Small-molecule proteostasis regulators for protein conformational diseases

Nature Chemical Biology

Volumn 8, Issue 2, 2012, Pages 185-196

  Calamini, Barbara ^a   Silva, Maria Catarina ^a,b   Madoux, Franck ^c   Hutt, Darren M ^d   Khanna, Shilpi ^e   Chalfant, Monica A ^d   Saldanha, S Adrian ^c   Hodder, Peter ^c   Tait, Bradley D ^e   Garza, Dan ^e   Balch, William E ^d   Morimoto, Richard I ^a  

^a NORTHWESTERN UNIVERSITY   (United States)

^b UNIVERSITY OF LISBON   (Portugal)

^c SCRIPPS RESEARCH INSTITUTE   (United States)

^d SCRIPPS RESEARCH INSTITUTE   (United States)

^e Proteostasis Therapeutics Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BENZYLOXYCARBONYLLEUCYLLEUCYLLEUCINAL; CADMIUM CHLORIDE; CELASTROL; CHAPERONE; HEAT SHOCK PROTEIN 90; HEAT SHOCK TRANSCRIPTION FACTOR 1; PROTEASOME; PROTEIN INHIBITOR; PROTEOME; SMALL MOLECULE PROTEOSTASIS REGULATOR; TRANSCRIPTION FACTOR FOXO; TRANSCRIPTION FACTOR NRF2; UNCLASSIFIED DRUG;

ANIMAL CELL; APOPTOSIS; ARTICLE; CAENORHABDITIS ELEGANS; CELL STRESS; CONTROLLED STUDY; DRUG SCREENING; DRUG STRUCTURE; GENE ACTIVITY; HIGH THROUGHPUT SCREENING; HUMAN; HUMAN CELL; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN AGGREGATION; PROTEIN CONFORMATION; PROTEIN EXPRESSION; PROTEIN FOLDING; PROTEIN HOMEOSTASIS; PROTEIN INDUCTION; PROTEIN STABILITY; QUALITY CONTROL;

EID: 84856089134     PISSN: 15524450     EISSN: 15524469     Source Type: Journal    
DOI: 10.1038/nchembio.763     Document Type: Article

References (50)

