Functionally defined therapeutic targets in diffuse intrinsic pontine glioma

被引：412

作者：

Grasso, Catherine S. ^{[1
,2
]}

Tang, Yujie ^{[2
,3
,4
,5
]}

Truffaux, Nathalene ^{[6
]}

Berlow, Noah E. ^{[7
]}

Liu, Lining ^{[2
,3
,4
,5
]}

Debily, Marie-Anne ^{[8
]}

Quist, Michael J. ^{[1
]}

Davis, Lara E. ^{[8
]}

Huang, Elaine C. ^{[9
]}

Woo, Pamelyn J. ^{[2
,3
,4
,5
]}

Ponnuswami, Anitha ^{[2
,3
,4
,5
]}

Chen, Spenser ^{[2
,3
,4
,5
]}

Johung, Tessa B. ^{[2
,3
,4
,5
]}

Sun, Wenchao ^{[2
]}

Kogiso, Mari ^{[10
]}

Dui, Yuchen ^{[10
]}

Qi, Lin ^{[10
]}

Huang, Yulun

Huett-Cabezas, Marianne ^{[11
,12
]}

Warrenm, Katherine E. ^{[12
]}

Le Dret, Ludivine ^{[6
]}

Meltzerm, Paul S. ^{[13
]}

Mao, Hua ^{[10
]}

Quezado, Martha ^{[12
]}

van Vuurden, Dannis G. ^{[14
,15
]}

Abraham, Jinu ^{[9
]}

Fouladi, Maryam ^{[16
]}

Svalina, Matthew N. ^{[17
]}

Wang, Nicholas ^{[1
]}

Hawkins, Cynthia ^{[18
,19
]}

Nazarian, Javad ^{[20
]}

Alonso, Marta M. ^{[21
]}

Raabe, Eric H. ^{[11
,12
]}

Hulleman, Esther ^{[14
,15
]}

Spellman, Paul T. ^{[1
]}

Li, Xiao-Nan ^{[10
]}

Keller, Charles ^{[9
,17
]}

Pal, Ranadip ^{[7
]}

Grill, Jacques ^{[6
,22
,23
]}

Monje, Michelle ^{[2
,3
,4
,5
]}

机构：

[1] Oregon Hlth & Sci Univ, Ctr Spatial Syst Biomed, Dept Mol & Med Genet, Portland, OR 97201 USA

[2] Stanford Univ, Dept Neurol, Stanford, CA 94305 USA

[3] Stanford Univ, Dept Neurosurg, Stanford, CA 94305 USA

[4] Stanford Univ, Dept Pediat, Stanford, CA 94305 USA

[5] Stanford Univ, Dept Pathol, Stanford, CA 94305 USA

[6] Univ Paris Sud, CNRS, Gustave Roussy, UMR 8203, Villejuif, France

[7] Texas Tech Univ, Elect & Comp Engn, Lubbock, TX 79409 USA

[8] Univ Evry Val dEssone, Dept Biol, Evry, France

[9] Oregon Hlth & Sci Univ, Dept Pediat, Pape Family Pediat Res Inst, Pediat Canc Biol Program, Portland, OR 97201 USA

[10] Baylor Coll Med, Texas Childrens Canc Ctr, Mol Neurooncol Lab, Houston, TX 77030 USA

[11] Johns Hopkins Univ, Dept Oncol, Baltimore, MD USA

[12] Johns Hopkins Univ, Dept Pathol, Baltimore, MD USA

[13] NCI, Bethesda, MD 20892 USA

[14] Vrije Univ Amsterdam, Med Ctr, Dept Pediat Oncol & Hematol, Amsterdam, Netherlands

[15] Vrije Univ Amsterdam, Med Ctr, Neuroncol Res Grp Canc Ctr Amsterdam, Amsterdam, Netherlands

[16] Cincinnati Childrens Hosp Med Ctr, Canc & Blood Dis Inst, Cincinnati, OH 45229 USA

