New sources of drugs for hematologic malignancies

Blood

Volumn 117, Issue 25, 2011, Pages 6747-6755

  Sukhai, Mahadeo A ^a   Spagnuolo, Paul A ^a   Weir, Scott ^b   Kasper, James ^c   Patton, Lavonne ^d   Schimmer, Aaron D ^a  

^a PRINCESS MARGARET HOSPITAL   (Canada)

^b UNIVERSITY OF KANSAS MEDICAL CENTER   (United States)

^c The Leukemia & Lymphoma Society   (United States)

^d Beckloff Associates Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

5 AZA 2' DEOXYCYTIDINE; AFFINITOR; ARSENIC TRIOXIDE; AZACITIDINE; BENDAMUSTINE; BORTEZOMIB; DASATINIB; ERLOTINIB; EVEROLIMUS; FULVESTRANT; GEFITINIB; GEMTUZUMAB OZOGAMICIN; IMATINIB; IXABEPILONE; LAPATINIB; LENALIDOMIDE; NELARABINE; NILOTINIB; OXALIPLATIN; PAZOPANIB; PRALATREXATE; ROMIDEPSIN; SORAFENIB; SUNITINIB; TEMSIROLIMUS; UNCLASSIFIED DRUG; VORINOSTAT;

DRUG APPROVAL; DRUG DEVELOPMENT; HEMATOLOGIC MALIGNANCY; HUMAN; NONHUMAN; PRIORITY JOURNAL; REVIEW;

EID: 79959515584     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2011-02-315283     Document Type: Review

References (42)

