Defibrotide (Defitelio): A New Addition to the Stockpile of Food and Drug Administration-approved Oligonucleotide Drugs

Molecular Therapy Nucleic Acids

Volumn 5, Issue , 2016, Pages e346-

  Stein, Cy ^a   Castanotto, Daniela ^a   Krishnan, Amrita ^a   Nikolaenko, Liana ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CELL SURFACE RECEPTOR; COLLAGEN TYPE 1; DEFIBROTIDE; FIBROBLAST GROWTH FACTOR 2; HEPARIN BINDING PROTEIN; PLATELET DERIVED GROWTH FACTOR BB; PROSTAGLANDIN E2; TISSUE PLASMINOGEN ACTIVATOR; TUMOR NECROSIS FACTOR; VASCULOTROPIN;

ANTIANGIOGENIC ACTIVITY; AUTOLOGOUS BONE MARROW TRANSPLANTATION; BINDING AFFINITY; CANCER PATIENT; CANCER SURVIVAL; CAPILLARY ENDOTHELIAL CELL; CYTOTOXIC T LYMPHOCYTE; DEPOLYMERIZATION; DRUG BINDING; EXTRACELLULAR MATRIX; FOOD AND DRUG ADMINISTRATION; GEL ELECTROPHORESIS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEPATIC STELLATE CELL; HUMAN; MACROMOLECULE; MITOGENESIS; MOLECULARLY TARGETED THERAPY; MORPHOGENESIS; MYELOABLATIVE CONDITIONING; NONHUMAN; NOTE; PERSONALIZED MEDICINE; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; SURVIVAL RATE; THROMBOCYTE AGGREGATION; UNITED STATES; VASCULAR ENDOTHELIAL CELL; VEIN BLOOD FLOW; DISEASE SEVERITY; DRUG APPROVAL; DRUG MARKETING; LIVER INJURY; LIVER VEIN OBSTRUCTION; LIVER VENOOCCLUSIVE DISEASE;

EID: 85015154737     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: 10.1038/mtna.2016.42     Document Type: Note

References (31)