[17] Childrens Canc Therapy Dev Inst, Ft Collins, CO USA

[18] Univ Toronto, Dept Pediat Lab Med, Toronto, ON, Canada

[19] Univ Toronto, Hosp Sick Children, Labatt Brain Tumor Res Ctr, Toronto, ON M5G 1X8, Canada

[20] Childrens Natl Hlth Syst, Ctr Res Inst, Washington, DC USA

[21] Univ Hosp Navarra, Dept Oncol, Pamplona, Spain

[22] Univ Paris Sud, Inst Gustave Roussy, Dept Cancerol Enfant & Adolescent, Villejuif, France

[23] Parke Davis Res Unit, Inst Gustave Roussy, Dept Cancerol Enfant & Adolescent, Villejuif, France

来源：

NATURE MEDICINE | 2015年 / 21卷 / 06期

基金：

美国国家科学基金会; 美国国家卫生研究院;

关键词：

HISTONE H3.3; MUTATIONS; INHIBITION; SUBGROUPS; CELL;

D O I：

10.1038/nm.3855

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

Diffuse intrinsic pontine glioma (DIPG) is a fatal childhood cancer. We performed a chemical screen in patient-derived DIPG cultures along with RNA-seq analyses and integrated computational modeling to identify potentially effective therapeutic strategies. The multi histone deacetylase inhibitor panobinostat demonstrated therapeutic efficacy both in vitro and in DIPG orthotopic xenograft models. Combination testing of panobinostat and the histone demethylase inhibitor GSK-J4 revealed that the two had synergistic effects. Together, these data suggest a promising therapeutic strategy for DIPG.

引用

页码：555 / 559

页数：5

共 19 条

[1] Reduced H3K27me3 and DNA Hypomethylation Are Major Drivers of Gene Expression in K27M Mutant Pediatric High-Grade Gliomas [J].

Bender, Sebastian ;

Tang, Yujie ;

Lindroth, Anders M. ;

Hovestadt, Volker ;

Jones, David T. W. ;

Kool, Marcel ;

Zapatka, Marc ;

Northcott, Paul A. ;

Sturm, Dominik ;

Wang, Wei ;

Radlwimmer, Bernhard ;

Hojfeldt, Jonas W. ;

Truffaux, Nathalene ;

Castel, David ;

Schubert, Simone ;

Ryzhova, Marina ;

Seker-Cin, Huriye ;

Gronych, Jan ;

Johann, Pascal David ;

Stark, Sebastian ;

Meyer, Jochen ;

Milde, Till ;

Schuhmann, Martin ;

Ebinger, Martin ;

Monoranu, Camelia-Maria ;

Ponnuswami, Anitha ;

Chen, Spenser ;

Jones, Chris ;

Witt, Olaf ;

Collins, V. Peter ;

von Deimling, Andreas ;

Jabado, Nada ;

Puget, Stephanie ;

Grill, Jacques ;

Helin, Kristian ;

Korshunov, Andrey ;

Lichter, Peter ;

Monje, Michelle ;

Plass, Christoph ;

Cho, Yoon-Jae ;

Pfister, Stefan M. .

CANCER CELL, 2013, 24 (05) :660-672

[2] A new approach for prediction of tumor sensitivity to targeted drugs based on functional data [J].

Berlow, Noah ;

Davis, Lara E. ;

Cantor, Emma L. ;

Seguin, Bernard ;

Keller, Charles ;

Pal, Ranadip .

BMC BIOINFORMATICS, 2013, 14

[3] Chemical phylogenetics of histone deacetylases [J].

Bradner, James E. ;

West, Nathan ;

Grachan, Melissa L. ;

Greenberg, Edward F. ;

Haggarty, Stephen J. ;

Warnow, Tandy ;

Mazitschek, Ralph .

NATURE CHEMICAL BIOLOGY, 2010, 6 (03) :238-243

[4] Strategy for "Detoxification" of a Cancer-Derived Histone Mutant Based on Mapping Its Interaction with the Methyltransferase PRC2 [J].

Brown, Zachary Z. ;

Mueller, Manuel M. ;

Jain, Siddhant U. ;

Allis, C. David ;

Lewis, Peter W. ;

Muir, Tom W. .