1. Balch, W.E., Morimoto, R.I., Dillin, A. & Kelly, J.W. Adapting proteostasis for disease intervention. Science 319, 916-919 (2008). (Pubitemid 351263754)
2. Powers, E.T., Morimoto, R.I., Dillin, A., Kelly, J.W. & Balch, W.E. Biological and chemical approaches to diseases of proteostasis deficiency. Annu. Rev. Biochem. 78, 959-991 (2009).
3. Morimoto, R.I. Proteotoxic stress and inducible chaperone networks in neurodegenerative disease and aging. Genes Dev. 22, 1427-1438 (2008). (Pubitemid 351793077)
4. Ron, D. & Walter, P. Signal integration in the endoplasmic reticulum unfolded protein response. Nat. Rev. Mol. Cell Biol. 8, 519-529 (2007). (Pubitemid 46985379)
5. Haynes, C.M. & Ron, D. The mitochondrial UPR -protecting organelle protein homeostasis. J. Cell Sci. 123, 3849-3855 (2010).
6. Lichtlen, P. & Schaffner, W. Putting its fingers on stressful situations: the heavy metal-regulatory transcription factor MTF-1. Bioessays 23, 1010-1017 (2001).
7. Vargas, M.R. & Johnson, J.A. The Nrf2-ARE cytoprotective pathway in astrocytes. Expert Rev. Mol. Med. 11, e17 (2009).
8. Åkerfelt, M., Morimoto, R.I. & Sistonen, L. Heat shock factors: integrators of cell stress, development and lifespan. Nat. Rev. Mol. Cell Biol. 11, 545-555 (2010).
9. Young, J.C., Agashe, V.R., Siegers, K. & Hartl, F.U. Pathways of chaperone-mediated protein folding in the cytosol. Nat. Rev. Mol. Cell Biol. 5, 781-791 (2004). (Pubitemid 39336272)
10. Gidalevitz, T., Ben-Zvi, A., Ho, K.H., Brignull, H.R. & Morimoto, R.I. Progressive disruption of cellular protein folding in models of polyglutamine diseases. Science 311, 1471-1474 (2006). (Pubitemid 43376703)
11. Gidalevitz, T., Kikis, E.A. & Morimoto, R.I. A cellular perspective on conformational disease: the role of genetic background and proteostasis networks. Curr. Opin. Struct. Biol. 20, 23-32 (2010).
12. Sittler, A. et al. Geldanamycin activates a heat shock response and inhibits huntingtin aggregation in a cell culture model of Huntington's disease. Hum. Mol. Genet. 10, 1307-1315 (2001). (Pubitemid 32600484)
13. Muchowski, P.J. & Wacker, J.L. Modulation of neurodegeneration by molecular chaperones. Nat. Rev. Neurosci. 6, 11-22 (2005). (Pubitemid 40052135)
14. Fujimoto, M. et al. Active HSF1 significantly suppresses polyglutamine aggregate formation in cellular and mouse models. J. Biol. Chem. 280, 34908-34916 (2005). (Pubitemid 41504625)
15. Vacher, C., Garcia-Oroz, L. & Rubinsztein, D.C. Overexpression of yeast hsp104 reduces polyglutamine aggregation and prolongs survival of a transgenic mouse model of Huntington's disease. Hum. Mol. Genet. 14, 3425-3433 (2005). (Pubitemid 41672126)
16. Waza, M. et al. 17-AAG, an Hsp90 inhibitor, ameliorates polyglutamine-mediated motor neuron degeneration. Nat. Med. 11, 1088-1095 (2005). (Pubitemid 41486831)
17. Zhang, Y.Q. & Sarge, K.D. Celastrol inhibits polyglutamine aggregation and toxicity though induction of the heat shock response. J. Mol. Med. 85, 1421-1428 (2007). (Pubitemid 350234097)
18. Fujikake, N. et al. Heat shock transcription factor 1-activating compounds suppress polyglutamine-induced neurodegeneration through induction of multiple molecular chaperones. J. Biol. Chem. 283, 26188-26197 (2008).
19. Hsu, A.L., Murphy, C.T. & Kenyon, C. Regulation of aging and age-related disease by DAF-16 and heat-shock factor. Science 300, 1142-1145 (2003). (Pubitemid 36583098)
20. Neef, D.W., Turski, M.L. & Thiele, D.J. Modulation of heat shock transcription factor 1 as a therapeutic target for small molecule intervention in neurodegenerative disease. PLoS Biol. 8, e1000291 (2010).
21. Westerheide, S.D. & Morimoto, R.I. Heat shock response modulators as therapeutic tools for diseases of protein conformation. J. Biol. Chem. 280, 33097-33100 (2005). (Pubitemid 41397104)
22. Taldone, T., Gozman, A., Maharaj, R. & Chiosis, G. Targeting Hsp90: small-molecule inhibitors and their clinical development. Curr. Opin. Pharmacol. 8, 370-374 (2008).
23. Luo, W., Sun, W., Taldone, T., Rodina, A. & Chiosis, G. Heat shock protein 90 in neurodegenerative diseases. Mol. Neurodegener. 5, 24 (2010).
24. Solit, D.B. et al. Phase II trial of 17-allylamino-17- demethoxygeldanamycin in patients with metastatic melanoma. Clin. Cancer Res. 14, 8302-8307 (2008).
25. Westerheide, S.D. et al. Celastrols as inducers of the heat shock response and cytoprotection. J. Biol. Chem. 279, 56053-56060 (2004). (Pubitemid 40066613)
26. Williams, G.T. & Morimoto, R.I. Maximal stress-induced transcription from the human HSP70 promoter requires interactions with the basal promoter elements independent of rotational alignment. Mol. Cell. Biol. 10, 3125-3136 (1990).
