SHH inhibitors for the treatment of medulloblastoma

References

• Eberhart CG, Kepner JL, Goldthwaite PT, et al. Histopathologic grading of medulloblastomas: a Pediatric Oncology Group study. Cancer 2002;94(2):552-60
   PubMed Web of Science ®Google Scholar
• Ries LAG SM, Gurney JG, Linet M, et al. editors. Cancer Incidence and Survival among Children and Adolescents: United States SEER Program 1975-1995. National Cancer Institute, SEER Program, NIH Pub, Bethesda, MD, USA; 1999. p. 99-4649
   Google Scholar
• Northcott PA, Jones DT, Kool M, et al. Medulloblastomics: the end of the beginning. Nat Rev Cancer 2012;12(12):818-34
   PubMed Web of Science ®Google Scholar
• Rice JS, Fischer DS. Blue rubber-bleb nevus syndrome. Generalized cavernous hemangiomatosis or venous hamartoma with medulloblastoma of the cerebellum: case report and review of the literature. Arch Dermatol 1962;86:503-11
   PubMedGoogle Scholar
• Louis DN, Ohgaki H, Wiestler OD, et al. The 2007 WHO classification of tumours of the central nervous system. Acta Neuropathol 2007;114(2):97-109
   PubMed Web of Science ®Google Scholar
• Packer RJ, Vezina G. Management of and prognosis with medulloblastoma: therapy at a crossroads. Arch Neurol 2008;65(11):1419-24
   PubMedGoogle Scholar
• Rutkowski S, von Hoff K, Emser A, et al. Survival and prognostic factors of early childhood medulloblastoma: an international meta-analysis. J Clin Oncol 2010;28(33):4961-8
   PubMed Web of Science ®Google Scholar
• Moxon-Emre I, Bouffet E, Taylor MD, et al. Impact of craniospinal dose, boost volume, and neurologic complications on intellectual outcome in patients with medulloblastoma. J Clin Oncol 2014;32(17):1760-8
   PubMed Web of Science ®Google Scholar
• Piscione PJ, Bouffet E, Mabbott DJ, et al. Physical functioning in pediatric survivors of childhood posterior fossa brain tumors. Neuro Oncol 2014;16(1):147-55
   PubMedGoogle Scholar
• Palmer SL, Armstrong C, Onar-Thomas A, et al. Processing speed, attention, and working memory after treatment for medulloblastoma: an international, prospective, and longitudinal study. J Clin Oncol 2013;31(28):3494-500
   PubMed Web of Science ®Google Scholar
• Gajjar A, Pfister SM, Taylor MD, Gilbertson RJ. Molecular insights into pediatric brain tumors have the potential to transform therapy. Clin Cancer Res 2014;20(22):5630-40
   PubMed Web of Science ®Google Scholar
• Morfouace M, Shelat A, Jacus M, et al. Pemetrexed and gemcitabine as combination therapy for the treatment of Group3 medulloblastoma. Cancer Cell 2014;25(4):516-29
   PubMed Web of Science ®Google Scholar
• Northcott PA, Shih DJ, Peacock J, et al. Subgroup-specific structural variation across 1,000 medulloblastoma genomes. Nature 2012;488(7409):49-56
   PubMed Web of Science ®Google Scholar
• Taylor MD, Northcott PA, Korshunov A, et al. Molecular subgroups of medulloblastoma: the current consensus. Acta Neuropathol 2012;123(4):465-72
   PubMed Web of Science ®Google Scholar
• Gajjar AJ, Robinson GW. Medulloblastoma-translating discoveries from the bench to the bedside. Nat Rev Clin Oncol 2014;11(12):714-22
   PubMed Web of Science ®Google Scholar
• Remke M, Ramaswamy V, Taylor MD. Medulloblastoma molecular dissection: the way toward targeted therapy. Curr Opin Oncol 2013;25(6):674-81
   PubMed Web of Science ®Google Scholar
• Northcott PA, Korshunov A, Pfister SM, Taylor MD. The clinical implications of medulloblastoma subgroups. Nat Rev Neurol 2012;8(6):340-51
   PubMed Web of Science ®Google Scholar
