Design, synthesis, and biological evaluation of a novel series of 2-(2,6-dioxopiperidin-3-yl)isoquinoline-1,3(2H,4H)-dione derivatives as cereblon modulators

References

• Agostini T, Sacco R, Bertolai R, et al. Solitary plasmacytoma of the jaw. J Craniofac Surg 2011;22:e2–e10.
   PubMed Web of Science ®Google Scholar
• Ho CL, Chen YC, Yiang YT, et al. Mandibular mass as the presenting manifestation of IgM myeloma in a 22-year-old man. Ann Hematol 1999;78:1715–5.
   Web of Science ®Google Scholar
• Hogan MC, Lee A, Solberg LA, et al. Unusual presentation of multiple myeloma with unilateral visual loss and numb chin syndrome in a young adult. Am J Hematol 2002;70:55–9.
   PubMed Web of Science ®Google Scholar
• Hess T, Egerer G, Kasper B, et al. Atypical manifestations of multiple myeloma: radiological appearance. Eur J Radiol 2006;58:280–5.
   PubMed Web of Science ®Google Scholar
• Pontes FSC, De Souza LL, Fonseca FP, et al. Mandibular lesion as the first sign of multiple myeloma in a young patient. J Oral Maxillofac Pathol 2019;23:94–6.
   PubMedGoogle Scholar
• Nijhof IS, van de Donk NWCJ, Zweegman S, et al. Current and new therapeutic strategies for relapsed and refractory multiple myeloma: an update. Drugs 2018;78:19–37.
   PubMed Web of Science ®Google Scholar
• Holstein SA, McCarthy PL. Immunomodulatory drugs in multiple myeloma: mechanisms of action and clinical experience. Drugs 2017;77:505–20.
   PubMed Web of Science ®Google Scholar
• Abe Y, Ishida T. Immunomodulatory drugs in the treatment of multiple myeloma. Jpn J Clin Oncol 2019;49:695–702.
   PubMed Web of Science ®Google Scholar
• El-Zahabi MA, Sakr H, El-Adl K, et al. Design, synthesis, and biological evaluation of new challenging thalidomide analogs as potential anticancer immunomodulatory agents. Bioorg Chem 2020;104:104218.
   PubMed Web of Science ®Google Scholar
• Zhu YX, Kortuem KM, Stewart AK. Molecular mechanism of action of immune-modulatory drugs thalidomide, lenalidomide and pomalidomide in multiple myeloma. Leuk Lymphoma 2013;54:683–7.
   PubMed Web of Science ®Google Scholar
• Gandhi AK, Kang J, Capone L, et al. Dexamethasone synergizes with lenalidomide to inhibit multiple myeloma tumor growth, but reduces lenalidomide-induced immunomodulation of T and NK cell function. Curr Cancer Drug Targets 2010;10:155–67.
   PubMed Web of Science ®Google Scholar
• Raza S, Safyan RA, Lentzsch S. Immunomodulatory drugs (IMiDs) in multiple myeloma. Curr Cancer Drug Targets 2017;17:846–57.
   PubMed Web of Science ®Google Scholar
• Quach H, Ritchie D, Stewart AK, et al. Mechanism of action of immunomodulatory drugs (IMiDs) in multiple myeloma. Leukemia 2010;24:22–32.
   PubMed Web of Science ®Google Scholar
• Fischer ES, Böhm K, Lydeard JR, et al. Structure of the DDB1-CRBN E3 ubiquitin ligase in complex with thalidomide. Nature 2014;512:49–53.
   PubMed Web of Science ®Google Scholar
• Ito T, Ando H, Suzuki T, et al. Identification of a primary target of thalidomide teratogenicity. Science 2010;327:1345–50.
   PubMed Web of Science ®Google Scholar
• Lopez-Girona A, Mendy D, Ito T, et al. Cereblon is a direct protein target for immunomodulatory and antiproliferative activities of lenalidomide and pomalidomide. Leukemia 2012;26:2326–35.
   PubMed Web of Science ®Google Scholar
• Steinebach C, Lindner S, Udeshi ND, et al. Homo-PROTACs for the chemical knockdown of cereblon. ACS Chem Biol 2018;13:2771–82.
   PubMed Web of Science ®Google Scholar
• Angers S, Li T, Yi X, et al. Molecular architecture and assembly of the DDB1-CUL4A ubiquitin ligase machinery. Nature 2006;443:590–3.
   PubMed Web of Science ®Google Scholar
• Higa L, Wu M, Ye T, et al. CUL4-DDB1 ubiquitin ligase interacts with multiple WD40-repeat proteins and regulates histone methylation. Nat Cell Biol 2006;8:1277–83.
   PubMed Web of Science ®Google Scholar
• Jin J, Arias EE, Chen J, et al. A family of diverse Cul4-Ddb1-interacting proteins includes Cdt2, which is required for S phase destruction of the replication factor Cdt1. Mol Cell 2006;23:709–21.
   PubMed Web of Science ®Google Scholar
• He YJ, McCall CM, Hu J, et al. DDB1 functions as a linker to recruit receptor WD40 proteins to CUL4-ROC1 ubiquitin ligases. Genes Dev 2006;20:2949–54.
