Phase I/II study of erlotinib and temsirolimus for patients with recurrent malignant gliomas: North American Brain Tumor Consortium trial 04-02

Neuro-Oncology

Volumn 16, Issue 4, 2014, Pages 567-578

  Wen, Patrick Y ^a   Chang, Susan M ^b   Lamborn, Kathleen R ^b   Kuhn, John G ^c   Norden, Andrew D ^a   Cloughesy, Timothy F ^d   Robins, H Ian ^e   Lieberman, Frank S ^f   Gilbert, Mark R ^g   Mehta, Minesh P ^e   Drappatz, Jan ^a   Groves, Morris D ^g   Santagata, Sandro ^a   Ligon, Azra H ^a   Yung, W K Alfred ^g   Wright, John J ^h   Dancey, Janet ^h   Aldape, Kenneth D ^g   Prados, Michael D ^b   Ligon, Keith L ^a  

^a BRIGHAM AND WOMEN S HOSPITAL   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

^c UNIVERSITY OF TEXAS HEALTH SCIENCE CENTER AT SAN ANTONIO   (United States)

^d UNIVERSITY OF CALIFORNIA   (United States)

^e UNIVERSITY OF WISCONSIN MADISON   (United States)

^f UNIVERSITY OF PITTSBURGH MEDICAL CENTER   (United States)

^g UNIVERSITY OF TEXAS MD ANDERSON CANCER CENTER   (United States)

^h NATIONAL CANCER INSTITUTE   (United States)

Author keywords

Anaplastic glioma; Clinical trial; Epidermal growth factor; Erlotinib; Glioblastoma; Temsirolimus

Indexed keywords

EPIDERMAL GROWTH FACTOR RECEPTOR; ERLOTINIB; MAMMALIAN TARGET OF RAPAMYCIN; OROSOMUCOID; OSI 420; PHOSPHATIDYLINOSITOL 3,4,5 TRISPHOSPHATE 3 PHOSPHATASE; PROTEIN KINASE B; S6 KINASE; TEMSIROLIMUS; UNCLASSIFIED DRUG;

ABNORMAL LABORATORY RESULT; ADULT; AGED; ANEMIA; ANOREXIA; ANTINEOPLASTIC ACTIVITY; AREA UNDER THE CURVE; ARTICLE; ASTROCYTOMA; CANCER RECURRENCE; CANCER SURVIVAL; DEHYDRATION; DIARRHEA; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG TREATMENT FAILURE; DRUG TUMOR LEVEL; DRY SKIN; FATIGUE; FEMALE; FLUORESCENCE IN SITU HYBRIDIZATION; GENE AMPLIFICATION; GENOTYPE; GLIOBLASTOMA; GRANULOCYTOPENIA; HEART MUSCLE ISCHEMIA; HUMAN; HUMAN TISSUE; HYPERCHOLESTEROLEMIA; HYPERGLYCEMIA; HYPERTRIGLYCERIDEMIA; HYPOCALCEMIA; HYPONATREMIA; HYPOPHOSPHATEMIA; HYPOTENSION; INFECTION; LEUKOPENIA; LIMB PAIN; LIVER DYSFUNCTION; LYMPHOCYTOPENIA; MAJOR CLINICAL STUDY; MALE; MAXIMUM PLASMA CONCENTRATION; MAXIMUM TOLERATED DOSE; MIDDLE AGED; MUCOSA INFLAMMATION; MULTIPLE CYCLE TREATMENT; NAUSEA; OLIGODENDROGLIOMA; OUTCOME ASSESSMENT; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PROGRESSION FREE SURVIVAL; PROTEIN EXPRESSION; PROTEIN PHOSPHORYLATION; PRURITUS; RASH; RETINOPATHY; THROMBOCYTOPENIA; VOMITING; WEAKNESS;

