Open access target validation is a more efficient way to accelerate drug discovery

PLoS Biology

Volumn 13, Issue 6, 2015, Pages 1-9

  Lee, Wen Hwa ^a  

^a UNIVERSITY OF OXFORD   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

HISTONE DEACETYLASE;

ARTICLE; CLINICAL TRIAL (TOPIC); COST; DRUG INDUSTRY; DRUG PROGRAM; DRUG RESEARCH; DRUG TARGETING; EPIGENETICS; FUNDING; HUNTINGTON CHOREA; LICENSING; OSSIFYING MYOSITIS; PROTEIN FAMILY; PROTEIN TARGETING; DRUG DEVELOPMENT; INFORMATION DISSEMINATION; MASS COMMUNICATION; MEDICAL RESEARCH; OFF LABEL DRUG USE;

BIOMEDICAL RESEARCH; DIFFUSION OF INNOVATION; DRUG DISCOVERY; INFORMATION DISSEMINATION; OFF-LABEL USE;

EID: 85016650523     PISSN: 15449173     EISSN: 15457885     Source Type: Journal    
DOI: 10.1371/journal.pbio.1002164     Document Type: Article

References (44)

1. Munos B. Lessons from 60 years of pharmaceutical innovation. Nat Rev Drug Discov. 2009 Dec; 8 (12):959-68. doi: 10. 1038/nrd2961 PMID: 19949401
2. Bunnage ME. Getting pharmaceutical R&D back on target. Nat Chem Biol. 2011 Jun; 7(6):335-9. doi: 10. 1038/nchembio. 581 PMID: 21587251
3. Abrams I, Leung G, Stevens AJ. How are U. S. technology transfer offices tasked and motivated: is it all about the money? Research Management Review. 2009; 17(1):1-34.
4. Heller MA, Eisenberg RS. Can patents deter innovation? The anticommons in biomedical research. Science. 1998 May 1; 280(5364):698-701. PMID: 9563938
5. Chesbrough H. The logic of open innovation: Managing intellectual property. Calif Manage Rev. 2003 Spr; 45(3):33.
6. Chesbrough HW. The era of open innovation. Mit Sloan Manage Rev. 2003 Spr; 44(3):35-41.
7. Sachidanandam R, Weissman D, Schmidt SC, Kakol JM, Stein LD, Marth G, et al. A map of human genome sequence variation containing 1. 42 million single nucleotide polymorphisms. Nature. 2001 Feb 15; 409(6822):928-33. PMID: 11237013
8. The International HapMap Project. Nature. 2003 Dec 18; 426(6968):789-96. PMID: 14685227
9. Robertson MN, Ylioja PM, Williamson AE, Woelfle M, Robins M, Badiola KA, et al. Open source drug discovery-a limited tutorial. Parasitology. 2014 Jan; 141(1):148-57. doi: 10. 1017/ S0031182013001121 PMID: 23985301
10. Gileadi O, Knapp S, Lee WH, Marsden BD, Muller S, Niesen FH, et al. The scientific impact of the Structural Genomics Consortium: a protein family and ligand-centered approach to medically-relevant human proteins. J Struct Funct Genomics. 2007 Sep; 8(2-3):107-19. doi: 10. 1007/s10969-007-9038-z PMID: 18167031
11. Edwards AM, Bountra C, Kerr DJ, Willson TM. Open access chemical and clinical probes to support drug discovery. Nat Chem Biol. 2009 Jul; 5(7):436-40. doi: 10. 1038/nchembio0709-436 PMID: 19536100
12. Weigelt J. The case for open-access chemical biology. A strategy for pre-competitive medicinal chemistry to promote drug discovery. EMBO Rep. 2009 Sep; 10(9):941-5. doi: 10. 1038/embor. 2009. 193 PMID: 19721463
13. Egger G, Liang G, Aparicio A, Jones PA. Epigenetics in human disease and prospects for epigenetic therapy. Nature. 2004 May 27; 429(6990):457-63. PMID: 15164071
14. Arrowsmith CH, Bountra C, Fish PV, Lee K, Schapira M. Epigenetic protein families: a new frontier for drug discovery. Nat Rev Drug Discov. 2012 May; 11(5):384-400. doi: 10. 1038/nrd3674 PMID: 22498752