• Kool M, Korshunov A, Remke M, et al. Molecular subgroups of medulloblastoma: an international meta-analysis of transcriptome, genetic aberrations, and clinical data of WNT, SHH, Group 3, and Group 4 medulloblastomas. Acta Neuropathol 2012;123(4):473-84
   PubMed Web of Science ®Google Scholar
• Markant SL, Esparza LA, Sun J, et al. Targeting sonic hedgehog-associated medulloblastoma through inhibition of Aurora and Polo-like kinases. Cancer Res 2013;73(20):6310-22
   PubMed Web of Science ®Google Scholar
• Robinson G, Parker M, Kranenburg TA, et al. Novel mutations target distinct subgroups of medulloblastoma. Nature 2012;488(7409):43-8
   PubMed Web of Science ®Google Scholar
• Heussler HS, Suri M. Sonic hedgehog. Mol Pathol 2003;56(3):129-31
   PubMedGoogle Scholar
• Wang X, Venugopal C, Manoranjan B, et al. Sonic hedgehog regulates Bmi1 in human medulloblastoma brain tumor-initiating cells. Oncogene 2012;31(2):187-99
   PubMed Web of Science ®Google Scholar
• Manoranjan B, Venugopal C, McFarlane N, et al. Medulloblastoma stem cells: where development and cancer cross pathways. Pediatric Res 2012;71(4 Pt 2):516-22
   PubMedGoogle Scholar
• Ruiz i Altaba A, Palma V, Dahmane N. Hedgehog-Gli signalling and the growth of the brain. Nat Rev Neurosci 2002;3(1):24-33
   PubMed Web of Science ®Google Scholar
• Teglund S, Toftgard R. Hedgehog beyond medulloblastoma and basal cell carcinoma. Biochim Biophys Acta 2010;1805(2):181-208
   PubMed Web of Science ®Google Scholar
• Varjosalo M, Taipale J. Hedgehog: functions and mechanisms. Genes Dev 2008;22(18):2454-72
   PubMed Web of Science ®Google Scholar
• Hooper JE, Scott MP. Communicating with Hedgehogs. Nat Rev Mol Cell Biol 2005;6(4):306-17
   PubMed Web of Science ®Google Scholar
• Sahebjam S, Siu LL, Razak AA. The utility of hedgehog signaling pathway inhibition for cancer. Oncologist 2012;17(8):1090-9
   PubMed Web of Science ®Google Scholar
• Hahn H, Wojnowski L, Zimmer AM, et al. Rhabdomyosarcomas and radiation hypersensitivity in a mouse model of Gorlin syndrome. Nat Med 1998;4(5):619-22
   PubMed Web of Science ®Google Scholar
• Xie J, Bartels CM, Barton SW, Gu D. Targeting hedgehog signaling in cancer: research and clinical developments. OncoTargets Therapy 2013;6:1425-35
   PubMed Web of Science ®Google Scholar
• Ellison DW, Clifford SC, Gajjar A, Gilbertson RJ. What’s new in neuro-oncology? Recent advances in medulloblastoma. Europ J Paediat Neurol 2003;7(2):53-66
   PubMedGoogle Scholar
• Reifenberger J, Wolter M, Weber RG, et al. Missense mutations in SMOH in sporadic basal cell carcinomas of the skin and primitive neuroectodermal tumors of the central nervous system. Cancer Res 1998;58(9):1798-803
   PubMed Web of Science ®Google Scholar
• Taylor MD, Liu L, Raffel C, et al. Mutations in SUFU predispose to medulloblastoma. Nat Genet 2002;31(3):306-10
   PubMed Web of Science ®Google Scholar
• Gorlin RJ. Nevoid basal cell carcinoma syndrome. Dermatol Clin 1995;13(1):113-25
   PubMed Web of Science ®Google Scholar
• Smith MJ, Beetz C, Williams SG, et al. Germline mutations in SUFU cause Gorlin syndrome-associated childhood medulloblastoma and redefine the risk associated with PTCH1 mutations. J Clin Oncol 2014;32(36):4155-61
   PubMed Web of Science ®Google Scholar
• Brugieres L, Remenieras A, Pierron G, et al. High frequency of germline SUFU mutations in children with desmoplastic/nodular medulloblastoma younger than 3 years of age. J Clin Oncol 2012;30(17):2087-93
   PubMed Web of Science ®Google Scholar