1. Adams CP, Brantner VV. Estimating the cost of new drug development: is it really 802 million dollars? Health Aff (Millwood). 2006;25(2):420-428. (Pubitemid 43529550)
2. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs. J Health Econ. 2003;22(2):151-185. (Pubitemid 36279392)
3. Tobinick EL. The value of drug repositioning in the current pharmaceutical market. Drug News Perspect. 2009;22(2):119-125.
4. Orlowski RZ, Stinchcombe TE, Mitchell BS, et al. Phase I trial of the proteasome inhibitor PS-341 in patients with refractory hematologic malignancies. J Clin Oncol. 2002;20(22):4420-4427. (Pubitemid 35334752)
5. Goy A, Younes A, McLaughlin P, et al. Phase II study of proteasome inhibitor bortezomib in relapsed or refractory B-cell non-Hodgkin's lymphoma. J Clin Oncol. 2005;23(4):667-675. (Pubitemid 46224166)
6. O'Connor OA, Wright J, Moskowitz C, et al. Phase II clinical experience with the novel proteasome inhibitor bortezomib in patients with indolent non-Hodgkin's lymphoma and mantle cell lymphoma. J Clin Oncol. 2005;23(4):676-684. (Pubitemid 46237441)
7. Belch A, Kouroukis CT, Crump M, et al. A phase II study of bortezomib in mantle cell lymphoma: the National Cancer Institute of Canada Clinical Trials Group trial IND.150. Ann Oncol. 2007;18(1):116-121. (Pubitemid 46152510)
8. Fisher RI, Bernstein SH, Kahl BS, et al. Multicenter phase II study of bortezomib in patients with relapsed or refractory mantle cell lymphoma. J Clin Oncol. 2006;24(30):4867-4874. (Pubitemid 46655613)
9. Miyachi K, Ihara A, Hankins RW, Murai R, Maehiro S, Miyashita H. Efficacy of imatinib mesylate (STI571) treatment for a patient with rheumatoid arthritis developing chronic myelogenous leukemia. Clin Rheumatol. 2003;22(4):329-332. (Pubitemid 37483630)
10. Bartlett JB, Dredge K, Dalgleish AG. The evolution of thalidomide and its IMiD derivatives as anticancer agents. Nat Rev Cancer. 2004;4(4):314-322. (Pubitemid 38525287)
11. Calabrese L, Fleischer AB. Thalidomide: current and potential clinical applications. Am J Med. 2000;108(6):487-495. (Pubitemid 30214201)
12. Sheskin J. Thalidomide in the treatment of lepra reactions. Clin Pharmacol Ther. 1965;6:303-306.
13. Singhal S, Mehta J, Desikan R, et al. Antitumor activity of thalidomide in refractory multiple myeloma. N Engl J Med. 1999;341(21):1565-1571.
14. Barlogie B, Tricot G, Anaissie E, et al. Thalidomide and hematopoietic-cell transplantation for multiple myeloma. N Engl J Med. 2006;354(10):1021-1030. (Pubitemid 43357806)
15. Rajkumar SV, Blood E, Vesole D, Fonseca R, Greipp PR. Phase III clinical trial of thalidomide plus dexamethasone compared with dexamethasone alone in newly diagnosed multiple myeloma: a clinical trial coordinated by the Eastern Cooperative Oncology Group. J Clin Oncol. 2006;24(3):431-436. (Pubitemid 46630462)
16. Alexanian R, Weber D, Giralt S, Delasalle K. Consolidation therapy of multiple myeloma with thalidomide-dexamethasone after intensive chemotherapy. Ann Oncol. 2002;13(7):1116-1119.
17. Strupp C, Germing U, Aivado M, Misgeld E, Haas R, Gattermann N. Thalidomide for the treatment of patients with myelodysplastic syndromes. Leukemia. 2002;16(1):1-6. (Pubitemid 34105592)
18. Bowen D, MacIlwaine L, Cavanagh J, et al. Thalidomide therapy for low-risk myelodysplasia. Leuk Res. 2005;29(2):235-236.
19. Richardson PG, Blood E, Mitsiades CS, et al. A randomized phase 2 study of lenalidomide therapy for patients with relapsed or relapsed and refractory multiple myeloma. Blood. 2006;108(10):3458-3464.
20. Merchionne F, Perosa F, Dammacco F. New therapies in multiple myeloma. Clin Exp Med. 2007;7(3):83-97. (Pubitemid 350045116)
21. Sarlomo-Rikala M, Kovatich AJ, Barusevicius A, Miettinen M. CD117: a sensitive marker for gastrointestinal stromal tumors that is more specific than CD34. Mod Pathol. 1998;11(8):728-734. (Pubitemid 28381606)
22. Joensuu H, Roberts PJ, Sarlomo-Rikala M, et al. Effect of the tyrosine kinase inhibitor STI571 in a patient with a metastatic gastrointestinal stromal tumor. N Engl J Med. 2001;344(14):1052-1056. (Pubitemid 32267975)