ANAPLASTIC GLIOMA; CLINICAL TRIAL; EPIDERMAL GROWTH FACTOR; ERLOTINIB; GLIOBLASTOMA; TEMSIROLIMUS;

ADULT; AGED; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; BRAIN NEOPLASMS; FEMALE; FOLLOW-UP STUDIES; GLIOMA; HUMANS; MALE; MIDDLE AGED; NEOPLASM RECURRENCE, LOCAL; NEOPLASM STAGING; PROGNOSIS; QUINAZOLINES; SIROLIMUS; SURVIVAL RATE; TISSUE DISTRIBUTION; YOUNG ADULT;

EID: 84896933653     PISSN: 15228517     EISSN: 15235866     Source Type: Journal    
DOI: 10.1093/neuonc/not247     Document Type: Article

References (49)

1. CBTRUS Statistical Report: Primary Brain and Central Nervous System Tumors Diagnosed in the United States in 2004-2007. Hinsdale, IL: Central Brain Tumor Registry of the United States; 2011.
2. Wen PY, Kesari S. Malignant gliomas in adults. N Engl J Med. 2008;359: 492-507.
3. Wong ET, Hess KR, Gleason MJ, et al. Outcomes and prognostic factors in recurrent glioma patients enrolled onto phase II clinical trials. J Clin Oncol. 1999;17:2572-2578.
4. Nicholas MK, Lukas RV, Jafri NF, et al. Epidermal growth factor receptor-mediated signal transduction in the development and therapy of gliomas. Clin Cancer Res. 2006;12:7261-7270.
5. Frederick L,Wang XY, Eley G, et al. Diversity and frequency of epidermal growth factor receptormutations inhumanglioblastomas. Cancer Res. 2000;60:1383-1387.
6. Dunn GP,Rinne ML,Wykosky J, et al.Emerginginsightsintothemolecular and cellular basis of glioblastoma. Genes Dev. 2012;26:756-784.
7. Bostrom J, Cobbers JM, Wolter M, et al. Mutation of the PTEN (MMAC1) tumor suppressor gene in a subset of glioblastomas but not in meningiomaswith loss of chromosomearm10q. Cancer Res. 1998;58:29-33.
8. Wang SI, Puc J, Li J, et al. Somatic mutations of PTEN in glioblastoma multiforme. Cancer Res. 1997;57:4183-4186.
9. Li J, Yen C, Liaw D, et al. PTEN, a putative protein tyrosine phosphatase gene mutated in human brain, breast, and prostate cancer. Science. 1997;275:1943-1947.
10. Comprehensive genomic characterization defines human glioblastoma genes and core pathways. Nature. 2008;455:1061-1068.
11. Omuro AM, Faivre S,Raymond E. Lessons learned in the development of targeted therapy for malignant gliomas. Mol Cancer Ther. 2007;6: 1909-1919.
12. Raizer JJ, Abrey LE, Lassman AB, et al. A phase II trial of erlotinib in patients with recurrent malignant gliomas and nonprogressive glioblastoma multiforme postradiation therapy. Neuro Oncol. 2010; 12:95-103.
13. Yung WK, Vredenburgh JJ, Cloughesy TF, et al. Safety and efficacy of erlotinib in first-relapse glioblastoma: a phase II open-label study. Neuro Oncol. 2010;12:1061-1070.
14. Mellinghoff IK,WangMY, Vivanco I, et al. Moleculardeterminants of the responseof glioblastomastoEGFRkinase inhibitors.NEngl JMed. 2005; 353:2012-2024.
15. Haas-Kogan DA, Prados MD, Tihan T, et al. Epidermal growth factor receptor, protein kinase B/Akt, and glioma response to erlotinib. J Natl Cancer Inst. 2005;97:880-887.
16. Wang MY, Lu KV, Zhu S, et al. Mammaliantarget of rapamycin inhibition promotes response to epidermal growth factor receptor kinase inhibitors in PTEN-deficient and PTEN-intact glioblastoma cells. Cancer Res. 2006;66:7864-7869.
