• Encourage incorporation of biomarker tests early in drug development, even at the level of preclinical models.

  • Devote greater efforts to establishing guidelines for the evaluation, standardization, validation, and qualification of biomarkers, especially those used in clinical decision-making.

  • Develop a new funding mechanism to support the management of existing tissue repositories, and to support rational access to these valuable resources for testing biomarkers.

Addressing some issues will require a great deal of work and research, Dr. Mendelsohn noted, but there was also some “low-hanging fruit” that could be accomplished in the near future as well. In particular, he noted that developing partnerships among Federal agencies like the NCI and the FDA in exploratory areas of research were likely to help move the field forward.

REFERENCES

Arias-Mendoza, F., M.R. Smith, and T.R. Brown. 2004. Predicting treatment response in non-Hodgkin’s lymphoma from the pretreatment tumor content of phosphoethanolamine plus phosphocholine. Acad Radiol 11(4):368-376.

Berry, D.A. 2005. Statistical innovations in cancer research. In Cancer Medicine e.7: 411-425. London: BC Decker.

Berry, D.A. 2006. Bayesian clinical trials. Nature Reviews Drug Discovery 5:27-36.

Chin, K., S. DeVries, J. Fridlyand, P.T. Spellman, R. Roydasgupta, W.-L. Kuo, A. Lapuk, R.M. Neve, Z. Qian, T. Ryder, F. Chen, H. Feiler, T. Tokuyasu, C. Kingsley, S. Dairkee, Z. Meng, K. Chew, D. Pinkel, A. Jain, B.M. Ljung, L. Esserman, D.G. Albertson, F.M. Waldman, and J.W. Gray. 2006. Genomic and transcriptional aberrations linked to breast cancer pathophysiologies. Cancer Cell 10:529-541.

Comis, R.L., J.D. Miller, C.R. Aldigé, L. Krebs, and E. Stoval. 2003. Public attitudes toward participation in cancer clinical trials. J Clin Oncol 21(5):830-835.

Dave, S.S., K. Fu, G.W. Wright, L.T. Lam, P. Kluin, E.J. Boerma, T.C. Greiner, D.D. Weisenburger, A. Rosenwald, G. Ott, H.K. Müller-Hermelink, R.D. Gascoyne, J. Delabie, L.M. Rimsza, R.M. Braziel, T.M. Grogan, E. Campo, E.S. Jaffe, B.J. Dave, W. Sanger, M. Bast, J.M. Vose, J.O. Armitage, J.M. Connors, E.B. Smeland, S. Kvaloy, H. Holte, R.I. Fisher, T.P. Miller, E. Montserrat, W.H. Wilson, M. Bahl, H. Zhao, L. Yang, J. Powell, R. Simon, W.C. Chan, L.M. Staudt, and Lymphoma/Leukemia Molecular Profiling Project. 2006. Molecular diagnosis of Burkitt’s lymphoma. N Engl J Med 354(23):2431-2442.

Dehdashti, F., M.A. Mintun, J.S. Lewis, J. Bradley, R. Govindan, R. Laforest, M.J. Welch, and B.A. Siegel. 2003. In vivo assessment of tumor hypoxia in lung cancer with 60Cu-ATSM. Eur J Nucl Med Mol Imaging 30(6):844-850.