15. Walport LJ, Hopkinson RJ, Vollmar M, Madden SK, Gileadi C, Oppermann U, et al. Human UTY (KDM6C) is a male-specific N-methyl lysyl demethylase. J Biol Chem. 2014 Jun 27; 289(26):18302-13. doi: 10. 1074/jbc. M114. 555052 PMID: 24798337
16. Kruidenier L, Chung CW, Cheng Z, Liddle J, Che K, Joberty G, et al. A selective jumonji H3K27 demethylase inhibitor modulates the proinflammatory macrophage response. Nature. 2012 Aug 16; 488(7411):404-8. doi: 10. 1038/nature11262 PMID: 22842901
17. Hillringhaus L, Yue WW, Rose NR, Ng SS, Gileadi C, Loenarz C, et al. Structural and evolutionary basis for the dual substrate selectivity of human KDM4 histone demethylase family. J Biol Chem. 2011 Dec 2; 286(48):41616-25. doi: 10. 1074/jbc. M111. 283689 PMID: 21914792
18. King ON, Li XS, Sakurai M, Kawamura A, Rose NR, Ng SS, et al. Quantitative high-throughput screening identifies 8-hydroxyquinolines as cell-active histone demethylase inhibitors. PLoS One. 2010; 5 (11):e15535. doi: 10. 1371/journal. pone. 0015535 PMID: 21124847
19. Rose NR, Ng SS, Mecinovic J, Lienard BM, Bello SH, Sun Z, et al. Inhibitor scaffolds for 2-oxoglutaratedependent histone lysine demethylases. J Med Chem. 2008 Nov 27; 51(22):7053-6. doi: 10. 1021/ jm800936s PMID: 18942826
20. Siarheyeva A, Senisterra G, Allali-Hassani A, Dong A, Dobrovetsky E, Wasney GA, et al. An allosteric inhibitor of protein arginine methyltransferase 3. Structure. 2012 Aug 8; 20(8):1425-35. doi: 10. 1016/j. str. 2012. 06. 001 PMID: 22795084
21. Vedadi M, Barsyte-Lovejoy D, Liu F, Rival-Gervier S, Allali-Hassani A, Labrie V, et al. A chemical probe selectively inhibits G9a and GLP methyltransferase activity in cells. Nat Chem Biol. 2011 Aug; 7 (8):566-74. doi: 10. 1038/nchembio. 599 PMID: 21743462
22. Wu H, Min J, Lunin VV, Antoshenko T, Dombrovski L, Zeng H, et al. Structural biology of human H3K9 methyltransferases. PLoS One. 2010; 5(1):e8570. doi: 10. 1371/journal. pone. 0008570 PMID: 20084102
23. Yu W, Chory EJ, Wernimont AK, Tempel W, Scopton A, Federation A, et al. Catalytic site remodelling of the DOT1L methyltransferase by selective inhibitors. Nat Commun. 2012; 3:1288. doi: 10. 1038/ ncomms2304 PMID: 23250418
24. Senisterra G, Wu H, Allali-Hassani A, Wasney GA, Barsyte-Lovejoy D, Dombrovski L, et al. Small-molecule inhibition of MLL activity by disruption of its interaction with WDR5. Biochem J. 2013 Jan 1; 449 (1):151-9. doi: 10. 1042/BJ20121280 PMID: 22989411
25. Muller S, Filippakopoulos P, Knapp S. Bromodomains as therapeutic targets. Expert Rev Mol Med. 2011 Sep 13; 13:1-21.
26. Filippakopoulos P, Knapp S. The bromodomain interaction module. Febs Lett. 2012 Aug 14; 586 (17):2692-704. doi: 10. 1016/j. febslet. 2012. 04. 045 PMID: 22710155
27. Filippakopoulos P, Knapp S. Targeting bromodomains: epigenetic readers of lysine acetylation. Nat Rev Drug Discov. 2014 May; 13(5):337-56. doi: 10. 1038/nrd4286 PMID: 24751816
28. Filippakopoulos P, Qi J, Picaud S, Shen Y, Smith WB, Fedorov O, et al. Selective inhibition of BET bromodomains. Nature. 2010 Dec 23; 468(7327):1067-73. doi: 10. 1038/nature09504 PMID: 20871596
29. Taverna SD, Cole PA. Drug discovery: Reader's block. Nature. 2010 Dec 23; 468(7327):1050-1. doi: 10. 1038/4681050a PMID: 21179160
