Clinical trial design for target-based therapy

Oncologist

Volumn 7, Issue 5, 2002, Pages 401-409

  Fox, Elizabeth ^a   Curt, Gregory A ^a   Balis, Frank M ^a  

^a NATIONAL CANCER INSTITUTE   (United States)

Author keywords

Clinical trials; Drug development; Molecular targets; Pharmacodynamics; Pharmacokinetics

Indexed keywords

ANTINEOPLASTIC AGENT; IMATINIB;

ANTINEOPLASTIC ACTIVITY; ARTICLE; CANCER; CANCER SURVIVAL; CELL DEATH; CELL PROLIFERATION; CLINICAL TRIAL; CYTOSTASIS; CYTOTOXICITY; DRUG EFFECT; HUMAN; MALIGNANT TRANSFORMATION; PATHOGENESIS; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PHASE 3 CLINICAL TRIAL; PRIORITY JOURNAL; RISK BENEFIT ANALYSIS; SCREENING;

ANTINEOPLASTIC AGENTS; CLINICAL TRIALS; DRUG DESIGN; DRUG EVALUATION; DRUG THERAPY; HUMANS; NEOPLASMS; RESEARCH DESIGN;

EID: 0036411742     PISSN: 10837159     EISSN: None     Source Type: Journal    
DOI: 10.1634/theoncologist.7-5-401     Document Type: Article

References (38)