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0 IMPROVING THE QUALITY OF CANCER CLINICAL TRIALS • Encourage incorporation of biomarker tests early in drug develop- ment, even at the level of preclinical models. • Devote greater efforts to establishing guidelines for the evaluation, standardization, validation, and qualification of biomarkers, espe- cially those used in clinical decision-making. • Develop a new funding mechanism to support the management of existing tissue repositories, and to support rational access to these valuable resources for testing biomarkers. Addressing some issues will require a great deal of work and research, Dr. Mendelsohn noted, but there was also some “low-hanging fruit” that could be accomplished in the near future as well. In particular, he noted that developing partnerships among Federal agencies like the NCI and the FDA in exploratory areas of research were likely to help move the field forward. REFERENCES Arias-Mendoza, F., M.R. Smith, and T.R. Brown. 2004. Predicting treatment response in non- Hodgkin’s lymphoma from the pretreatment tumor content of phosphoethanolamine plus phosphocholine. Acad Radiol 11(4):368-376. Berry, D.A. 2005. Statistical innovations in cancer research. In Cancer Medicine e.7: 411-425. London: BC Decker. Berry, D.A. 2006. Bayesian clinical trials. Nature Reviews Drug Discovery 5:27-36. Chin, K., S. DeVries, J. Fridlyand, P.T. Spellman, R. Roydasgupta, W.-L. Kuo, A. Lapuk, R.M. Neve, Z. Qian, T. Ryder, F. Chen, H. Feiler, T. Tokuyasu, C. Kingsley, S. Dairkee, Z. Meng, K. Chew, D. Pinkel, A. Jain, B.M. Ljung, L. Esserman, D.G. Albertson, F.M. Waldman, and J.W. Gray. 2006. Genomic and transcriptional aberrations linked to breast cancer pathophysiologies. Cancer Cell 10:529-541. Comis, R.L., J.D. Miller, C.R. Aldigé, L. Krebs, and E. Stoval. 2003. Public attitudes toward participation in cancer clinical trials. J Clin Oncol 21(5):830-835. Dave, S.S., K. Fu, G.W. Wright, L.T. Lam, P. Kluin, E.J. Boerma, T.C. Greiner, D.D. Weisenburger, A. Rosenwald, G. Ott, H.K. Müller-Hermelink, R.D. Gascoyne, J. Delabie, L.M. Rimsza, R.M. Braziel, T.M. Grogan, E. Campo, E.S. Jaffe, B.J. Dave, W. Sanger, M. Bast, J.M. Vose, J.O. Armitage, J.M. Connors, E.B. Smeland, S. Kvaloy, H. Holte, R.I. Fisher, T.P. Miller, E. Montserrat, W.H. Wilson, M. Bahl, H. Zhao, L. Yang, J. Powell, R. Simon, W.C. Chan, L.M. Staudt, and Lymphoma/Leukemia Molecular Profiling Project. 2006. Molecular diagnosis of Burkitt’s lymphoma. N Engl J Med 354(23):2431-2442. Dehdashti, F., M.A. Mintun, J.S. Lewis, J. Bradley, R. Govindan, R. Laforest, M.J. Welch, and B.A. Siegel. 2003. In vivo assessment of tumor hypoxia in lung cancer with 60Cu- ATSM. Eur J Nucl Med Mol Imaging 30(6):844-850.