30. Nicodeme E, Jeffrey KL, Schaefer U, Beinke S, Dewell S, Chung CW, et al. Suppression of inflammation by a synthetic histone mimic. Nature. 2010 Dec 23; 468(7327):1119-23. doi: 10. 1038/nature09589 PMID: 21068722
31. Picaud S, Da Costa D, Thanasopoulou A, Filippakopoulos P, Fish PV, Philpott M, et al. PFI-1, a highly selective protein interaction inhibitor, targeting BET Bromodomains. Cancer Res. 2013 Jun 1; 73 (11):3336-46. doi: 10. 1158/0008-5472. CAN-12-3292 PMID: 23576556
32. Blobel GA, Kalota A, Sanchez PV, Carroll M. Short hairpin RNA screen reveals bromodomain proteins as novel targets in acute myeloid leukemia. Cancer Cell. 2011 Sep 13; 20(3):287-8. doi: 10. 1016/j. ccr. 2011. 08. 019 PMID: 21907920
33. Zuber J, Shi J, Wang E, Rappaport AR, Herrmann H, Sison EA, et al. RNAi screen identifies Brd4 as a therapeutic target in acute myeloid leukaemia. Nature. 2011 Oct 27; 478(7370):524-8. doi: 10. 1038/ nature10334 PMID: 21814200
34. Delmore JE, Issa GC, Lemieux ME, Rahl PB, Shi J, Jacobs HM, et al. BET bromodomain inhibition as a therapeutic strategy to target c-Myc. Cell. 2011 Sep 16; 146(6):904-17. doi: 10. 1016/j. cell. 2011. 08. 017 PMID: 21889194
35. Spiltoir JI, Stratton MS, Cavasin MA, Demos-Davies K, Reid BG, Qi J, et al. BET acetyl-lysine binding proteins control pathological cardiac hypertrophy. J Mol Cell Cardiol. 2013 Oct; 63:175-9. doi: 10. 1016/ j. yjmcc. 2013. 07. 017 PMID: 23939492
36. Anand P, Brown JD, Lin CY, Qi J, Zhang R, Artero PC, et al. BET bromodomains mediate transcriptional pause release in heart failure. Cell. 2013 Aug 1; 154(3):569-82. doi: 10. 1016/j. cell. 2013. 07. 013 PMID: 23911322
37. Boehm D, Conrad RJ, Ott M. Bromodomain proteins in HIV infection. Viruses. 2013 Jun; 5(6):1571-86. doi: 10. 3390/v5061571 PMID: 23793227
38. Mertz JA, Conery AR, Bryant BM, Sandy P, Balasubramanian S, Mele DA, et al. Targeting MYC dependence in cancer by inhibiting BET bromodomains. Proc Natl Acad Sci U S A. 2011 Oct 4; 108 (40):16669-74. doi: 10. 1073/pnas. 1108190108 PMID: 21949397
39. Edwards AM, Arrowsmith CH, Bountra C, Bunnage ME, Feldmann M, Knight JC, et al. Preclinical target validation using patient-derived cells. Nat Rev Drug Discov. 2015 Mar; 14(3):149-50. doi: 10. 1038/ nrd4565 PMID: 25722227
40. Mohedas AH, Xing X, Armstrong KA, Bullock AN, Cuny GD, Yu PB. Development of an ALK2-biased BMP type I receptor kinase inhibitor. ACS Chem Biol. 2013; 8(6):1291-302. doi: 10. 1021/cb300655w PMID: 23547776
41. Sanvitale CE, Kerr G, Chaikuad A, Ramel MC, Mohedas AH, Reichert S, et al. A new class of small molecule inhibitor of BMP signaling. PLoS One. 2013; 8(4):e62721. doi: 10. 1371/journal. pone. 0062721 PMID: 23646137
42. Chaikuad A, Alfano I, Kerr G, Sanvitale CE, Boergermann JH, Triffitt JT, et al. Structure of the bone morphogenetic protein receptor ALK2 and implications for fibrodysplasia ossificans progressiva. J Biol Chem. 2012 Oct 26; 287(44):36990-8. doi: 10. 1074/jbc. M112. 365932 PMID: 22977237
43. Dolgin E. Patent-free pact pushes the boundaries of precompetitive research. Nat Med. 2014 Jun; 20 (6):564-5. doi: 10. 1038/nm0614-564 PMID: 24901552
44. Edwards AM, Isserlin R, Bader GD, Frye SV, Willson TM, Yu FH. Too many roads not taken. Nature. 2011 Feb 10; 470(7333):163-5. doi: 10. 1038/470163a PMID: 21307913