Advances on Epigenetic Drugs for Pediatric Brain Tumors

Liu, K.W.; Pajtler, K.W.; Worst, B.C.; Pfister, S.M.; Wechsler-Reya, R.J. Molecular mechanisms and therapeutic targets in pediatric brain tumors. Sci. Signal., 2017, 10(470), eaaf7593.
[http://dx.doi.org/10.1126/scisignal.aaf7593] [PMID: 28292958]

Ostrom, Q.T.; Gittleman, H.; Farah, P.; Ondracek, A.; Chen, Y.; Wolinsky, Y.; Stroup, N.E.; Kruchko, C.; Barnholtz-Sloan, J.S. CBTRUS statistical report: Primary brain and central nervous system tumors diagnosed in the United States in 2006-2010. Neurooncol., 2013, 15(Suppl. 2), ii1-ii56.
[http://dx.doi.org/10.1093/neuonc/not151] [PMID: 24137015]

Macdonald, T.J.; Aguilera, D.; Castellino, R.C. The rationale] for targeted therapies in medulloblastoma. Neuro. Oncol., 2014, 16(11), 9-20.
[http://dx.doi.org/10.1093/neuonc/not147]

Louis, D.N.; Perry, A.; Wesseling, P.; Brat, D.J.; Cree, I.A.; Figarella-Branger, D.; Hawkins, C.; Ng, H.K.; Pfister, S.M.; Reifenberger, G.; Soffietti, R.; von Deimling, A.; Ellison, D.W. The 2021 WHO classification of tumors of the central nervous system: A summary. Neuro-oncol., 2021, 23(8), 1231-1251.
[http://dx.doi.org/10.1093/neuonc/noab106] [PMID: 34185076]

Abedalthagafi, M.; Mobark, N.; Al-Rashed, M.; AlHarbi, M. Epigenomics and immunotherapeutic advances in pediatric brain tumors. NPJ Precis. Oncol., 2021, 5(1), 34.
[http://dx.doi.org/10.1038/s41698-021-00173-4] [PMID: 33931704]

Lu, Y.; Chan, Y. T.; Tan, H. Y.; Li, S.; Wang, N.; Feng, Y. Epigenetic regulation in human cancer: The potential role of epi-drug in cancer therapy. Mol. Cancer, 2020, 19(1), 1-16.
[http://dx.doi.org/10.1186/s12943-020-01197-3]

Cosgrove, M.S.; Boeke, J.D.; Wolberger, C. Regulated nucleosome mobility and the histone code. Nat. Struct. Mol. Biol., 2004, 11(11), 1037-1043.
[http://dx.doi.org/10.1038/nsmb851] [PMID: 15523479]

Khorasanizadeh, S. The nucleosome. Cell, 2004, 116(2), 259-272.
[http://dx.doi.org/10.1016/S0092-8674(04)00044-3] [PMID: 14744436]

Greer, E.L.; Shi, Y. Histone methylation: A dynamic mark in health, disease and inheritance. Nat. Rev. Genet., 2012, 13(5), 343-357.
[http://dx.doi.org/10.1038/nrg3173] [PMID: 22473383]

Priesterbach-Ackley, L.P.; Boldt, H.B.; Petersen, J.K.; Bervoets, N.; Scheie, D.; Ulhøi, B.P.; Gardberg, M.; Brännström, T.; Torp, S.H.; Aronica, E.; Küsters, B.; den Dunnen, W.F.A.; Vos, F.Y.F.L.; Wesseling, P.; Leng, W.W.J.; Kristensen, B.W. Brain tumour diagnostics using a DNA methylation‐based classifier as a diagnostic support tool. Neuropathol. Appl. Neurobiol., 2020, 46(5), 478-492.
[http://dx.doi.org/10.1111/nan.12610] [PMID: 32072658]