17. van den Bent MJ, Brandes AA, Rampling R, et al. Randomized phase II trial of erlotinib versus temozolomide or carmustine in recurrent glioblastoma: EORTC brain tumor group study 26034. J Clin Oncol. 2009;27:1268-1274.
18. de Groot JF, Gilbert MR, Aldape K, et al. Phase II studyof carboplatin and erlotinib (Tarceva, OSI-774) in patients with recurrent glioblastoma. J Neurooncol. 2008;90:89-97.
19. Franceschi E, Cavallo G, Lonardi S, et al. Gefitinib in patients with progressive high-grade gliomas: a multicentre phase II study by Gruppo Italiano Cooperativo di Neuro-Oncologia (GICNO). Br J Cancer. 2007;96:1047-1051.
20. Lassman AB, Rossi MR, Raizer JJ, et al. Molecular study of malignant gliomas treated with epidermal growth factor receptor inhibitors: tissue analysis from North American Brain Tumor Consortium Trials 01-03 and 00-01. Clin Cancer Res. 2005;11:7841-7850.
21. Geoerger B, Kerr K, Tang CB, et al. Antitumor activity of the rapamycin analog CCI-779 in human primitive neuroectodermal tumor/ medulloblastoma models as single agent and in combination chemotherapy. Cancer Res. 2001;61:1527-1532.
22. Chang SM, Wen P, Cloughesy T, et al. Phase II study of CCI-779 in patients with recurrent glioblastoma multiforme. Invest New Drugs. 2005;23:357-361.
23. Galanis E, Buckner JC, Maurer MJ, et al. Phase II trial of temsirolimus (CCI-779) in recurrent glioblastoma multiforme: a North Central Cancer Treatment Group Study. J Clin Oncol. 2005;23:5294-5304.
24. Rao RD, Mladek AC, Lamont JD, et al. Disruption of parallel and converging signaling pathways contributes to the synergistic antitumor effects of simultaneous mTOR and EGFR inhibition in GBM cells. Neoplasia. 2005;7:921-929.
25. Fan QW, Cheng CK, Nicolaides TP, et al. A dual phosphoinositide- 3-kinase alpha/mTOR inhibitor cooperates with blockade of epidermal growth factor receptor in PTEN-mutant glioma. Cancer Res. 2007;67:7960-7965.
26. Reardon DA, Quinn JA, Vredenburgh JJ, et al. Phase 1 trial of gefitinib plus sirolimus in adults with recurrent malignant glioma. Clin Cancer Res. 2006;12:860-868.
27. Doherty L, Gigas DC, Kesari S, et al. Pilot study of the combination of EGFR and mTOR inhibitors in recurrent malignant gliomas. Neurology. 2006;67:156-158.
28. Macdonald DR, Cascino TL, Schold SC Jr, et al. Response criteria for phase II studies of supratentorial malignant glioma. J Clin Oncol. 1990;8:1277-1280.
29. Hamilton M, Wolf JL, Rusk J, et al. Effects of smoking on the pharmacokinetics of erlotinib. Clin Cancer Res. 2006;12:2166-2171.
30. Kuhn JG, Chang SM, Wen PY, et al. Pharmacokinetic and tumor distribution characteristics of temsirolimus in patients with recurrent malignant glioma. Clin Cancer Res. 2007;13:7401-7406.
31. Yoshimoto K, Dang J, Zhu S, et al. Development of a real-time RT-PCR assay for detecting EGFRvIII in glioblastoma samples. Clin Cancer Res. 2008;14:488-493.
32. Snuderl M, Eichler AF, Ligon KL, et al. Polysomy for chromosomes 1 and 19 predicts earlier recurrence in anaplastic oligodendrogliomas with concurrent 1p/19q loss. Clin Cancer Res. 2009;15:6430-6437.