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0 WORKSHOP SUMMARY Di Leo, A., H. Gomez, Z. Aziz, Z. Zvirbule, M. Arbushites, C.R. Oliva, M. Koehler, L.S. Williams, J. Dering, and R.S. Finn. 2007. Lapatinib (L) with paclitaxel compared to paclitaxel as first-line treatment for patients with metastatic breast cancer: A phase III randomized, double-blind study of 580 patients. J Clin Oncol 00 ASCO Annual Meeting Proceedings Part I 25(18S):1011. Flaherty, K.T., M.A. Rosen, D.F. Heitjan, M.L. Gallagher, B. Schwart, M.D. Schnall, and P.J. Dwyer. 2008. Pilot study of DCE-MRI to predict progression-free survival with sorafenib therapy in renal cell carcinoma. Cancer Biol Ther 7(4):Epub ahead of print. Fridlyand, J., A.M. Snijders, B. Ylstra, H. Li, A. Olshen, R. Segraves, S. Dairkee, T. Tokuyasu, B.M. Ljung, A.N. Jain, J. McLennan, J. Ziegler, K. Chin, S. Devries, H. Feiler, J.W. Gray, F. Waldman, D. Pinkel, and D.G. Albertson. 2006. Breast tumor copy number aberration phenotypes and genomic instability. BMC Cancer 6:96. Giles, F.J., H.M. Kantarjian, J.E. Cortes, G. Garcia-Manero, S. Verstovsek, S. Faderl, D.A. Thomas, A. Ferrajoli, S. O’Brien, J.K. Wathen, L.C. Xiao, D.A. Berry, and E.H. Estey. 2003. Adaptive randomized study of idarubicin and cytarabine versus troxacitabine and cytarabine versus troxacitabine and idarubicin in untreated patients 50 years or older with adverse karyotype acute myeloid leukemia. J Clin Oncol 21(9):1722-1727. Grosso, F., R.L. Jones, G.D. Demetri, I.R. Judson, J.Y. Blay, A. Le Cesne, R. Sanfilippo, P. Casieri, P. Collini, P. Dileo, C. Spreafico, S. Stacchiotti, E. Tamborini, J.C. Tercero, J. Jimeno, M. D’Incalci, A. Gronchi, J.A. Fletcher, S. Pilotti, and P.G. Casali. 2007. Efficacy of trabectedin (ecteinascidin-743) in advanced pretreated myxoid liposarcomas: A retrospective study. Lancet Oncol 8(7):595-602. Hamstra, D.A., T.L. Chenevert, B.A. Moffat, T.D. Johnson, C.R. Meyer, S.K. Mukherji, D.J. Quint, S.S. Gebarski, X. Fan, C.I. Tsien, T.S. Lawrence, L. Junck, A. Rehemtulla, and B.D. Ross. 2005. Evaluation of the functional diffusion map as an early biomarker of time-to-progression and overall survival in high-grade glioma. Proc Natl Acad Sci USA 102(46):16759-16764. Hecht, J.R., T. Trarbach, E. Jaeger, J. Hainsworth, R. Wolff, K. Lloyd, G. Bodoky, M. Borner, D. Laurent, and C. Jacques. 2005. A randomized, double-blind, placebo-controlled, phase III study in patients (Pts) with metastatic adenocarcinoma of the colon or rectum receiving first-line chemotherapy with oxaliplatin/5-fluorouracil/leucovorin and PTK787/ZK 222584 or placebo (CONFIRM-1). J Clin Oncol 00 ASCO Annual Meeting Proceedings Part I of II 23(16S):3. Karrison, T.G., D. Huo, and R. Chappell. 2003. A group sequential, response-adaptive design for randomized clinical trials. Control Clin Trials 24(5):506-522. Kola, I., and J. Landis. 2004. Can the pharmaceutical industry reduce attrition rates? Nat Rev Drug Discov 3(8):711-715. The Lewin Group. 2005. Guidance document for implementing effective cancer clinical trials. http://www.c-changetogether.org/pubs/default.asp (accessed Jan. 15, 2008). Linden, H.M., S.A. Stekhova, J.M. Link, J.R. Gralow, R.B. Livingston, G.K. Ellis, P.H. Petra, L.M. Peterson, E.K. Schubert, L.K. Dunnwald, K.A. Krohn, and D.A. Mankoff. 2006. Quantitative fluoroestradiol positron emission tomography imaging predicts response to endocrine treatment in breast cancer. J Clin Oncol 24(18):2793-2799. Ludwig, J.A., and J.N. Weinstein. 2005. Biomarkers in cancer staging, prognosis and treatment selection. Nat Rev Cancer 5(11):845-856.