Nebbioso, A.; Tambaro, F.P.; Dell’Aversana, C.; Altucci, L. Cancer epigenetics: Moving forward. PLoS Genet., 2018, 14(6), e1007362.
[http://dx.doi.org/10.1371/journal.pgen.1007362] [PMID: 29879107]

Souweidane, M.M.; Kramer, K.; Pandit-Taskar, N.; Zhou, Z.; Haque, S.; Zanzonico, P.; Carrasquillo, J.A.; Lyashchenko, S.K.; Thakur, S.B.; Donzelli, M.; Turner, R.S.; Lewis, J.S.; Cheung, N.K.V.; Larson, S.M.; Dunkel, I.J. Convection-enhanced delivery for diffuse intrinsic pontine glioma: A single-centre, dose-escalation, phase 1 trial. Lancet Oncol., 2018, 19(8), 1040-1050.
[http://dx.doi.org/10.1016/S1470-2045(18)30322-X] [PMID: 29914796]

Schwalbe, E.C.; Lindsey, J.C.; Nakjang, S.; Crosier, S.; Smith, A.J.; Hicks, D.; Rafiee, G.; Hill, R.M.; Iliasova, A.; Stone, T.; Pizer, B.; Michalski, A.; Joshi, A.; Wharton, S.B.; Jacques, T.S.; Bailey, S.; Williamson, D.; Clifford, S.C. Novel molecular subgroups for clinical classification and outcome prediction in childhood medulloblastoma: A cohort study. Lancet Oncol., 2017, 18(7), 958-971.
[http://dx.doi.org/10.1016/S1470-2045(17)30243-7] [PMID: 28545823]

Hovestadt, V.; Ayrault, O.; Swartling, F.J.; Robinson, G.W.; Pfister, S.M.; Northcott, P.A. Medulloblastomics revisited: Biological and clinical insights from thousands of patients. Nat. Rev. Cancer, 2020, 20(1), 42-56.
[http://dx.doi.org/10.1038/s41568-019-0223-8] [PMID: 31819232]

Kumar, R.; Liu, A.P.Y.; Northcott, P.A. Medulloblastoma genomics in the modern molecular era. Brain Pathol., 2020, 30(3), 679-690.
[http://dx.doi.org/10.1111/bpa.12804] [PMID: 31799776]

Cavalli, F.M.G.; Remke, M.; Rampasek, L.; Peacock, J.; Shih, D.J.H.; Luu, B.; Garzia, L.; Torchia, J.; Nor, C.; Morrissy, A.S.; Agnihotri, S.; Thompson, Y.Y.; Kuzan-Fischer, C.M.; Farooq, H.; Isaev, K.; Daniels, C.; Cho, B.K.; Kim, S.K.; Wang, K.C.; Lee, J.Y.; Grajkowska, W.A.; Perek-Polnik, M.; Vasiljevic, A.; Faure-Conter, C.; Jouvet, A.; Giannini, C.; Nageswara Rao, A.A.; Li, K.K.W.; Ng, H.K.; Eberhart, C.G.; Pollack, I.F.; Hamilton, R.L.; Gillespie, G.Y.; Olson, J.M.; Leary, S.; Weiss, W.A.; Lach, B.; Chambless, L.B.; Thompson, R.C.; Cooper, M.K.; Vibhakar, R.; Hauser, P.; van Veelen, M.L.C.; Kros, J.M.; French, P.J.; Ra, Y.S.; Kumabe, T.; López-Aguilar, E.; Zitterbart, K.; Sterba, J.; Finocchiaro, G.; Massimino, M.; Van Meir, E.G.; Osuka, S.; Shofuda, T.; Klekner, A.; Zollo, M.; Leonard, J.R.; Rubin, J.B.; Jabado, N.; Albrecht, S.; Mora, J.; Van Meter, T.E.; Jung, S.; Moore, A.S.; Hallahan, A.R.; Chan, J.A.; Tirapelli, D.P.C.; Carlotti, C.G.; Fouladi, M.; Pimentel, J.; Faria, C.C.; Saad, A.G.; Massimi, L.; Liau, L.M.; Wheeler, H.; Nakamura, H.; Elbabaa, S.K.; Perezpeña-Diazconti, M.; Chico Ponce de León, F.; Robinson, S.; Zapotocky, M.; Lassaletta, A.; Huang, A.; Hawkins, C.E.; Tabori, U.; Bouffet, E.; Bartels, U.; Dirks, P.B.; Rutka, J.T.; Bader, G.D.; Reimand, J.; Goldenberg, A.; Ramaswamy, V.; Taylor, M.D. Intertumoral heterogeneity within medulloblastoma subgroups. Cancer Cell, 2017, 31(6), 737-754.e6.
[http://dx.doi.org/10.1016/j.ccell.2017.05.005] [PMID: 28609654]