27. Butina, D. Unsupervised data base clustering based on Daylight's fingerprint and Tanimoto similarity: a fast and automated way to cluster small and large data sets. J. Chem. Inf. Comput. Sci. 39, 747-750 (1999).
28. Amici, C., Sistonen, L., Santoro, M.G. & Morimoto, R.I. Antiproliferative prostaglandins activate heat shock transcription factor. Proc. Natl. Acad. Sci. USA 89, 6227-6231 (1992).
29. Trott, A. et al. Activation of heat shock and antioxidant responses by the natural product celastrol: transcriptional signatures of a thiol-targeted molecule. Mol. Biol. Cell 19, 1104-1112 (2008). (Pubitemid 351481823)
30. Li, C. et al. Pharmacologic induction of heme oxygenase-1. Antioxid. Redox Signal. 9, 2227-2239 (2007). (Pubitemid 350059011)
31. Liu, Y. & Chang, A. Heat shock response relieves ER stress. EMBO J. 27, 1049-1059 (2008). (Pubitemid 351508152)
32. Wyttenbach, A. et al. Polyglutamine expansions cause decreased CRE-mediated transcription and early gene expression changes prior to cell death in an inducible cell model of Huntington's disease. Hum. Mol. Genet. 10, 1829-1845 (2001). (Pubitemid 32899099)
33. Riordan, J.R. CFTR function and prospects for therapy. Annu. Rev. Biochem. 77, 701-726 (2008).
34. Morley, J.F., Brignull, H.R., Weyers, J.J. & Morimoto, R.I. The threshold for polyglutamine-expansion protein aggregation and cellular toxicity is dynamic and influenced by aging in Caenorhabditis elegans. Proc. Natl. Acad. Sci. USA 99, 10417-10422 (2002).
35. Nollen, E.A. et al. Genome-wide RNA interference screen identifies previously undescribed regulators of polyglutamine aggregation. Proc. Natl. Acad. Sci. USA 101, 6403-6408 (2004). (Pubitemid 38585986)
36. Garcia, S.M., Casanueva, M.O., Silva, M.C., Amaral, M.D. & Morimoto, R.I. Neuronal signaling modulates protein homeostasis in Caenorhabditis elegans post-synaptic muscle cells. Genes Dev. 21, 3006-3016 (2007). (Pubitemid 350133446)
37. Whitesell, L. & Lindquist, S.L. HSP90 and the chaperoning of cancer. Nat. Rev. Cancer 5, 761-772 (2005). (Pubitemid 41400776)
38. Stringer, J.R., Bowman, M.D., Weisblum, B. & Blackwell, H.E. Improved small-molecule macroarray platform for the rapid synthesis and discovery of antibacterial chalcones. ACS Comb. Sci. 13, 175-180 (2011).
39. Yadav, V.R., Prasad, S., Sung, B. & Aggarwal, B.B. The role of chalcones in suppression of NF-?B-mediated inflammation and cancer. Int. Immunopharmacol. 11, 295-309 (2010).
40. Dinkova-Kostova, A.T., Massiah, M.A., Bozak, R.E., Hicks, R.J. & Talalay, P. Potency of Michael reaction acceptors as inducers of enzymes that protectagainst carcinogenesis depends on their reactivity with sulfhydryl groups. Proc. Natl. Acad. Sci. USA 98, 3404-3409 (2001). (Pubitemid 32220858)
41. Dinkova-Kostova, A.T. et al. Direct evidence that sulfhydryl groups of Keap1 are the sensors regulating induction of phase 2 enzymes that protect against carcinogens and oxidants. Proc. Natl. Acad. Sci. USA 99, 11908-11913 (2002). (Pubitemid 34994497)
42. Tangallapally, R.P. et al. Synthesis and evaluation of nitrofuranylamides as novel antituberculosis agents. J. Med. Chem. 47, 5276-5283 (2004). (Pubitemid 39314926)
43. Roesslein, M. et al. Thiopental protects human T lymphocytes from apoptosis in vitro via the expression of heat shock protein 70. J. Pharmacol. Exp. Ther. 325, 217-225 (2008). (Pubitemid 351439174)
44. Yang, H. et al. Nanomolar affinity small molecule correctors of defective ? F508-CFTR chloride channel gating. J. Biol. Chem. 278, 35079-35085 (2003). (Pubitemid 37102270)
45. Yang, F. et al. Curcumin inhibits formation of amyloid β oligomers and fibrils, binds plaques, and reduces amyloid in vivo. J. Biol. Chem. 280, 5892-5901 (2005). (Pubitemid 40280072)
46. Teiten, M.H., Reuter, S., Schmucker, S., Dicato, M. & Diederich, M. Induction of heat shock response by curcumin in human leukemia cells. Cancer Lett. 279, 145-154 (2009).
47. Mu, T.W. et al. Chemical and biological approaches synergize to ameliorate protein-folding diseases. Cell 134, 769-781 (2008).
48. Parenti, G. Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics. EMBO Mol. Med. 1, 268-279 (2009).
49. Gorbatyuk, M.S. et al. Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78. Proc. Natl. Acad. Sci. USA 107, 5961-5966 (2010).
50. Tam, L.C. et al. Prevention of autosomal dominant retinitis pigmentosa by systemic drug therapy targeting heat shock protein 90 (Hsp90). Hum. Mol. Genet. 19, 4421-4436 (2010).