• Shih DJ, Northcott PA, Remke M, et al. Cytogenetic prognostication within medulloblastoma subgroups. J Clin Oncol 2014;32(9):886-96
   PubMed Web of Science ®Google Scholar
• Faria CC, Golbourn BJ, Dubuc AM, et al. Foretinib is effective therapy for metastatic sonic hedgehog medulloblastoma. Cancer Res 2015;75(1):134-46
   PubMed Web of Science ®Google Scholar
• Tang Y, Gholamin S, Schubert S, et al. Epigenetic targeting of Hedgehog pathway transcriptional output through BET bromodomain inhibition. Nat Med 2014;20(7):732-40
   PubMed Web of Science ®Google Scholar
• Chen JK, Taipale J, Cooper MK, Beachy PA. Inhibition of Hedgehog signaling by direct binding of cyclopamine to Smoothened. Genes Dev 2002;16(21):2743-8
   PubMed Web of Science ®Google Scholar
• Taipale J, Chen JK, Cooper MK, et al. Effects of oncogenic mutations in Smoothened and Patched can be reversed by cyclopamine. Nature 2000;406(6799):1005-9
   PubMed Web of Science ®Google Scholar
• Berman DM, Karhadkar SS, Hallahan AR, et al. Medulloblastoma growth inhibition by hedgehog pathway blockade. Science 2002;297(5586):1559-61
   PubMed Web of Science ®Google Scholar
• Tremblay MR, Lescarbeau A, Grogan MJ, et al. Discovery of a potent and orally active hedgehog pathway antagonist (IPI-926). J Med Chem 2009;52(14):4400-18
   PubMed Web of Science ®Google Scholar
• Jimeno A, Weiss GJ, Miller WHJr, et al. Phase I study of the Hedgehog pathway inhibitor IPI-926 in adult patients with solid tumors. Clin Cancer Res 2013;19(10):2766-74
   PubMed Web of Science ®Google Scholar
• Campbell VT, Nadesan P, Ali SA, et al. Hedgehog pathway inhibition in chondrosarcoma using the smoothened inhibitor IPI-926 directly inhibits sarcoma cell growth. Mol Cancer Ther 2014;13(5):1259-69
   PubMed Web of Science ®Google Scholar
• Lee MJ, Hatton BA, Villavicencio EH, et al. Hedgehog pathway inhibitor saridegib (IPI-926) increases lifespan in a mouse medulloblastoma model. Proc Natl Acad Sci U S A 2012;109(20):7859-64
   PubMed Web of Science ®Google Scholar
• Sekulic A, Migden MR, Oro AE, et al. Efficacy and safety of vismodegib in advanced basal-cell carcinoma. Eng J Med 2012;366(23):2171-9
   PubMed Web of Science ®Google Scholar
• Berlin J, Bendell JC, Hart LL, et al. A randomized phase II trial of vismodegib versus placebo with FOLFOX or FOLFIRI and bevacizumab in patients with previously untreated metastatic colorectal cancer. Clin Cancer Res 2013;19(1):258-67
   PubMedGoogle Scholar
• Kim EJ, Sahai V, Abel EV, et al. Pilot clinical trial of hedgehog pathway inhibitor GDC-0449 (vismodegib) in combination with gemcitabine in patients with metastatic pancreatic adenocarcinoma. Clin Cancer Res 2014;20(23):5937-45
   PubMed Web of Science ®Google Scholar
• Rudin CM, Hann CL, Laterra J, et al. Treatment of medulloblastoma with hedgehog pathway inhibitor GDC-0449. Eng J Med 2009;361(12):1173-8
   PubMed Web of Science ®Google Scholar
• Gajjar A, Stewart CF, Ellison DW, et al. Phase I study of vismodegib in children with recurrent or refractory medulloblastoma: a pediatric brain tumor consortium study. Clin Cancer Res 2013;19(22):6305-12
   PubMed Web of Science ®Google Scholar
• A clinical and molecular risk-directed therapy for newly diagnosed medulloblastoma. Available from: https://clinicaltrials.gov/ct2/show/NCT01878617
   Google Scholar
• Pan S, Wu X, Jiang J, et al. Discovery of NVP-LDE225, a potent and selective smoothened antagonist. ACS Med Chem Lett 2010;1(3):130-4
   PubMed Web of Science ®Google Scholar