Packer, R.J.; Gajjar, A.; Vezina, G.; Rorke-Adams, L.; Burger, P.C.; Robertson, P.L.; Bayer, L.; LaFond, D.; Donahue, B.R.; Marymont, M.H.; Muraszko, K.; Langston, J.; Sposto, R. Phase III study of craniospinal radiation therapy followed by adjuvant chemotherapy for newly diagnosed average-risk medulloblastoma. J. Clin. Oncol., 2006, 24(25), 4202-4208.
[http://dx.doi.org/10.1200/JCO.2006.06.4980] [PMID: 16943538]

Needle, M.N.; Goldwein, J.W.; Grass, J.; Cnaan, A.; Bergman, I.; Molloy, P.; Sutton, L.; Zhao, H.; Garvin, J.H., Jr; Phillips, P.C. Adjuvant chemotherapy for the treatment of intracranial ependymoma of childhood. Cancer, 1997, 80(2), 341-347.
[http://dx.doi.org/10.1002/(SICI)1097-0142(19970715)80:2<341::AID-CNCR23>3.0.CO;2-T] [PMID: 9217048]

Gajjar, A.; Finlay, J.L. The management of children and adolescents with medulloblastoma in low and middle income countries. Pediatr. Blood Cancer, 2015, 62(4), 549-550.
[http://dx.doi.org/10.1002/pbc.25371] [PMID: 25545387]

Roussel, M.F.; Stripay, J.L. Epigenetic drivers in pediatric medulloblastoma. Cerebellum, 2018, 17(1), 28-36.
[http://dx.doi.org/10.1007/s12311-017-0899-9] [PMID: 29178021]

Azzolin, L.; Panciera, T.; Soligo, S.; Enzo, E.; Bicciato, S.; Dupont, S.; Bresolin, S.; Frasson, C.; Basso, G.; Guzzardo, V.; Fassina, A.; Cordenonsi, M.; Piccolo, S. YAP/TAZ incorporation in the β-catenin destruction complex orchestrates the Wnt response. Cell, 2014, 158(1), 157-170.
[http://dx.doi.org/10.1016/j.cell.2014.06.013] [PMID: 24976009]

Shi, X.; Zhang, Z.; Zhan, X.; Cao, M.; Satoh, T.; Akira, S.; Shpargel, K.; Magnuson, T.; Li, Q.; Wang, R.; Wang, C.; Ge, K.; Wu, J. An epigenetic switch induced by Shh signalling regulates gene activation during development and medulloblastoma growth. Nat. Commun., 2014, 5(1), 5425.
[http://dx.doi.org/10.1038/ncomms6425] [PMID: 25370275]

Ivanov, D.P.; Coyle, B.; Walker, D.A.; Grabowska, A.M. In vitro models of medulloblastoma: Choosing the right tool for the job. J. Biotechnol., 2016, 236, 10-25.
[http://dx.doi.org/10.1016/j.jbiotec.2016.07.028] [PMID: 27498314]