1. Balis FM. Evolution of anticancer drug discovery and the role of cell-based screening. J Natl Cancer Inst 2002;94:78-79.
2. Turner KJ, Moore JW, Jones A et al. Expression of hypoxia-inducible factors in human renal cancer: Relationship to angiogenesis and to the von Hippel-Lindau gene mutation. Cancer Res 2002;62:2957-2961.
3. Lowe ES, Balis FM. Dose-effect and concentration effect analysis. In: Atkinson AJ, Daniels CE, Dedrick RL et al., eds. Principles of Clinical Pharmacology. San Diego: Academic Press, 2001:235-244.
4. Sun GL, Ouyang RR, Chen SJ et al. Treatment of acute promyelocytic leukemia with all-trans retinoic acid. A five-year experience. Chin Med J (Engl) 1993;106:743-748.
5. Tallman MS, Andersen JW, Schiffer CA et al. All-transretinoic acid in acute promyelocytic leukemia. N Engl J Med 1997;337:1021-1028.
6. Druker BJ, Sawyers CL, Kantarjian H et al. Activity of a specific inhibitor of the BCR-ABL tyrosine kinase in the blast crisis of chronic myeloid leukemia and acute lymphoblastic leukemia with the Philadelphia chromosome. N Engl J Med 2001;344:1038-1042.
7. Druker BJ, Talpaz M, Resta DJ et al. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med 2001;344:1031-1037.
8. van Oosterom AT, Judson I, Verweij J et al. Safety and efficacy of imatinib (STI571) in metastatic gastrointestinal stromal tumours: A phase I study. Lancet 2001;358:1421-1423.
9. Piantadosi S. Principles of clinical trial design. Semin Oncol 1988;15:423-433.
10. Korn EL, Arbuck SG, Pluda JM et al. Clinical trial designs for cytostatic agents: Are new approaches needed? J Clin Oncol 2001;19:265-272.
11. Buchdunger E, Cioffi CL, Law N et al. AbI protein-tyrosine kinase inhibitor STI571 inhibits in vitro signal transduction mediated by c-kit and platelet-derived growth factor receptors. J Pharmacol Exp Ther 2000;295:139-145.
12. Saijo N, Tamura T, Nishio K. Problems in the development of target-based drugs. Cancer Chemother Pharmacol 2000;46(suppl 1):S43-S45.
13. Gelmon KA, Eisenhauer EA, Harris AL et al. Anticancer agents targeting signaling molecules and cancer cell environment: Challenges for drug development? J Natl Cancer Inst 1999;91:1281-1287.
14. 6-benzylguanine for patients undergoing surgery for malignant glioma. J Clin Oncol 1998;16:3570-3575.
15. 6-enzylguanine: A clinical trial establishing the biochemical modulatory dose in tumor tissue for alkyltransferase-directed DNA repair. Cancer Res 1999;59:2402-2410.
16. Dowlati A, Haaga J, Remick SC et al. Sequential tumor biopsies in early phase clinical trials of anticancer agents for pharmacodynamic evaluation. Clin Cancer Res 2001,7:2971-2976.
17. Tabernero J, Sonnichsen D, Albanell J et al. A phase 1 pharmacokinetic (PK) and serial tumor and PBMC pharmacodynamic (PD) study of weekly BMS-214662, a farnesyltransferase (FT) inhibitor, in patients with advanced solid tumors. Proc Am Soc Clin Oncol 2001;20:77a.
18. End DW, Smets G, Todd AV et al. Characterization of the antitumor effects of the selective farnesyl protein transferase inhibitor R115777 in vivo and in vitro. Cancer Res 2001;61:131-137.
19. Rowinsky EK, Windle JJ, Von Hoff DD. Ras protein farnesyltransferase: A strategic target for anticancer therapeutic development. J Clin Oncol 1999;17:3631-3652.
20. 1 block in p53 wild type tumor cells. J Biol Chem 1998;273:20243-20251.
21. Sepp-Lorenzino L, Ma Z, Rands E et al. A peptidomimetic inhibitor of farnesyl:protein transferase blocks the anchorage-dependent and -independent growth of human tumor cell lines. Cancer Res 1995;55:5302-5309.
22. Chabner BA, Boral AL, Multani P. Translational research: Walking the bridge between idea and cure-seventeenth Bruce F. Cain Memorial Award lecture. Cancer Res 1998;58:4211-4216.
23. Lightcap ES, McCormack TA, Pien CS et al. Proteasome inhibition measurements: Clinical application. Clin Chem 2000;46:673-683.
24. Erlichman C, Adjeii A, Thomas JP et al. A phase I trial of the proteasome inhibitor PS-341 in patients with advanced cancer. Proc Am Soc Clin Oncol 2001;20:85a.
25. Hamilton AL, Eder J, Pavlick A et al. PS-341: Phase I study of a novel proteasome inhibitor with pharmacodynamic end points. Proc Am Soc Clin Oncol 2001;20:85a.
26. Aghahanian C, Soignet S, Dizon D et al. A phase I trial of the novel proteasome inhibitor PS-341 in advanced solid tumor malignancies. Proc Am Soc Clin Oncol 2001;20:85a.
27. Partridge AH, Avom J, Wandg PS et al. Adherence to therapy with oral antineoplastic agents. J Natl Cancer Inst 2002;94:652-661.
28. Therasse P, Arbuck SG, Eisenhauer EA et al. New guidelines to evaluate the response to treatment in solid tumors. J Natl Cancer Inst 2000;92:205-216.
29. Simon R. Optimal two-stage designs for phase II clinical trials. Control Clin Trials 1989;10:1-10.
30. Eisenhauer EA. Phase I and II trials of novel anti-cancer agents: Endpoints, efficacy and existentialism. Ann Oncol 1998;9:1047-1052.
31. Deplanque G, Harris AL. Antiangiogenic agents: Clinical trial design and therapies in development. Eur J Cancer 2000;36:1713-1724.
32. Poon RT, Fan S-T, Wong J. Clinical implications of circulating angiogenic factors in cancer patients. J Clin Oncol 2001;19:1207-1225.
33. Haas N, Peereboom D, Ranganathan N et al. Phase II trial of R11577, an inhibitor of farnesyltransferase, in patients with hormone refractory prostate cancer. Proc Am Soc Clin Oncol 2002;21:181a.
34. Janicek MJ, Janicek MR, Merriam P et al. Imaging response to imatinib mesylate (Gleevec, STI571) in gastrointestinal stromal tumors (GIST): Vascular perfusion patterns with doppler ultrasound (DUS) and dynamic infrared imaging (DIRI). Proc Am Soc Clin Oncol 2002;21:84a.
35. Kopec JA, Abrahamowicz M, Esdaile JM. Randomized discontinuation trials: Utility and efficiency. J Clin Epidemiol 1993;46:959-971.
36. Stadler WM, Ratain MJ. Development of target-based antineoplastic agents. Invest New Drugs 2000;18:7-16.
37. Zucker S, Cao J, Chen WT. Critical appraisal of the use of matrix metalloproteinase inhibitors in cancer treatment. Oncogene 2000;19:6642-6650,
38. Eskens FA, Verweij J. Clinical studies in the development of new anticancer agents exhibiting growth inhibition in models: Facing the challenge of a proper study design. Crit Rev Oncol Hematol 2000;34:83-88.