1. 1 Pescador, R, Capuzzi, L, Mantovani, M, Fulgenzi, A, Ferrero, ME, Defibrotide: properties and clinical use of an old/new drug. Vascul Pharmacol 59 (2013), 1–10.
2. 2 Benimetskaya, L, Wu, S, Voskresenskiy, AM, Echart, C, Zhou, JF, Shin, J, et al. Angiogenesis alteration by defibrotide: implications for its mechanism of action in severe hepatic veno-occlusive disease. Blood 112 (2008), 4343–4352.
3. 3 Valla, DC, Cazals-Hatem, D, Sinusoidal obstruction syndrome. Clin Res Hepatol Gastroenterol, 2016 epub ahead of print.
4. 4 Dalle, JH, Giralt, SA, Hepatic veno-occlusive disease after hematopoietic stem cell transplantation: risk factors and stratification, prophylaxis, and treatment. Biol Blood Marrow Transplant 22 (2016), 400–409.
5. 5 Fan, CQ, Crawford, JM, Sinusoidal obstruction syndrome (hepatic veno-occlusive disease). J Clin Exp Hepatol 4 (2014), 332–346.
6. 6 Mohty, M, Malard, F, Abecassis, M, Aerts, E, Alaskar, AS, Aljurf, M, et al. Sinusoidal obstruction syndrome/veno-occlusive disease: current situation and perspectives-a position statement from the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant 50 (2015), 781–789.
7. 7 Dignan, FL, Wynn, RF, Hadzic, N, Karani, J, Quaglia, A, Pagliuca, A, et al., Haemato-oncology Task Force of British Committee for Standards in Haematology, British Society for Blood and Marrow Transplantation, BCSH/BSBMT guideline: diagnosis and management of veno-occlusive disease (sinusoidal obstruction syndrome) following haematopoietic stem cell transplantation. Br J Haematol 163 (2013), 444–457.
8. 8 DeLeve, LD, Ito, Y, Bethea, NW, McCuskey, MK, Wang, X, McCuskey, RS, Embolization by sinusoidal lining cells obstructs the microcirculation in rat sinusoidal obstruction syndrome. Am J Physiol Gastrointest Liver Physiol 284 (2003), G1045–G1052.
9. 9 DeLeve, LD, Shulman, HM, McDonald, GB, Toxic injury to hepatic sinusoids: sinusoidal obstruction syndrome (veno-occlusive disease). Semin Liver Dis 22 (2002), 27–42.
10. 10 Richardson, PG, Riches, ML, Kernan, NA, Brochstein, JA, Mineishi, S, Termuhlen, AM, et al. Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure. Blood 127 (2016), 1656–1665.
11. 11 Cheuk, DK, Chiang, AK, Ha, SY, Chan, GC, Interventions for prophylaxis of hepatic veno-occlusive disease in people undergoing haematopoietic stem cell transplantation. Cochrane Database Syst Rev, 5, 2015 CD009311.
12. 12 Koehl, GE, Geissler, EK, Iacobelli, M, Frei, C, Burger, V, Haffner, S, et al. Defibrotide: an endothelium protecting and stabilizing drug, has an anti-angiogenic potential in vitro and in vivo. Cancer Biol Ther 6 (2007), 686–690.
13. 13 Akimoto, T, Hammerman, MR, Fibroblast growth factor 2 promotes microvessel formation from mouse embryonic aorta. Am J Physiol Cell Physiol 284 (2003), C371–C377.
14. 14 Bikfalvi, A, Klein, S, Pintucci, G, Rifkin, DB, Biological roles of fibroblast growth factor-2. Endocr Rev 18 (1997), 26–45.
15. 15 Basilico, C, Moscatelli, D, The FGF family of growth factors and oncogenes. Adv Cancer Res 59 (1992), 115–165.
16. 16 Seghezzi, G, Patel, S, Ren, CJ, Gualandris, A, Pintucci, G, Robbins, ES, et al. Fibroblast growth factor-2 (FGF-2) induces vascular endothelial growth factor (VEGF) expression in the endothelial cells of forming capillaries: an autocrine mechanism contributing to angiogenesis. J Cell Biol 141 (1998), 1659–1673.
17. 17 Jones, LS, Yazzie, B, Middaugh, CR, Polyanions and the proteome. Mol Cell Proteomics 3 (2004), 746–769.
18. 18 Bashkin, P, Doctrow, S, Klagsbrun, M, Svahn, CM, Folkman, J, Vlodavsky, I, Basic fibroblast growth factor binds to subendothelial extracellular matrix and is released by heparitinase and heparin-like molecules. Biochemistry 28 (1989), 1737–1743.
19. 19 Vlodavsky, I, Folkman, J, Sullivan, R, Fridman, R, Ishai-Michaeli, R, Sasse, J, et al. Endothelial cell-derived basic fibroblast growth factor: synthesis and deposition into subendothelial extracellular matrix. Proc Natl Acad Sci USA 84 (1987), 2292–2296.
20. 20 Baird, A, Ling, N, Fibroblast growth factors are present in the extracellular matrix produced by endothelial cells in vitro: implications for a role of heparinase-like enzymes in the neovascular response. Biochem Biophys Res Commun 142 (1987), 428–435.
21. 21 Powers, CJ, McLeskey, SW, Wellstein, A, Fibroblast growth factors, their receptors and signaling. Endocr Relat Cancer 7 (2000), 165–197.
22. 22 Lazarus, HM, McCrae, KR, SOS! Defibrotide to the rescue. Blood 112 (2008), 3924–3925.
23. 23 Kornblum, N, Ayyanar, K, Benimetskaya, L, Richardson, P, Iacobelli, M, Stein, CA, Defibrotide, a polydisperse mixture of single-stranded phosphodiester oligonucleotides with lifesaving activity in severe hepatic veno-occlusive disease: clinical outcomes and potential mechanisms of action. Oligonucleotides 16 (2006), 105–114.
24. 24 Richardson, P, Guinan, E, Hepatic veno-occlusive disease following hematopoietic stem cell transplantation. Acta Haematol 106 (2001), 57–68.
25. 25 Palomo, M, Mir, E, Rovira, M, Escolar, G, Carreras, E, Diaz-Ricart, M, What is going on between defibrotide and endothelial cells? Snapshots reveal the hot spots of their romance. Blood 127 (2016), 1719–1727.
26. 26 Bianchi, G, Barone, D, Lanzarotti, E, Tettamanti, R, Porta, R, Moltrasio, D, et al. Defibrotide, a single-stranded polydeoxyribonucleotide acting as an adenosine receptor agonist. Eur J Pharmacol 238 (1993), 327–334.
27. 27 Francischetti, IM, Oliveira, CJ, Ostera, GR, Yager, SB, Debierre-Grockiego, F, Carregaro, V, et al. Defibrotide interferes with several steps of the coagulation-inflammation cycle and exhibits therapeutic potential to treat severe malaria. Arterioscler Thromb Vasc Biol 32 (2012), 786–798.
28. 28 Eissner, G, Multhoff, G, Gerbitz, A, Kirchner, S, Bauer, S, Haffner, S, et al. Fludarabine induces apoptosis, activation, and allogenicity in human endothelial and epithelial cells: protective effect of defibrotide. Blood 100 (2002), 334–340.
29. 29 Echart, CL, Graziadio, B, Somaini, S, Ferro, LI, Richardson, PG, Fareed, J, et al. The fibrinolytic mechanism of defibrotide: effect of defibrotide on plasmin activity. Blood Coagul Fibrinolysis 20 (2009), 627–634.
30. 30 Cella, G, Sbarai, A, Mazzaro, G, Motta, G, Carraro, P, Andreozzi, GM, et al. Tissue factor pathway inhibitor release induced by defibrotide and heparins. Clin Appl Thromb Hemost 7 (2001), 225–228.
31. 31 Coccheri, S, Biagi, G, Legnani, C, Bianchini, B, Grauso, F, Acute effects of defibrotide, an experimental antithrombotic agent, on fibrinolysis and blood prostanoids in man. Eur J Clin Pharmacol 35 (1988), 151–156.