• Buonamici S, Williams J, Morrissey M, et al. Interfering with resistance to smoothened antagonists by inhibition of the PI3K pathway in medulloblastoma. Sci Transl Med 2010;2(51):51ra70
   PubMed Web of Science ®Google Scholar
• D’Amato C, Rosa R, Marciano R, et al. Inhibition of Hedgehog signalling by NVP-LDE225 (Erismodegib) interferes with growth and invasion of human renal cell carcinoma cells. Br J Cancer 2014;111(6):1168-79
   PubMed Web of Science ®Google Scholar
• Ishii T, Shimizu Y, Nakashima K, et al. Inhibition mechanism exploration of investigational drug TAK-441 as inhibitor against Vismodegib-resistant Smoothened mutant. Eur J Pharmacol 2014;723:305-13
   PubMed Web of Science ®Google Scholar
• Goldman J, Eckhardt SG, Borad MJ, et al. Phase I Dose-escalation trial of the oral investigational hedgehog signaling pathway inhibitor TAK-441 in patients with advanced solid tumors. Clin Cancer Res 2015;21(5):1002-9
   PubMed Web of Science ®Google Scholar
• Wagner AJ, Messersmith WA, Shaik MN, et al. A Phase I Study of PF-04449913, an oral hedgehog inhibitor, in patients with advanced solid tumors. Clin Cancer Res 2015;21(5):1044-51
   PubMed Web of Science ®Google Scholar
• Lin TL, Matsui W. Hedgehog pathway as a drug target: Smoothened inhibitors in development. Onco Targets Ther 2012;5:47-58
   PubMed Web of Science ®Google Scholar
• Ibuki N, Ghaffari M, Pandey M, et al. TAK-441, a novel investigational smoothened antagonist, delays castration-resistant progression in prostate cancer by disrupting paracrine hedgehog signaling. Int J Cancer 2013;133(8):1955-66
   PubMed Web of Science ®Google Scholar
• Siu LLKP, Alberts SR, Kirchoff-Ross R, et al. A first-in-human, phase I study of an oral hedgehog (HH) pathway antagonist, BMS-833923 (XL139), in subjects with advanced or metastatic solid tumors. In: ASCO Annual Meeting. Journal od Clinical Oncology; 2010
   Google Scholar
• Steven B, Gendreau DH, Ching Ping Ho, et al. Preclinical characterization of BMS 833923 (XL139), a hedgehog (HH) pathway inhibitor in early clinical development. In: AACR-NCI-EORTC International Conference. Molecular Cancer Therapeutics, Boston, MA; 2009
   Google Scholar
• Kimura H, Ng JM, Curran T. Transient inhibition of the Hedgehog pathway in young mice causes permanent defects in bone structure. Cancer Cell 2008;13(3):249-60
   PubMed Web of Science ®Google Scholar
• Kool M, Jones DT, Jager N, et al. Genome sequencing of SHH medulloblastoma predicts genotype-related response to smoothened inhibition. Cancer Cell 2014;25(3):393-405
   PubMed Web of Science ®Google Scholar
• Meani RE, Lim SW, Chang AL, Kelly JW. Emergence of chemoresistance in a metastatic basal cell carcinoma patient after complete response to hedgehog pathway inhibitor vismodegib (GDC-0449). Australas J Dermatol 2014;55(3):218-21
   PubMedGoogle Scholar
• Yauch RL, Dijkgraaf GJ, Alicke B, et al. Smoothened mutation confers resistance to a Hedgehog pathway inhibitor in medulloblastoma. Science 2009;326(5952):572-4
   PubMed Web of Science ®Google Scholar
• Kim J, Lee JJ, Kim J, et al. Arsenic antagonizes the Hedgehog pathway by preventing ciliary accumulation and reducing stability of the Gli2 transcriptional effector. Proc Nat Acad Sci USA 2010;107(30):13432-7
   PubMed Web of Science ®Google Scholar
• Kim J, Aftab BT, Tang JY, et al. Itraconazole and arsenic trioxide inhibit Hedgehog pathway activation and tumor growth associated with acquired resistance to smoothened antagonists. Cancer Cell 2013;23(1):23-34
