Retinal toxicity, in vivo and in vitro, associated with inhibition of nicotinamide phosphoribosyltransferase

Toxicological Sciences

Volumn 144, Issue 1, 2015, Pages 163-172

  Zabka, Tanja S ^a   Singh, Jatinder ^a   Dhawan, Preeti ^a   Liederer, Bianca M ^a   Oeh, Jason ^a   Kauss, Mara A ^a   Xiao, Yang ^a   Zak, Mark ^a   Lin, Tori ^a   McCray, Bobbi ^a   La, Nghi ^a   Nguyen, Trung ^a   Beyer, Joseph ^a   Farman, Cynthia ^a   Uppal, Hirdesh ^a   Dragovich, Peter S ^a   O'Brien, Thomas ^a   Sampath, Deepak ^a   Misner, Dinah L ^a  

^a GENENTECH INC   (United States)

Author keywords

Eye; Messenger rna; Nicotinamide adenine dinucleotide; Nicotinic acid mononucleotide; Pathology; Retinal degeneration; Retinal pigmented epithelial cell culture; Tumor metabolism

Indexed keywords

MESSENGER RNA; NICOTINAMIDE NUCLEOTIDE ADENYLYLTRANSFERASE; NICOTINAMIDE PHOSPHORIBOSYLTRANSFERASE; NICOTINATE PHOSPHORIBOSYLTRANSFERASE; PENTOSYLTRANSFERASE INHIBITOR; CYANIDE; CYTOKINE; ENZYME INHIBITOR; FUSED HETEROCYCLIC RINGS; GLYCOSYLTRANSFERASE; GNE-617; GUANIDINE DERIVATIVE; N-(6-CHLOROPHENOXYHEXYL)-N''-CYANO-N''-4-PYRIDYLGUANIDINE; NICOTINAMIDE PHOSPHORIBOSYLTRANSFERASE, HUMAN; NICOTINAMIDE PHOSPHORIBOSYLTRANSFERASE, MOUSE; NICOTINAMIDE PHOSPHORIBOSYLTRANSFERASE, RAT; NICOTINIC ACID; SULFONE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; ENZYME INHIBITION; ENZYME SYNTHESIS; EPITHELIUM CELL; EYE TOXICITY; FEMALE; GENE EXPRESSION; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; RAT; RETINA; ANIMAL; ANTAGONISTS AND INHIBITORS; CELL LINE; CELL SURVIVAL; CHEMICAL STRUCTURE; CHEMISTRY; DRUG EFFECTS; ENZYMOLOGY; GENE EXPRESSION REGULATION; GENETICS; MALE; METABOLISM; NUDE MOUSE; PATHOLOGY; PIGMENT EPITHELIUM; RISK ASSESSMENT; SPECIES DIFFERENCE; SPRAGUE DAWLEY RAT; STRUCTURE ACTIVITY RELATION;

ANIMALIA; RATTUS; RODENTIA;

ANIMALS; CELL LINE; CELL SURVIVAL; CYANIDES; CYTOKINES; ENZYME INHIBITORS; FEMALE; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GUANIDINES; HETEROCYCLIC COMPOUNDS, 2-RING; HUMANS; MALE; MICE, NUDE; MOLECULAR STRUCTURE; NIACIN; NICOTINAMIDE PHOSPHORIBOSYLTRANSFERASE; NICOTINAMIDE-NUCLEOTIDE ADENYLYLTRANSFERASE; PENTOSYLTRANSFERASES; RATS, SPRAGUE-DAWLEY; RETINAL PIGMENT EPITHELIUM; RISK ASSESSMENT; RNA, MESSENGER; SPECIES SPECIFICITY; STRUCTURE-ACTIVITY RELATIONSHIP; SULFONES;

EID: 84924453810     PISSN: 10966080     EISSN: 10960929     Source Type: Journal    
DOI: 10.1093/toxsci/kfu268     Document Type: Article

References (29)

