol Pathol 38:776-795.
54. Maziasz, T., Kadambi, V. J., Silverman, L. et al. 2010. Predictive toxicology approaches for small molecule oncology drugs. Toxicol Pathol 38:148-64.
55. McConnell, E. E., Solleveld, H. A., Swenberg, J. A. et al. 1986. Guidelines for combining neoplasms for evaluation of rodent carcinogenesis studies. J Natl Cancer Inst 76:283-9.
56. McKay, J. S., Barale-Thomas. E., Bolon, B. et al. 2010. A commentary on the process of peer review and pathology data locking. Toxicol Pathol 38:508-10.
57. Menke, A. L., Spitsbergen, J. M., Wolterbeek, A. P. M. et al. 2011. Normal anatomy and histology of the adult zebrafish. Toxicol Pathol 39:759-775.
58. Michael, B., Yano, B., Sellers, R. S. et al. 2007. Evaluation of organ weights for rodent and non-rodent toxicity studies: a review of regulatory guidelines and survey of current practices. Toxicol Pathol 35:742-50.
59. Morawietz, G., Ruehl-Fehlert, C., Kittel, B. et al. 2004. Revised guides for organ sampling and trimming in rats and mice-part 2. A joint publication of the RITA and NACAD groups. Exp Toxicol Pathol 55:433-49.
60. Morton, D., Elwell, M., Fairweather, W. et al. 2002. The Society of Toxicologic Pathology’s recommendations on statistical analysis of rodent carcinogenicity studies. Toxicol Pathol 30:415-8.
61. Morton, D., Sellers, R. S., Barale-Thomas, E. et al. 2010. Recommendations for pathology peer review. Toxicol Pathol 38:1118-27.
62. Mullard, A. 2011. 2010 FDA drug approvals. Nat Rev Drug Discov. 10:82-5.
63. Muller, P. Y., Milton, M., Lloyd, P. et al. 2009. The minimum anticipated biological effect level (MABEL) for selection of first in human dose in clinical trials with monoclonal antibodies. Curr Opin Biotechnol 20:1-8.
64. Munos, B. 2009. Lessons from 60 years of pharmaceutical innovation. Nat Rev Drug Discov 8:965-8.
65. Nolte, T., Kaufman, W., Schorsh, F. et al. 2005. Standardized assessment of cell proliferation: the approach of RITA-CEPA working group. Exp Toxicol Pathol 57:91-103.
66. Ochoa, R. and Rousseaux, C. 2009. The role of toxicologic pathologist in risk management. Toxicol Pathol 37:705-6.
67. Olson, H., Betton, G., Robinson, D. et al. 2000. Concordance of the toxicity of pharmaceuticals in humans and in animals. Regul Toxicol Pharmacol 32:56-67.
68. Painter, J. T., Clayton, N. P., and Herbert, R. A. 2010. Useful immunohistochemistry markers of tumor differentiation. Toxicol Pathol 38:131-41.
69. Pandher, K., Leach, M. W., Burns-Naas, L. A. 2012. Appropriate use of recovery groups in nonclinical toxicity studies: value in a science-driven case-by-case approach. Vet Pathol 49:357-61.
70. Peto, R., Pike, M. C., Day, N. E. et al, 1980. Guidelines for simple sensitive significance tests for carcinogenic effects in long-term animal experiments. In: IARC Monographs on the Evaluation of the Carcinogenic Risk of Chemicals to Humans, upplement 2: Long-term and Short-term Screening Assays for Carcinogens: A Critical Appraisal. Lyon: International Agency for Research on Cancer, pp. 311-346
71. Ploemen, J. P. H. T. M., Kramer, H., Krajnc, E. I. et al. 2007. The use of toxicokinetic data in preclinical safety assessment: a toxicologic pathologist perspective. Toxicol Pathol 35:834-7.
72. Ponce, R. 2011. ICH S9: developing anticancer drugs, one year later. Toxicol Pathol 39:913-5.
73. Reddy, M. V., Sistare, F. D., Christensen, J. S. et al. 2010. An evaluation of chronic 6-and 12-month rat toxicology studies as predictors of 2-year tumor outcome. Toxicol Pathol 47:614-29.
74. Revised guides for organ sampling and trimming in rats and mice. http://reni.item.fraunhofer.de/reni/trimming/index.php (accessed January 2012).
75. RITA. http://reni.item.fraunhofer.de/reni/public/rita/ (accessed January 2012).
76. Rojas, C., Stathis, M., Polydefkis, M. et al. 2011. Glutamate carboxypeptidase activity in human skin biopsies as a pharmacodynamic marker for clinical studies. J Transl Med 9:27-34.
77. Roman, D., Greiner, B., Ibrahim, M. et al. 2002. Laser technologies in toxicopathology. Toxicol Pathol 30:11-14.
78. Ross, J. S. and Ginsburg, G. S. 2003. The integration of molecular diagnostics with therapeutics. Implication for drug development and pathology practice. Am J Clin Pathol 119:26-36.