Guerreiro Stucklin, A.S.; Ramaswamy, V.; Daniels, C.; Taylor, M.D. Review of molecular classification and treatment implications of pediatric brain tumors. Curr. Opin. Pediatr., 2018, 30(1), 3-9.
[http://dx.doi.org/10.1097/MOP.0000000000000562] [PMID: 29315108]

Gajjar, A.; Chintagumpala, M.; Ashley, D.; Kellie, S.; Kun, L.E.; Merchant, T.E.; Woo, S.; Wheeler, G.; Ahern, V.; Krasin, M.J.; Fouladi, M.; Broniscer, A.; Krance, R.; Hale, G.A.; Stewart, C.F.; Dauser, R.; Sanford, R.A.; Fuller, C.; Lau, C.; Boyett, J.M.; Wallace, D.; Gilbertson, R.J. Risk-adapted craniospinal radiotherapy followed by high-dose chemotherapy and stem-cell rescue in children with newly diagnosed medulloblastoma (St Jude Medulloblastoma-96): Long-term results from a prospective, multicentre trial. Lancet Oncol., 2006, 7(10), 813-820.
[http://dx.doi.org/10.1016/S1470-2045(06)70867-1] [PMID: 17012043]

Clifford, S.C.; Lusher, M.E.; Lindsey, J.C.; Langdon, J.A.; Gilbertson, R.J.; Straughton, D.; Ellison, D.W. Wnt/Wingless pathway activation and chromosome 6 loss characterize a distinct molecular sub-group of medulloblastomas associated with a favorable prognosis. Cell Cycle, 2006, 5(22), 2666-2670.
[http://dx.doi.org/10.4161/cc.5.22.3446] [PMID: 17172831]

Gururangan, S.; Schroeder, K.; Pediatric, G.; Services, C.; Tisch, P.R.; Tumor, B. Molecular variants and mutations in medulloblastoma. Pharm. Genomics Pers. Med., 2014, 7, 43-51.
[http://dx.doi.org/10.2147/PGPM.S38698] [PMID: 24523595]

Robinson, G.; Parker, M.; Kranenburg, T.A.; Lu, C.; Chen, X.; Ding, L.; Phoenix, T.N.; Hedlund, E.; Wei, L.; Zhu, X.; Chalhoub, N.; Baker, S.J.; Huether, R.; Kriwacki, R.; Curley, N.; Thiruvenkatam, R.; Wang, J.; Wu, G.; Rusch, M.; Hong, X.; Becksfort, J.; Gupta, P.; Ma, J.; Easton, J.; Vadodaria, B.; Onar-Thomas, A.; Lin, T.; Li, S.; Pounds, S.; Paugh, S.; Zhao, D.; Kawauchi, D.; Roussel, M.F.; Finkelstein, D.; Ellison, D.W.; Lau, C.C.; Bouffet, E.; Hassall, T.; Gururangan, S.; Cohn, R.; Fulton, R.S.; Fulton, L.L.; Dooling, D.J.; Ochoa, K.; Gajjar, A.; Mardis, E.R.; Wilson, R.K.; Downing, J.R.; Zhang, J.; Gilbertson, R.J. Novel mutations target distinct subgroups of medulloblastoma. Nature, 2012, 488(7409), 43-48.
[http://dx.doi.org/10.1038/nature11213] [PMID: 22722829]

Ellison, D.W.; Onilude, O.E.; Lindsey, J.C.; Lusher, M.E.; Weston, C.L.; Taylor, R.E.; Pearson, A.D.; Clifford, S.C. β-Catenin status predicts a favorable outcome in childhood medulloblastoma: The United Kingdom Children’s Cancer Study Group Brain Tumour Committee. J. Clin. Oncol., 2005, 23(31), 7951-7957.
[http://dx.doi.org/10.1200/JCO.2005.01.5479] [PMID: 16258095]

