A five-gene hedgehog signature developed as a patient preselection tool for hedgehog inhibitor therapy in medulloblastoma

Clinical Cancer Research

Volumn 21, Issue 3, 2015, Pages 585-593

  Shou, Yaping ^a,r   Robinson, Douglas M ^a   Amakye, Dereck D ^b,s   Rose, Kristine L ^b   Cho, Yoon Jae ^c   Ligon, Keith L ^d,e,f   Sharp, Thad ^a   Haider, Asifa S ^b   Bandaru, Raj ^a,t   Ando, Yuichi ^g   Geoerger, Birgit ^h   Doz, Franҫois ⁱ   Ashley, David M ^j   Hargrave, Darren R ^k   Casanova, Michela ^l   Tawbi, Hussein A ^m   Rodon, Jordi ⁿ   Thomas, Anne L ^o   Mita, Alain C ^p   MacDonald, Tobey J ^q   Kieran, Mark W ^d   more..

^a NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH   (United States)

^b NOVARTIS PHARMA AG   (Switzerland)

^c STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d BOSTON CHILDREN S HOSPITAL   (United States)

^e BRIGHAM AND WOMEN S HOSPITAL   (United States)

^f DANA FARBER CANCER INSTITUTE   (United States)

^g NAGOYA UNIVERSITY HOSPITAL   (Japan)

^h INSTITUT GUSTAVE ROUSSY   (France)

ⁱ UNIVERSITÉ PARIS DESCARTES   (France)

^j BARWON HEALTH   (Australia)

^k GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^l NATIONAL CANCER INSTITUTE   (Italy)

^m UNIVERSITY OF PITTSBURGH CANCER INSTITUTE   (United States)

ⁿ UNIVERSITAT AUTÒNOMA DE BARCELONA   (Spain)

^o UNIVERSITY OF LEICESTER   (United Kingdom)

^p University of Texas Health Science Center at San Antonio   (United States)

^q EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^r TAKEDA PHARMACEUTICAL COMPANY LIMITED   (Japan)

^s BAYER CORPORATION   (United States)

^t ASTRAZENECA   (United Kingdom)

more..

Author keywords

[No Author keywords available]

Indexed keywords

SONIDEGIB; SPHINGOSINE KINASE 1; TRANSCRIPTION FACTOR GLI1; TRANSCRIPTION FACTOR OTX2;

ADULT; ARTICLE; CANCER CHEMOTHERAPY; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG EFFECT; DRUG RESPONSE; GENE EXPRESSION PROFILING; GENETIC ASSOCIATION; GENETIC MODEL; GENETIC SELECTION; GLI1 GENE; HUMAN; HUMAN TISSUE; MEDULLOBLASTOMA; OTX2 GENE; PDLIM3 GENE; PREDICTIVE VALUE; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SENSITIVITY AND SPECIFICITY; SHROOM2 GENE; SPHK1 GENE; TUMOR GENE;

EID: 84964225093     PISSN: 10780432     EISSN: 15573265     Source Type: Journal    
DOI: 10.1158/1078-0432.CCR-13-1711     Document Type: Article

References (35)