1. Bair, K., Baumeister, T. R., Dragovich, P., Gosselin, F., Yuen, P. W., Zak, M., and Zheng, X. (2013). Preparation of pyridinyl and pyrimidinyl sulfoxide and sulfone derivatives as NAMPT inhibitors. WO2013127267.
2. Burgos, E. S. (2011). NAMPT in regulated NAD biosynthesis and its pivotal role in human metabolism. Curr. Med. Chem. 18, 1947-1961.
3. Cerna, D., Li, H., Flaherty, S., Takebe, N., Coleman, C. N., and Yoo, S. S. (2012). Inhibition of nicotinamide phosphoribosyltransferase (NAMPT) activity by small molecule GMX1778 regulates reactive oxygen species (ROS)-mediated cytotoxicity in a p53- and nictonic acid phosphoribosyltransferase1 (NAPRT1)-dependent manner. J. Biol. Chem. 287, 22408-22417.
4. Cideciyana, A. V., Jacobson, S. G., Beltran, W. A., Sumaroka, A., Swider, M., Iwabe, S., Roman, A. J., Olivares, M. B., Schwartz, S. B., Komáromy, A. M., Hauswirth, W. W., and Aguirre, G. D. (2013). Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. PNAS. 110, E517-E525.
5. Falk, M. J., Zhang, Q., Nakamaru-Ogiso, E., Kannabiran, C., Fonseca-Kelly, Z., Chakarova, C., Audo, I., Mackay, D. S., Zeitz, C., Borman, A. D., et al. (2012). NMNAT1 mutations cause Leber congenital amaurosis. Nat. Genet. 44, 1040-1045.
6. Hasmann, M., and Schemainda, I. (2003). FK866, a highly specific noncompetitive inhibitor of nicotinamide phosphoryltransferase, represents a novel mechanism for induction of tumor cell apoptosis. Cancer Res. 63, 7436-7442.
7. Holen, K., Saltz, L. B., Hollywood, E., Burk, K., and Hanauske, A. R. (2008). The pharmacokinetics, toxicities, and biologic effects of FK866, a nicotinamide adenine dinucleotide biosynthesis inhibitor. Investigational New Drugs 26, 45-51.
8. Hovstadius, P., Larsson, R., Jonsson, E., Skov, T., Kissmeyer, A. M., Krasilnikoff, K., Bergh, J., Karlsson, M. O., Lonnebo, A., and Ahlgren, J. (2002). A Phase I study of CHS 828 in patients with solid tumor malignancy. Clin. Cancer Res. 8, 2843-2850.
9. Kaneko, A. (1979). Physiology of the retina. Ann. Rev. Neurosci. 2, 169-191.
10. Koenekoop, R. K., Wang, H., Majewski, J., Wang, X., Lopez, I., Ren, H., Chen, Y., Li, Y., Fishman, G. A., Genead, M., et al.; Finding of Rare Disease Genes (FORGE) Canada Consortium Poulter, J. A., Mohamed, M. D., Jafri, H., Rashid, Y., Taylor, G. R., Keser, V., Mardon, G., Xu, H., Inglehearn, C. F., Fu, Q., et al. (2012). Mutations in NMNAT1 cause Leber congenital amaurosis and identify a new disease pathway for retinal degeneration. Nat. Genet. 44, 1035-1039.
11. O'Brien, T., Oeh, J., Xiao, Y., Liang, X., Vanderbilt, A., Qin, A., Yang, L., Lee, L. B., Ly, J., Cosino, E., et al. (2013). Supplementation of nicotonic acid with NAMPT inhibitors results in loss of in vivo efficacy in NAPRT1-deficient tumor models. Neoplasia 15, 1314-1329.
12. Oh, A., Ho, Y. C., Zak, M., Yongbo, L., Chen, X., Yuen, P., Zheng X., Liu, Y., Dragovich, P. S., and Wang, W. (2014). Structural and biochemical analyses of the catalysis and potency impact of inhibitor phosphoribosylation by human nicotinamide phosphoribosyltransferase. ChemBioChem 15, 1121-1131.
13. Olesen, U. H., Thougaard, A. V., Jensen, P. B., and Sehested, M. (2010). A preclinical study on the rescue of normal tissue by nicotinic acid in high-dose treatment with APO866, a specific nicotinamide phosphoribosyltransferase inhibitor. Mol. Cancer Ther. 9, 1609-1617.
14. Perrault, I, Hanein, S., Zanlonghi, X., Serre, V., Nicouleau, M., Defoort-Delhemmes, S., Delphin, N., Fares-Taie, L., Gerber, S., Xerri, O., et al. (2012). Mutations in NMNAT1 cause Leber congenital amaurosis with early-onset severe macular and optic atrophy. Nat. Genet. 44, 975-977.
15. Phan, V. T., Wu, X., Cheng, J. H., Sheng, R. X., Chung, A. S., Zhuang, G., Tran, C., Song, Q., Kowanetz, M., Sambrone, A., et al. (2013). Oncogenic RAS pathway activation promotes resistance to anti-VEGF therapy through G-CSF-induced neutrophil recruitment. Proc. Natl. Acad. Sci. U.S.A. 110, 6079-6084.