   PubMed Web of Science ®Google Scholar
• Larsen AR, Bai RY, Chung JH, et al. Repurposing the antihelmintic mebendazole as a hedgehog inhibitor. Mol Cancer Ther 2015;14(1):3-13
   PubMedGoogle Scholar
• Bai RY, Staedtke V, Rudin CM, et al. Effective treatment of diverse medulloblastoma models with mebendazole and its impact on tumor angiogenesis. Neuro Oncol 2015;17(4):545-54
   PubMed Web of Science ®Google Scholar
• Bladt F, Riethmacher D, Isenmann S, et al. Essential role for the c-met receptor in the migration of myogenic precursor cells into the limb bud. Nature 1995;376(6543):768-71
   PubMed Web of Science ®Google Scholar
• Ieraci A, Forni PE, Ponzetto C. Viable hypomorphic signaling mutant of the Met receptor reveals a role for hepatocyte growth factor in postnatal cerebellar development. Proc Natl Acad Sci U S A 2002;99(23):15200-5
   PubMedGoogle Scholar
• Gherardi E, Birchmeier W, Birchmeier C, Vande Woude G. Targeting MET in cancer: rationale and progress. Nat Rev Cancer 2012;12(2):89-103
   PubMed Web of Science ®Google Scholar
• Li Y, Lal B, Kwon S, et al. The scatter factor/hepatocyte growth factor: c-met pathway in human embryonal central nervous system tumor malignancy. Cancer Res 2005;65(20):9355-62
   PubMedGoogle Scholar
• Kongkham PN, Onvani S, Smith CA, Rutka JT. Inhibition of the MET Receptor Tyrosine Kinase as a Novel Therapeutic Strategy in Medulloblastoma. Trans Oncol 2010;3(6):336-43
   PubMedGoogle Scholar
• Faria CC, Golbourn BJ, Dubuc AM, et al. Foretinib is effective therapy for metastatic sonic hedgehog medulloblastoma. Cancer Res 2015;75(1):134-46
   PubMed Web of Science ®Google Scholar
• Vismodegib in treating younger patients with recurrent or refractory medulloblastoma. Available from: https://clinicaltrials.gov/ct2/show/NCT01239316
   Google Scholar
• Study of Vismodegib in combination with temozolomide versus temozolomide alone in patients with medulloblastomas with an activation of the sonic hedgehog pathway. Available from: https://clinicaltrials.gov/ct2/show/NCT01601184
   Google Scholar
• GDC-0449 in Treating young patients with medulloblastoma that is recurrent or did not respond to previous treatment. Available from: https://clinicaltrials.gov/ct2/show/NCT00822458
   Google Scholar
• Vismodegib in Treating patients with recurrent or refractory medulloblastoma. Available from: https://clinicaltrials.gov/ct2/show/NCT00939484
   Google Scholar
• A Phase II study of oral LDE225 in patients with hedge-hog (Hh)-pathway activated relapsed medulloblastoma (MB). Available from: https://clinicaltrials.gov/ct2/show/NCT01708174
   Google Scholar
• A phase i dose finding and safety study of oral lde225 in children and a phase ii portion to assess preliminary efficacy in recurrent or refractory MB. Available from: https://clinicaltrials.gov/ct2/show/NCT01125800
   Google Scholar
• Dose Finding and safety of oral LDE225 in patients with advanced solid tumors. Available from: https://clinicaltrials.gov/ct2/show/NCT00880308
   Google Scholar
• An East Asian Study of LDE225. Available from: https://clinicaltrials.gov/ct2/show/NCT01208831
   Google Scholar
• A Phase 1 Study of IPI-926 in Patients With Advanced and/or Metastatic Solid Tumor Malignancies. Available from: https://clinicaltrials.gov/ct2/show/NCT00761696
   Google Scholar
• A Study of TAK-441 in Adult Patients With Advanced Nonhematologic Malignancies. Available from: https://clinicaltrials.gov/ct2/show/NCT01204073
   Google Scholar