1. CBTRUS. CBTRUS statistical report: primary brain and central nervous system tumors diagnosed in the United States in 2004-2007. Available from: http://www.cbtrus.org/2011-NPCR-SEER/WEB-0407-Report-3-3-2011.pdf. Accessed October 2012.
2. Packer RJ, Vezina G. Management of and prognosis with medulloblastoma: therapy at a crossroads. Arch Neurol 2008;65:1419-24.
3. Packer RJ, Cogen P, Vezina G, Rorke LB. Medulloblastoma: clinical and biologic aspects. Neuro Oncol 1999;1:232-50.
4. Thompson MC, Fuller C, Hogg TL, Dalton J, Finkelstein D, Lau CC, et al. Genomics identifies medulloblastoma subgroups that are enriched for specific genetic alterations. J Clin Oncol 2006;24:1924-31.
5. Kool M, Koster J, Bunt J, Hasselt NE, Lakeman A, van Sluis P, et al. Integrated genomics identi fies five medulloblastoma subtypes with distinct genetic profiles, pathway signatures and clinicopathological features. PLoS One 2008;3:e3088.
6. Northcott PA, Korshunov A, Witt H, Hielscher T, Eberhart CG, Mack S, et al. Medulloblastoma comprises four distinct molecular variants. J Clin Oncol 2011;29:1408-14.
7. Cho YJ, Tsherniak A, Tamayo P, Santagata S, Ligon A, Greulich H, et al. Integrative genomic analysis of medulloblastoma identi fies a molecular subgroup that drives poor clinical outcome. J Clin Oncol 2011;29:1424-30.
8. Taylor MD, Northcott PA, Korshunov A, Remke M, Cho YJ, Clifford SC, et al. Molecular subgroups of medulloblastoma:the current consensus. Acta Neuropathol 2012;123:465-72.
9. Lee Y, Miller HL, Jensen P, Hernan R, Connelly M, Wetmore C, et al. A molecular fingerprint for medulloblastoma. Cancer Res 2003;63:5428-37.
10. Slade I, Murray A, Hanks S, Kumar A, Walker L, Hargrave D, et al. Heterogeneity of familial medulloblastoma and contribution of germline PTCH1 and SUFU mutations to sporadic medulloblastoma. Fam Cancer 2011;10:337-42.
11. Teglund S, Toftgard R. Hedgehog beyond medulloblastoma and basal cell carcinoma. Biochim Biophys Acta 2010;1805:181-208.
12. Rubin LL, de Sauvage FJ. Targeting the Hedgehog pathway in cancer. Nat Rev Drug Discov 2006;5:1026-33.
13. Pan S, Wu X, Jiang J, Gao W, Wan Y, Cheng D, et al. Discovery of NVPLDE225, a potent and selective smoothened antagonist. ACS Med Chem Lett 2010;1:130-4.
14. Buonamici S, Williams J, Morrissey M, Wang A, Guo R, Vattay A, et al. Interfering with resistance to smoothened antagonists by inhibition of the PI3K pathway in medulloblastoma. Sci Transl Med 2010;2:51ra70.
15. Rodon J, Tawbi H, Thomas A, Dummer R, Stoller R, Turtschi CP, et al. A phase 1, multicenter, open-label, first-in-human, dose-escalation study of the oral hedgehog inhibitor sonidegib (LDE225) in patients with advanced solid tumors. Clin Cancer Res 2014;20:1900-9.
16. Geoerger B, Aerts I, Casanova M, Chisholm J, Hargrave D, Leary SES, et al. A phase I/II study of LDE225, a smoothened (Smo) antagonist, in pediatric patients with recurrent medulloblastoma (MB) or other solid tumors. J Clin Oncol 2012 ASCO Ann Mtg Proc 2012;30:abstr 9519.
17. Lindstrom E, Shimokawa T, Toftgard R, Zaphiropoulos PG. PTCH mutations: distribution and analyses. Hum Mutat 2006;27:215-9.
18. Kim K, Zakharkin SO, Allison DB. Expectations, validity, and reality in gene expression profiling. J Clin Epidemiol 2010;63:950-9.
19. ClinicalTrials.gov. [homepage on the Internet]. Dose finding and safety of oral LDE225 in patients with advanced solid tumors. Available from: http://ClinicalTrials.gov/show/NCT00880308. Accessed October 2012; last updated August 2014.
20. ClinicalTrials.gov. [homepage on the Internet]. A dose finding and safety study of oral LDE225 in children. Available from: http://ClinicalTrials.gov/show/NCT01125800. Accessed October 2012; last updated August 2014.
21. ClinicalTrials.gov. [homepage on the Internet]. An East Asian study of LDE225. Available from: http://ClinicalTrials.gov/show/NCT01208831. Accessed October 2012; last updated August 2014.
22. Tibshirani R. Regression shrinkage and selection via the lasso. J R Stat Soc Series B Stat Methodol 1996;58:267-88.
23. Hoerl A, Kennard R. Ridge regression: applications to nonorthogonal problems. Technometrics 1970;12:69-82.
24. Wu Z, Irizarry R, Gentleman R, Murillo F, Spencer F. A model-based background adjustment for oligonucleotide expression arrays. J Am Stat Assoc 2004;99:909-17.
25. Friedman J, Hastie T, Tibshirani R. Sparse inverse covariance estimation with the graphical lasso. Biostatistics 2008;9:432-41.
26. Castaldi PJ, Dahabreh IJ, Ioannidis JP. An empirical assessment of validation practices for molecular classifiers. Brief Bioinform 2011;12:189-202.
27. Scales SJ, de Sauvage FJ. Mechanisms of Hedgehog pathway activation in cancer and implications for therapy. Trends Pharmacol Sci 2009;30:303-12.
28. Bonifas JM, Pennypacker S, Chuang PT, McMahon AP, Williams M, Rosenthal A, et al. Activation of expression of hedgehog target genes in basal cell carcinomas. J Invest Dermatol 2001;116:739-42.
29. Pomies P, Pashmforoush M, Vegezzi C, Chien KR, Auffray C, Beckerle MC. The cytoskeleton-associated PDZ-LIMprotein, ALP, acts on serum response factor activity to regulate muscle differentiation. Mol Biol Cell 2007;18:1723-33.
30. Farber MJ, Rizaldy R, Hildebrand JD. Shroom2 regulates contractility to control endothelial morphogenesis. Mol Biol Cell 2011;22:795-805.
31. Van Brocklyn JR, Jackson CA, Pearl DK, Kotur MS, Snyder PJ, Prior TW. Sphingosine kinase-1 expression correlates with poor survival of patients with glioblastoma multiforme: roles of sphingosine kinase isoforms in growth of glioblastoma cell lines. J Neuropathol Exp Neurol 2005;64:695-705.
32. Adamson DC, Shi Q, Wortham M, Northcott PA, Di C, Duncan CG, et al. OTX2 is critical for the maintenance and progression of Shh-independent medulloblastomas. Cancer Res 2010;70:181-91.
33. Therasse P, Arbuck SG, Eisenhauer EA, Wanders J, Kaplan RS, Rubinstein L, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst 2000;92:205-16.
34. Wen PY, Macdonald DR, Reardon DA, Cloughesy TF, Sorensen AG, Galanis E, et al. Updated response assessment criteria for high-grade gliomas: response assessment in Neuro-Oncology Working Group. J Clin Oncol 2010;28:1963-72.
35. Clopper CJ, Pearson ES. The use of con fidence or fiducial limits illustrated in the case of the binomial. Biometrika 1934;26:404-13.