Dubuc, A.M.; Remke, M.; Korshunov, A.; Northcott, P.A.; Zhan, S.H.; Mendez-Lago, M.; Kool, M.; Jones, D.T.W.; Unterberger, A.; Morrissy, A.S.; Shih, D.; Peacock, J.; Ramaswamy, V.; Rolider, A.; Wang, X.; Witt, H.; Hielscher, T.; Hawkins, C.; Vibhakar, R.; Croul, S.; Rutka, J.T.; Weiss, W.A.; Jones, S.J.M.; Eberhart, C.G.; Marra, M.A.; Pfister, S.M.; Taylor, M.D. Aberrant patterns of H3K4 and H3K27 histone lysine methylation occur across subgroups in medulloblastoma. Acta Neuropathol., 2013, 125(3), 373-384.
[http://dx.doi.org/10.1007/s00401-012-1070-9] [PMID: 23184418]

Cohen, K.J.; Pollack, I.F.; Zhou, T.; Buxton, A.; Holmes, E.J.; Burger, P.C.; Brat, D.J.; Rosenblum, M.K.; Hamilton, R.L.; Lavey, R.S.; Heideman, R.L. Temozolomide in the treatment of high-grade gliomas in children: A report from the Children’s Oncology Group. Neuro-oncol., 2011, 13(3), 317-323.
[http://dx.doi.org/10.1093/neuonc/noq191] [PMID: 21339192]

Harutyunyan, A. S.; Krug, B.; Chen, H.; Papillon-Cavanagh, S.; Zeinieh, M.; De Jay, N.; Deshmukh, S.; Chen, C. C. L.; Belle, J.; Mikael, L. G.; Marchione, D. M.; Li, R.; Nikbakht, H.; Hu, B.; Cagnone, G.; Cheung, W. A.; Mohammadnia, A.; Bechet, D.; Faury, D.; McConechy, M. K.; Pathania, M.; Jain, S. U.; Ellezam, B.; Weil, A. G.; Montpetit, A.; Salomoni, P.; Pastinen, T.; Lu, C.; Lewis, P. W.; Garcia, B. A.; Kleinman, C. L.; Jabado, N.; Majewski, J. H3K27M induces defective chromatin spread of PRC2-mediated repressive H3K27me2/Me3 and is essential for glioma tumorigenesis. Nat. Commun., 2019, 10(1), 1-13.
[http://dx.doi.org/10.1038/s41467-019-09140-x]

Khuong-Quang, D. A.; Buczkowicz, P.; Rakopoulos, P.; Liu, X. Y.; Fontebasso, A. M.; Bouffet, E.; Bartels, U.; Albrecht, S.; Schwartzentruber, J.; Letourneau, L.; Bourgey, M.; Bourque, G.; Montpetit, A.; Bourret, G.; Lepage, P.; Fleming, A.; Lichter, P.; Kool, M.; Von Deimling, A.; Sturm, D.; Korshunov, A.; Faury, D.; Jones, D. T.; Majewski, J.; Pfister, S. M.; Jabado, N.; Hawkins, C. K27M mutation in histone H3.3 defines clinically and biologically distinct subgroups of pediatric diffuse intrinsic pontine gliomas. 2012, 124(3), 439-447.

Silveira, A. B.; Kasper, L. H.; Fan, Y.; Jin, H.; Wu, G.; Shaw, T. I.; Zhu, X.; Larson, J. D.; Easton, J.; Shao, Y.; Yergeau, D. A.; Rosencrance, C.; Boggs, K.; Rusch, M. C.; Ding, L.; Zhang, J.; Finkelstein, D.; Noyes, R. M.; Russell, B. L.; Xu, B.; Broniscer, A.; Wetmore, C.; Pounds, S. B.; Ellison, D. W.; Zhang, J.; Baker, S. J. H3.3 K27M depletion increases differentiation and extends latency of diffuse intrinsic pontine glioma growth in vivo. Acta Neuropathol., 2019, 137(4), 637-655.
[http://dx.doi.org/10.1007/s00401-019-01975-4]