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0 IMPROVING THE QUALITY OF CANCER CLINICAL TRIALS Lynch, T.J., D.W. Bell, R. Sordella, S. Gurubhagavatula, R.A. Okimoto, B.W. Brannigan, P.L. Harris, S.M. Haserlat, J.G. Supko, F.G. Haluska, D.N. Louis, D.C. Christiani, J. Settleman, and D.A. Haber. 2004. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib. N Engl J Med 350(21):2129-2139. Metz, J.M., C. Coyle, C. Hudson, and M. Hampshire. 2005. An Internet-based cancer clinical trials matching resource. J Med Internet Res 7(3):e24. Morgan, B., A.L. Thomas, J. Drevs, J. Hennig, M. Buchert, A. Jivan, M.A. Horsfield, K. Mross, H.A. Ball, L. Lee, W. Mietlowski, S. Fuxuis, C. Unger, K. O’Byrne, A. Henry, G.R. Cherryman, D. Laurent, M. Dugan, D. Marmé, and W.P. Steward. 2003. Dynamic contrast-enhanced magnetic resonance imaging as a biomarker for the pharmacological response of PTK787/ZK 222584, an inhibitor of the vascular endothelial growth factor receptor tyrosine kinases, in patients with advanced colorectal cancer and liver metastases: Results from two phase I studies. J Clin Oncol 21(21):3955-3964. Mortimer, J.E., F. Dehdashti, B.A. Siegel, K. Trinkaus, J.A. Katzenellenbogen, and M.J. Welch. 2001. Metabolic flare: indicator of hormone responsiveness in advanced breast cancer. J Clin Oncol 19(11):2797-2803. Neve, R.M., K. Chin, J. Fridlyand, J. Yeh, F.L. Baehner, T. Fevr, L. Clark, N. Bayani, J.-P. Coppe, F. Tong, T. Speed, P.T. Spellman, S. DeVries, A. Lapuk, N.J. Wang, W.-L. Kuo, J.L. Stilwell, D. Pinkel, D.G. Albertson, F.M. Waldman, F. McCormick, R.B. Dickson, M.D. Johnson, M. Lippman, S. Ethier, A. Gazdar, and J.W. Gray. 2006. A collection of breast cancer cell lines for the study of functionally distinct cancer subtypes. Cancer Cell 10(6):515-527. Nunn, A.D. 2006. The cost of developing imaging agents for routine clinical use. Invest Radiol 41:206-212. Paez, J.G., P.A. Jänne, J.C. Lee, S. Tracy, H. Greulich, S. Gabriel, P. Herman, F.J. Kaye, N. Lindeman, T.J. Boggon, K. Naoki, H. Sasaki, Y. Fujii, M.J. Eck, W.R. Sellers, B.E. Johnson, and M. Meyerson. 2004. EGFR mutations in lung cancer: Correlation with clinical response to gefitinib therapy. Science 304(5676):1497-1500. Paik, S., S. Shak, G. Tang, C. Kim, J. Baker, M. Cronin, F.L. Baehner, M.G. Walker, D. Watson, T. Park, W. Hiller, E.R. Fisher, D.L. Wickerham, J. Bryant, and N. Wolmark. 2004. A multigene assay to predict recurrence of tamoxifen-treated, node- negative breast cancer. N Engl J Med 351(27):2817-2826. Pao, W., V. Miller, M. Zakowski, J. Doherty, K. Politi, I. Sarkaria, B. Singh, R. Heelan, V. Rusch, L. Fulton, E. Mardis, D. Kupfer, R. Wilson, M. Kris, H. Varmus. 2004. EGF receptor gene mutations are common in lung cancers from “never smokers” and are associated with sensitivity of tumors to gefitinib and erlotinib. Proc Natl Acad Sci USA 101(36):13306-13311. Peto, R. 1985. Discussion of “On the Allocation of Treatments in Sequential Medical Trials” by J.A. Bather and “The Search for Optimality in Clinical Trials.” Int Stat Rev 53(1):31-34. Reddick, W.E., S. Wang, X. Xiong, J.O. Glass, S. Wu, S.C. Kaste, C.B. Pratt, W.H. Meyer, and B.D. Fletcher. 2001. Dynamic magnetic resonance imaging of regional contrast access as an additional prognostic factor in pediatric osteosarcoma. Cancer 91(12):2230- 2237.