   PubMed Web of Science ®Google Scholar
• Gandhi AK, Kang J, Havens CG, et al. Immunomodulatory agents lenalidomide and pomalidomide co-stimulate T cells by inducing degradation of T cell repressors Ikaros and Aiolos via modulation of the E3 ubiquitin ligase complex CRL4(CRBN). Br J Haematol 2014;164:811–21.
   PubMed Web of Science ®Google Scholar
• Krönke J, Udeshi ND, Narla A, et al. Lenalidomide causes selective degradation of IKZF1 and IKZF3 in multiple myeloma cells. Science 2014;343:301–5.
   PubMed Web of Science ®Google Scholar
• Krönke J, Fink EC, Hollenbach PW, et al. Lenalidomide induces ubiquitination and degradation of CK1α in del(5q) MDS. Nature 2015;523:183–8.
   PubMed Web of Science ®Google Scholar
• Lu G, Middleton RE, Sun H, et al. The myeloma drug lenalidomide promotes the cereblon-dependent destruction of Ikaros proteins. Science 2014;343:305–9.
   PubMed Web of Science ®Google Scholar
• Zhu YX, Braggio E, Shi CX, et al. Identification of cereblon-binding proteins and relationship with response and survival after IMiDs in multiple myeloma. Blood 2014;124:536–45.
   PubMed Web of Science ®Google Scholar
• Singhal S, Mehta J, Desikan R, et al. Antitumor activity of thalidomide in refractory multiple myeloma. N Engl J Med 1999;341:1565–71.
   PubMed Web of Science ®Google Scholar
• Bartlett JB, Dredge K, Dalgleish AG. The evolution of thalidomide and its IMiD derivatives as anticancer agents. Nat Rev Cancer 2004;4:314–22.
   PubMed Web of Science ®Google Scholar
• Weisel K, Kanz L. Lenalidomide. Recent Results Cancer Res 2014;201:347–57.
   PubMedGoogle Scholar
• Sievers QL, Gasser JA, Cowley GS, et al. Genome-wide screen identifies cullin–RING ligase machinery required for lenalidomide-dependent CRL4CRBN activity. Blood 2018;132:1293–303.
   PubMed Web of Science ®Google Scholar
• Fouquet G, Bories C, Guidez S, et al. Pomalidomide for multiple myeloma. Expert Rev Hematol 2014;7:719–31.
   PubMed Web of Science ®Google Scholar
• Das DS, Ray A, Song Y, et al. Synergistic anti-myeloma activity of the proteasome inhibitor marizomib and the IMiD immunomodulatory drug pomalidomide. Br J Haematol 2015;171:798–812.
   PubMed Web of Science ®Google Scholar
• Hagner PR, Man HW, Fontanillo C, et al. CC-122, a pleiotropic pathway modifier, mimics an interferon response and has antitumor activity in DLBCL. Blood 2015;126:779–89.
   PubMed Web of Science ®Google Scholar
• Rasco DW, Papadopoulos KP, Pourdehnad M, et al. A first-in-human study of novel cereblon modulator avadomide (CC-122) in advanced malignancies. Clin Cancer Res 2019;25:90–8.
   PubMed Web of Science ®Google Scholar
• Kim SA, Go A, Jo SH, et al. A novel cereblon modulator for targeted protein degradation. Eur J Med Chem 2019;166:65–74.
   PubMed Web of Science ®Google Scholar
• Mitsiades N, Mitsiades CS, Poulaki V, et al. Apoptotic signaling induced by immunomodulatory thalidomide analogs in human multiple myeloma cells: therapeutic implications. Blood 2002;99:4525–30.
   PubMed Web of Science ®Google Scholar
• Shi CX, Kortüm KM, Zhu YX, et al. Proteasome inhibitors block Ikaros degradation by lenalidomide in multiple myeloma. Haematologica 2015;100:315–7.
   PubMed Web of Science ®Google Scholar
• Shi CX, Zhu YX, Jedlowski P, et al. Ikaros degradation efficiency correlates with response of multiple myeloma (MM) cells to IMiD therapy and is blocked by proteasome inhibitors. Blood 2014;124:2247.
   Web of Science ®Google Scholar
• Mu X, Bai L, Xu Y, et al. Protein targeting chimeric molecules specific for dual bromodomain 4 (BRD4) and Polo-like kinase 1 (PLK1) proteins in acute myeloid leukemia cells. Biochem Biophys Res Commun 2020;521:833–9.
   PubMed Web of Science ®Google Scholar
• Almeida M, Tei Xe Ira FM, Souza M, et al. Thalidomide analogs from diamines: synthesis and evaluation as inhibitors of TNF-α production. Chem Pharm Bull 2007;55:223–6.
   PubMed Web of Science ®Google Scholar
• Boichenko I, Deiss S, Bär K, et al. A FRET-based assay for the identification and characterization of cereblon ligands. J Med Chem 2016;59:770–4.
   PubMed Web of Science ®Google Scholar
• Matyskiela ME, Zhang W, Man HW, et al. A cereblon modulator (CC-220) with improved degradation of Ikaros and Aiolos. J Med Chem 2018;61:535–42.
   PubMed Web of Science ®Google Scholar