16. Pishvaian, M. J., Marshall, J. L., Hwang, J. J., Malik, S., He, A. R., Deeken, J. F., Kelso, C. B., Cotarla, I., and Berger, M. S. (2009). A phase I trial of GMX1777, an inhibitor of nicotinamide phosphoribosyl transferase (NAMPRT), given as a 24-hour infusion. J. Clin. Oncol. 27, A3581.
17. Ravaud, A., Cerny, T., Terret, C., Wanders, J., Nguyen Bui, B, Hess, D., Droz, J.-P., Fumoleau, P., and Twelves, C. (2005). Phase I study and pharmacokinetic of CHS-828, a guanidinocontaining compound, administered orally as a single dose every 3 weeks in solid tumours: an ECSG/EORTC study. Eur. J. Cancer 41, 702-707.
18. Schwartz, S. G., and Mieler, W. F. (2002). Medications and retinal toxicity. Ophthalmol. Clin. N. Am. 15, 517-528.
19. Shames, D. S., Elkins, K., Walter, K., Holcomb, T., Du, P., Mohl, D., Xiao, Y., Pham, T., Haverty, P. M., Liederer, B., et al. (2013). Loss of NAPRT1 expression by tumor-specific promoter methylation provides a novel predictive biomarker for NAMPT inhibitors. Clin. Cancer Res. 19, 6912-6923.
20. Tarrant, J. M., Dhawan, P., Singh, J., Zabka, T. S., Clarke, E., DosSantos, G., Dragovich, P. S., Sampath, D., Lin, T., McCray, B., La, N., Nguyen, T., Kauss, M., Dambach, D., Misner, D. L., Diaz, D., and Uppal, H. (2014). Qualification of the Megakaryocyte Colony Forming Cell assay as an in vitro Model of Nicotinamide Phosphoribosyltransferase Inhibitormediated Clinical Thrombocytopenia and Mitigation by Co-Treatment with Nicotinic Acid. Forthcoming.
21. von Heideman, A., Berglund, A., Larsson, R., and Nygren, P. (2010). Safety and efficacy of NAD depleting cancer drugs:results of a phase I clinical trial of CHS 828 and overview of published data. Cancer Chemo. Pharm. 65, 1165-1172.
22. Ward, P. S., and Thompson C. B. (2012). Metabolic reprogramming:a cancer hallmark even Warburg did not anticipate. Cancer Cell 21, 297-308.
23. Watson, M., Roulston, A., Belec, L., Billot, X., Marcellus, R., Bedard, D., Bernier, C., Branchaud, S., Chan, H., Dairi, K., et al. (2009). The small molecule GMX1778 is a potent inhibitor of NAD{thorn} biosynthesis: strategy for enhanced therapy in nicotinic acid phosphoribosyltransferase 1-deficient tumors. Mol. Cell. Biol. 29, 5872-5888.
24. Xiao, Y., Elkins, K., Durieux, J. K., Lee, L., Oeh, J., Yang, L. X., Liang, X., Delnagro, C., Tremayne, J., Kwong, M., et al. (2013). Dependence of tumor cell lines and patient-derived tumors on the NAD salvage pathway renders them sensitive to NAMPT inhibition with GNE-618. Neoplasia 15, 1151-1160.
25. Ying, W. (2008). NAD{thorn}/NADH and NADP{thorn}/NADPH in cellular functions and cell death: regulation and biological consequences. Antioxid. Redox. Signal. 10, 179-206.
26. Zhang, L. Q., Heruth, D. P., and Ye, S. Q. (2011). Nicotinamide phosphoribosylatransferase in human diseases. J. Bioanal. Biomed. 3, 13-25.
27. Zheng, X., Bauer, P., Baumeister, T., Buckmelter, A. J., Caligiuri, M., Clodfelter, K. H., Han, B., Ho, Y. C., Kley, N., Lin, J., et al. (2013a). Structure-based discovery of novel amide-containing nicotinamide phosphoribosyltransferase (nampt) inhibitors. J. Med. Chem. 56, 6413-6433.
28. Zheng, X., Bair, K. W., Bauer, P., Baumeister, T., Bowman, K. K., Buckmelter, A. J., Caligiuri, M., Clodfelter, K. H., Feng, Y., Han, B., et al. (2013b). Identification of amides derived from 1Hpyrazolo[ 3,4-b]pyridine-5-carboxylic acid as potent inhibitors of human nicotinamide phosphoribosyltransferase (NAMPT). Bioorg. Med. Chem. Lett. 2, 5488.
29. Zhou, D., Liu, Y., Ye, J., Ying, W., Zhang, S., Ogawa, L. S., Inoue, T., Tatsuta, N., Yumiko, W., Sonderfan, A., and Koya, K. (2011). A critical role for the tissue distribution profile in heat shock protein (Hsp) 90 inhibitor-induced ocular toxicity in rats. Mol. Cancer Ther. 10(Suppl. 1), 11.