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0 WORKSHOP SUMMARY Rischin, D., R.J. Hicks, R. Fisher, D. Binns, J. Corry, S. Porceddu, and L.J. Peters. 2006. Prognostic significance of [18F]-misonidazole positron emission tomography-detected tumor hypoxia in patients with advanced head and neck cancer randomly assigned to chemoradiation with or without tirapazamine: A substudy of Trans-Tasman Radiation Oncology Group Study 98.02. J Clin Oncol 24(13):2098-2104. Schwartz, L.H., M. Mazumdar, L. Wang, A. Smith, S. Marion, D.M. Panicek, and R.J. Motzer. 2003. Response assessment classification in patients with advanced renal cell carcinoma treated on clinical trials. Cancer 98(8):1611-1619. Shukla-Dave, A., H. Hricak, M.W. Kattan, D. Pucar, K. Kuroiwa, H.N. Chen, J. Spector, J.A. Koutcher, K.L. Zakian, and P.T. Scardino. 2007. The utility of magnetic resonance imaging and spectroscopy for predicting insignificant prostate cancer: an initial analysis. BJU Int 99(4):786-793. Sjöblom, T., S. Jones, L.D. Wood, D.W. Parsons, J. Lin, T.D. Barber, D. Mandelker, R.J. Leary, J. Ptak, N. Silliman, S. Szabo, P. Buckhaults, C. Farrell, P. Meeh, S.D. Markowitz, J. Willis, D. Dawson, J.K. Willson, A.F. Gazdar, J. Hartigan, L. Wu, C. Liu, G. Parmigiani, B.H. Park, K.E. Bachman, N. Papadopoulos, B. Vogelstein, K.W. Kinzler, and V.E. Velculescu. 2006. The consensus coding sequences of human breast and colorectal cancers. Science 314(5797):268-274. Steward, W.P., A. Thomas, B. Morgan, B. Wiedenmann, C. Bartel, U. Vanhoefer, T. Trarbach, U. Junker, D. Laurent, and D. Lebwohl. 2004. Expanded phase I/II study of PTK787/ ZK 222584 (PTK/ZK), a novel, oral angiogenesis inhibitor, in combination with FOLFOX-4 as first-line treatment for patients with metastatic colorectal cancer. J Clin Oncol, 00 ASCO Annual Meeting Proceedings (Post-Meeting Edition) 22(14S):3556. Taguchi, F., B. Solomon, V. Gregorc, H. Roder, R. Gray, K. Kasahara, M. Nishio, J. Brahmer, A. Spreafico, V. Ludovini, P.P. Massion, R. Dziadziuszko, J. Schiller, J. Grigorieva, M. Tsypin, S.W. Hunsucker, R. Caprioli, M.W. Duncan, F.R. Hirsch, P.A. Bunn, Jr., and D.P. Carbone. 2007. Mass spectrometry to classify non-small-cell lung cancer patients for clinical outcome after treatment with epidermal growth factor receptor tyrosine kinase inhibitors: a multicohort cross-institutional study. J Natl Cancer Inst 99(11):838-846. Tomaszewski, J.E. 2004. Multi-species toxicology approaches for oncology drugs: The U.S. perspective. Eur J Cancer 40(6):907-913. Tsiatis, A.A. and C. Mehta. 2003. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 90(2):367-378. Tufts Center for the Study of Drug Development. 2007. Outlook 00. http://csdd.tufts. edu/InfoServices/OutlookPDFs/Outlook2007.pdf (accessed Jan. 15, 2008). Von Hoff, D.D., R. Penny, S. Shack, E. Campbell, D. Taverna, M. Borad, D. Love, J. Trent, and M. Bittner. 2006. Frequency of potential therapeutic targets identified by immunohistochemistry (IHC) and DNA microarray (DMA) in tumors from patients who have progressed on multiple therapeutic agents. J Clin Oncol, 00 ASCO Annual Meeting Proceedings Part I 24(18S):3071. Wiener, M.B., H.M. Newman, and E.A. Spradley. 2007. Revolutionizing oncology patient enrollment in clinical trials: Just-in-time approach. J Clin Oncol, 00 ASCO Annual Meeting Proceedings Part I 25(18S):6577.

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0 IMPROVING THE QUALITY OF CANCER CLINICAL TRIALS Yildiz, P.B., Y. Shyr, J.S. Rahman, N.R. Wardwell, L.J. Zimmerman, B. Shakhtour, W.H. Gray, S. Chen, M. Li, H. Roder, D.C. Liebler, W.L. Bigbee, J.M. Siegfried, J.L. Weissfeld, A.L. Gonzalez, M. Ninan, D.H. Johnson, D.P. Carbone, R.M. Caprioli, and P.P. Massion. 2007. Diagnostic accuracy of MALDI mass spectrometric analysis of unfractionated serum in lung cancer. J Thorac Oncol 2(10):893-901. Zhang, W., A.F. Purchio, K. Chen, J. Wu, L. Lu, R. Coffee, P.R. Contag, and D.B. West. 2003. A transgenic mouse model with a luciferase reporter for studying in vivo transcriptional regulation of the human CYP3A4 gene. Drug Metab Dispos 31(8):1054-1064.