33. Hidalgo M, Siu LL, Nemunaitis J, et al. Phase I and pharmacologic study of OSI-774, an epidermal growth factor receptor tyrosine kinase inhibitor, in patients with advanced solid malignancies. J Clin Oncol. 2001;19:3267-3279.
34. Atkins MB, Hidalgo M, Stadler WM, et al. Randomized phase II study of multiple dose levels of CCI-779, a novel mammalian target of rapamycin kinase inhibitor, in patients with advanced refractory renal cell carcinoma. J Clin Oncol. 2004;22:909-918.
35. Hidalgo M, Buckner JC, Erlichman C, et al. A phase I and pharmacokinetic study of temsirolimus (CCI-779) administered intravenously daily for 5 days every 2 weeks to patients with advanced cancer. Clin Cancer Res. 2006;12:5755-5763.
36. Fischer I, de la Cruz C, Rivera AL, Aldape K. Utility of chromogenic in situ hybridization (CISH) for detection of EGFR amplification in glioblastoma: comparison with fluorescence in situ hybridization (FISH). Diagn Mol Pathol. 2008;17:227-230.
37. Stommel JM, Kimmelman AC, Ying H, et al. Coactivation of receptor tyrosine kinases affects the response of tumor cells to targeted therapies. Science. 2007;318:287-290.
38. Salvesen HB, Carter SL, Mannelqvist M, et al. Integrated genomic profiling of endometrial carcinoma associates aggressive tumors with indicators of PI3 kinase activation. Proc Natl Acad Sci U S A. 2009;106:4834-4839.
39. Cloughesy TF, Yoshimoto K, Nghiemphu P, et al. Antitumor activity of rapamycin in a phase I trial for patients with recurrent PTEN-deficient glioblastoma. PLoS Med. 2008;5:e8.
40. Kreisl TN, Lassman AB, Mischel PS, et al. A pilot study of everolimus and gefitinib in the treatment of recurrent glioblastoma(GBM). JNeurooncol. 2009;92:99-105.
41. Reardon DA, Desjardins A, Vredenburgh JJ, et al. Phase2trial of erlotinib plus sirolimus in adults with recurrent glioblastoma. J Neurooncol. 2010;96:219-230.
42. Vivanco I, Robins HI, Rohle D, et al. Differential sensitivity of gliomaversus lung cancer-specific EGFR mutations to EGFR kinase inhibitors. Cancer Discov. 2012;2:458-471.
43. O'Reilly KE, Rojo F, She QB, et al. mTOR inhibition induces upstream receptor tyrosine kinase signaling and activates Akt. Cancer Res. 2006;66:1500-1508.
44. Sun SY, Rosenberg LM, Wang X, et al. Activation of Akt and eIF4E survival pathways by rapamycin-mediated mammalian target of rapamycin inhibition. Cancer Res. 2005;65:7052-7058.
45. Carracedo A, Ma L, Teruya-Feldstein J, et al. Inhibition ofmTORC1 leads to MAPK pathway activation through a PI3K-dependent feedback loop in human cancer. J Clin Invest. 2008;118:3065-3074.
46. Wang X, Hawk N, Yue P, et al. Overcoming mTOR inhibition-induced paradoxical activation of survival signaling pathways enhances mTOR inhibitors' anticancer efficacy. Cancer Biol Ther. 2008;7:1952-1958.
47. Zhang H, Bajraszewski N, Wu E, et al. PDGFRs are critical for PI3 K/Akt activation and negatively regulated by mTOR. J Clin Invest. 2007;117: 730-738.
48. Sarbassov DD, Guertin DA, Ali SM, et al. Phosphorylation and regulation of Akt/PKB by the rictor-mTOR complex. Science. 2005;307: 1098-1101.
49. Wen PY, Lee EQ, Reardon DA, et al. Current clinical development of PI3 K pathway inhibitors in glioblastoma. Neuro Oncol. 2012;14: 819-829.