23. Dagher R, Cohen M, Williams G, et al. Approval summary: imatinib mesylate in the treatment of metastatic and/or unresectable malignant gastrointestinal stromal tumors. Clin Cancer Res. 2002;8(10):3034-3038. (Pubitemid 35155011)
24. Kindler T, Breitenbuecher F, Marx A, et al. Sustained complete hematologic remission after administration of the tyrosine kinase inhibitor imatinib mesylate in a patient with refractory, secondary AML. Blood. 2003;101(8):2960-2962. (Pubitemid 36857980)
25. Walker AR, Komrokji RS, Ifthikharuddin J, et al. Phase I study of cladribine, cytarabine (Ara-C), granulocyte colony stimulating factor (G-CSF) (CLAG Regimen) and simultaneous escalating doses of imatinib mesylate (Gleevec) in relapsed/refractory AML. Leuk Res. 2008;32(12):1830-1836.
26. Tefferi A, Pardanani A. Imatinib therapy in clonal eosinophilic disorders, including systemic mastocytosis. Int J Hematol. 2004;79(5):441-447. (Pubitemid 38808359)
27. McArthur GA, Demetri GD, van Oosterom A, et al. Molecular and clinical analysis of locally advanced dermatofibrosarcoma protuberans treated with imatinib: Imatinib Target Exploration Consortium Study B2225. J Clin Oncol. 2005;23(4):866-873. (Pubitemid 46224187)
28. Rousseau D, Kaspar R, Rosenwald I, Gehrke L, Sonenberg N. Translation initiation of ornithine decarboxylase and nucleocytoplasmic transport of cyclin D1 mRNA are increased in cells overexpressing eukaryotic initiation factor 4E. Proc Natl Acad Sci U S A. 1996;93(3):1065-1070. (Pubitemid 26054368)
29. Lai HK, Borden KL. The promyelocytic leukemia (PML) protein suppresses cyclin D1 protein production by altering the nuclear cytoplasmic distribution of cyclin D1 mRNA. Oncogene. 2000;19(13):1623-1634. (Pubitemid 30200964)
30. Cohen N, Sharma M, Kentsis A, Perez JM, Strudwick S, Borden KL. PML RING suppresses oncogenic transformation by reducing the affinity of eIF4E for mRNA. EMBO J. 2001;20(16):4547-4559. (Pubitemid 32772048)
31. Topisirovic I, Guzman ML, McConnell MJ, et al. Aberrant eukaryotic translation initiation factor 4E-dependent mRNA transport impedes hematopoietic differentiation and contributes to leukemogenesis. Mol Cell Biol. 2003;23(24):8992-9002. (Pubitemid 37499790)
32. Kentsis A, Topisirovic I, Culjkovic B, Shao L, Borden KL. Ribavirin suppresses eIF4E-mediated oncogenic transformation by physical mimicry of the 7-methyl guanosine mRNA cap. Proc Natl Acad Sci U S A. 2004;101(52):18105-18110. (Pubitemid 40054081)
33. Assouline S, Culjkovic B, Cocolakis E, et al. Molecular targeting of the oncogene eIF4E in acute myeloid leukemia (AML): a proof-of-principle clinical trial with ribavirin. Blood. 2009;114(2):257-260.
34. Flaherty KT, Puzanov I, Kim KB, et al. Inhibition of mutated, activated BRAF in metastatic melanoma. N Engl J Med. 2010;363(9):809-819.
35. Eberhard Y, McDermott SP, Wang X, et al. Chelation of intracellular iron with the antifungal agent ciclopirox olamine induces cell death in leukemia and myeloma cells. Blood. 2009;114(14):3064-3073.
36. Rickles RJ, Pierce LT, Giordano TP III, et al. Adenosine A2A receptor agonists and PDE inhibitors: a synergistic multitarget mechanism discovered through systematic combination screening in B-cell malignancies. Blood. 2010;116(4):593-602.
37. Stegmaier K, Ross KN, Colavito SA, O'Malley S, Stockwell BR, Golub TR. Gene expression-based high-throughput screening (GE-HTS) and application to leukemia differentiation. Nat Genet. 2004;36(3):257-263. (Pubitemid 38282750)
38. Stegmaier K, Corsello SM, Ross KN, Wong JS, Deangelo DJ, Golub TR. Gefitinib induces myeloid differentiation of acute myeloid leukemia. Blood. 2005;106(8):2841-2848. (Pubitemid 41510762)
39. Quartel A, Lennertz J. Orphan drugs: BioMarin Europe replies. BMJ. 2010;341:c7006.
40. Kesselheim AS, Solomon DH. Incentives for drug development: the curious case of colchicine. N Engl J Med. 2010;362(22):2045-2047.
41. Uhl K, Cox E, Rogan R, et al. Thalidomide use in the US: experience with pregnancy testing in the S.T.E.P.S. programme. Drug Saf. 2006;29(4):321-329.
42. Anderson KC, Kyle RA, Rajkumar SV, Stewart AK, Weber D, Richardson P. Clinically relevant end points and new drug approvals for myeloma. Leukemia. 2008;22(2):231-239. (Pubitemid 351250526)