Chammas, P.; Mocavini, I.; Di Croce, L. Engaging chromatin: PRC2 structure meets function. Br. J. Cancer, 2019, 122(3), 315-328.
[http://dx.doi.org/10.1038/s41416-019-0615-2]

Lewis, P.W.; Müller, M.M.; Koletsky, M.S.; Cordero, F.; Lin, S.; Banaszynski, L.A.; Garcia, B.A.; Muir, T.W.; Becher, O.J.; Allis, C.D. Inhibition of PRC2 activity by a gain-of-function H3 mutation found in pediatric glioblastoma. Science, 2013, 340(6134), 857-861.
[http://dx.doi.org/10.1126/science.1232245] [PMID: 23539183]

Krug, B.; De Jay, N.; Harutyunyan, A. S.; Deshmukh, S.; Marchione, D. M.; Guilhamon, P.; Bertrand, K. C.; Mikael, L. G.; McConechy, M. K.; Chen, C. C. L.; Khazaei, S.; Koncar, R. F.; Agnihotri, S.; Faury, D.; Ellezam, B.; Weil, A. G.; Ursini-Siegel, J.; De Carvalho, D. D.; Dirks, P. B.; Lewis, P. W.; Salomoni, P.; Lupien, M.; Arrowsmith, C.; Lasko, P. F.; Garcia, B. A.; Kleinman, C. L.; Jabado, N.; Mack, S. C. Pervasive H3K27 acetylation leads to ERV expression and a therapeutic vulnerability in H3K27M gliomas. Cancer Cell, 2019, 35(5), 782-797.e8.
[http://dx.doi.org/10.1016/j.ccell.2019.04.004] [PMID: 31085178]

Nagaraja, S.; Quezada, M.A.; Gillespie, S.M.; Arzt, M.; Lennon, J.J.; Woo, P.J.; Hovestadt, V.; Kambhampati, M.; Filbin, M.G.; Suva, M.L.; Nazarian, J.; Monje, M. Histone variant and cell context determine H3K27M reprogramming of the enhancer landscape and oncogenic state. Mol. Cell, 2019, 76(6), 965-980.e12.
[http://dx.doi.org/10.1016/j.molcel.2019.08.030] [PMID: 31588023]

Jonkers, I.; Lis, J.T. Getting up to speed with transcription elongation by RNA polymerase II. Nat. Rev. Mol. Cell Biol., 2015, 16(3), 167-177.
[http://dx.doi.org/10.1038/nrm3953] [PMID: 25693130]

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Fontebasso, A. M.; Papillon-Cavanagh, S.; Schwartzentruber, J.; Nikbakht, H.; Gerges, N.; Fiset, P. O.; Bechet, D.; Faury, D.; De Jay, N.; Ramkissoon, L. A.; Corcoran, A.; Jones, D. T. W.; Sturm, D.; Johann, P.; Tomita, T.; Goldman, S.; Nagib, M.; Bendel, A.; Goumnerova, L.; Bowers, D. C.; Leonard, J. R.; Rubin, J. B.; Alden, T.; Browd, S.; Geyer, J. R.; Leary, S.; Jallo, G.; Cohen, K.; Gupta, N.; Prados, M. D.; Carret, A. S.; Ellezam, B.; Crevier, L.; Klekner, A.; Bognar, L.; Hauser, P.; Garami, M.; Myseros, J.; Dong, Z.; Siegel, P. M.; Malkin, H.; Ligon, A. H.; Albrecht, S.; Pfister, S. M.; Ligon, K. L.; Majewski, J.; Jabado, N.; Kieran, M. W. Recurrent somatic mutations in ACVR1 in pediatric midline high-grade astrocytoma. Nat. Genet., 2014, 46(5), 462-466.
[http://dx.doi.org/10.1038/ng.2950]

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[http://dx.doi.org/10.1158/1078-0432.CCR-18-3890]

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