79. Ruehl-Fehlert, C., Kittel, B., Morawietz, G. et al. 2003. Revised guides for organ sampling and trimming in rats and mice-part 1. Exp Toxicol Pathol 55:91-106.
80. Sarker, D., Pacey, S., and Workman, P. 2007. Use of pharmacokinetic/pharmacodynamic biomarkers to support rational cancer drug development. Biomarkers Med 1:399-417.
81. Sasseville, V. G., Lane, J. H., Kadambi, V. J. et al. 2004. Testing paradigm for prediction of development-limiting barriers and human drug toxicity. Chem Biol Interact 150:9-25.
82. Schofield, P. N., Dubus, P., Klein, L. et al. 2011. Pathology of the laboratory mouse: an international workshop on challenges for high throughput phenotyping. Toxicol Pathol 39:559-62.
83. Schultze, A. E., Bounous, D. I., and Provencher Bollinger, A. 2008. Veterinary clinical pathologists in the biopharmaceutical industry. Vet Clin Pathol 37:146-58.
84. Sellers, R. S., Morton, D., Michael, B. et al. 2007. Society of Toxicologic Pathology position paper: organ weight recommendations for toxicology studies. Toxicol Pathol 35:751-5.
85. Shackelford, C., Long, G., Wolf, J. et al. 2002. Qualitative and quantitative analysis of nonneoplastic lesions in toxicology studies. Toxicol Pathol 30:93-6.
86. Sistare, F., Dieterle, F., Troth, S. et al. 2010. Toward consensus practices to qualify safety biomarkers for use in early drug development. Nat Biotechnol 28:446-454.
87. Sistare, F. D., Morton, D., Alden, C. et al, 2011. An analysis of pharmaceutical experience with decades of rat carcinogenicity testing: support for a proposal to modify current regulatory guidelines.
88. Squire, R. A. 1997. A quarter century of toxicologic pathology: a personal perspective. Toxicol Pathol 25:423-5.
89. Standardized system of nomenclature and diagnostic criteria (SSNDC) guides. http://www.toxpath.org/ssndc.asp (accessed January 2012).
90. Stevens, J. L. and Baker, T. K. 2009. The future of drug safety testing: expanding the view and narrowing the focus. Drug Discov Today 14:162-7.
91. Swinney, D. C. and Anthony, J. 2011. How were new medicines discovered? Nat Rev Drug Discov 10:507-19.
92. Tesfamariam, B. and DeFelice, A. F. 2007. Endothelial injury in the initiation and progression of vascular disorders. Vasc Pharmacol 46:229-37.
93. Vahle, J. L., Finch, G. L., Heidel, S. M. et al. 2010. Carcinogenicity assessments of biotechnology-derived pharmaceuticals: a review of approved molecules and best practice recommendations. Toxicol Pathol 38:522-53.
94. Vet Pathol 2012. Special focus: phenotyping of genetically engineered mice. 49(1):4-235.
95. Wang, J., Urban, L., and Bojanic, D. 2007. Maximising use of in vitro ADMET tools to predict in vivo bioavailability and safety. Expert Opin Drug Metab Toxicol 3:641-65.
96. Wang, K., Zhang, S., Marzolf, B. et al. 2009. Circulating microRNAs, potential biomarkers for drug-induced liver injury. Proc Natl Acad Sci USA 106:4402-7.
97. Ward, J. M. 2010. The roles of the toxicologic pathologist in cancer research. Toxicol Pathol 38:39-42.
98. Ward, J. M., Erexson, C. R., Faucette, L. J. et al. 2006. Immunohistochemical markers for the rodent immune system. Toxicol Pathol 34:616-30.
99. Ward, J. M. and Thoolen, B. 2011. Grading of lesions. Toxicol Pathol 39:745-6.
100. Weingand, K., Brown, G., Hall, R. et al. 1996. Harmonization of animal clinical pathology testing in toxicity and safety studies. Fundam Appl Toxicol 29:198-201
101. Wetmore, B. A. and Merrick, B. A. 2004. Invited review: toxicoproteomics: proteomics applied to toxicology and pathology. Toxicol Pathol 32:619-42.
102. Wolf, D. C. and Mann, P. 2005. Confounders in interpreting pathology for safety and risk assessment. Toxicol Appl Pharmacol 202:302-8.
103. Ying, X. and Monticello, T. 2006. Modern imaging technologies in toxicologic pathology: an overview. Toxicol Pathol 34:815-26.
104. Young, J., Hall, R. L., O’Brien, P. et al. 2011. Best practices for clinical pathology testing in carcinogenicity studies. Toxicol Pathol 39:429-34.
105. Zhou, Y., Rideout, W. M., Zi, T. et al. 2010. Chimeric mouse tumor models reveal differences in pathway activation between ERBB family-and KRAS-dependent lung adenocarcinomas. Nat Biotechnol 28:71-8.