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, 2014, 136 (39) :13498-13501

[5] Human pontine glioma cells can induce murine tumors [J].

Caretti, Viola ;

Sewing, A. Charlotte P. ;

Lagerweij, Tonny ;

Schellen, Pepijn ;

Bugiani, Marianna ;

Jansen, Marc H. A. ;

van Vuurden, Dannis G. ;

Navis, Anna C. ;

Horsman, Ilona ;

Vandertop, W. Peter ;

Noske, David P. ;

Wesseling, Pieter ;

Kaspers, Gertjan J. L. ;

Nazarian, Javad ;

Vogel, Hannes ;

Hulleman, Esther ;

Monje, Michelle ;

Wurdinger, Thomas .

ACTA NEUROPATHOLOGICA, 2014, 127 (06) :897-909

[6] The histone H3.3K27M mutation in pediatric glioma reprograms H3K27 methylation and gene expression [J].

Chan, Kui-Ming ;

Fang, Dong ;

Gan, Haiyun ;

Hashizume, Rintaro ;

Yu, Chuanhe ;

Schroeder, Mark ;

Gupta, Nalin ;

Mueller, Sabine ;

James, C. David ;

Jenkins, Robert ;

Sarkaria, Jann ;

Zhang, Zhiguo .

GENES & DEVELOPMENT, 2013, 27 (09) :985-990

[7] Advances toward an understanding of brainstem gliomas [J].

Donaldson, SS ;

Laningham, F ;

Fisher, PG .

JOURNAL OF CLINICAL ONCOLOGY, 2006, 24 (08) :1266-1272

[8] K27M mutation in histone H3.3 defines clinically and biologically distinct subgroups of pediatric diffuse intrinsic pontine gliomas [J].

Dong-Anh Khuong-Quang ;

Buczkowicz, Pawel ;

Rakopoulos, Patricia ;

Liu, Xiao-Yang ;

Fontebasso, Adam M. ;

Bouffet, Eric ;

Bartels, Ute ;

Albrecht, Steffen ;

Schwartzentruber, Jeremy ;

Letourneau, Louis ;

Bourgey, Mathieu ;

Bourque, Guillaume ;

Montpetit, Alexandre ;

Bourret, Genevieve ;

Lepage, Pierre ;

Fleming, Adam ;

Lichter, Peter ;

Kool, Marcel ;

von Deimling, Andreas ;

Sturm, Dominik ;

Korshunov, Andrey ;

Faury, Damien ;

Jones, David T. ;

Majewski, Jacek ;

Pfister, Stefan M. ;

Jabado, Nada ;

Hawkins, Cynthia .

ACTA NEUROPATHOLOGICA, 2012, 124 (03) :439-447

[9] Pharmacologic inhibition of histone demethylation as a therapy for pediatric brainstem glioma [J].

Hashizume, Rintaro ;

Andor, Noemi ;

Ihara, Yuichiro ;

Lerner, Robin ;

Gan, Haiyun ;

Chen, Xiaoyue ;

Fang, Dong ;

Huang, Xi ;

Tom, Maxwell W. ;

Ngo, Vy ;

Solomon, David ;

Mueller, Sabine ;

Paris, Pamela L. ;

Zhang, Zhiguo ;

Petritsch, Claudia ;

Gupta, Nalin ;

Waldman, Todd A. ;

James, C. David .

NATURE MEDICINE, 2014, 20 (12) :1394-1396

[10] Characterization of a diffuse intrinsic pontine glioma cell line: implications for future investigations and treatment [J].

Hashizume, Rintaro ;

Smirnov, Ivan ;

Liu, Sharon ;

Phillips, Joanna J. ;

Hyer, Jeanette ;

McKnight, Tracy R. ;

Wendland, Michael ;

Prados, Michael ;

Banerjee, Anu ;

Nicolaides, Theodore ;

Mueller, Sabine ;

James, Charles D. ;

Gupta, Nalin .

JOURNAL OF NEURO-ONCOLOGY, 2012, 110 (03) :305-313

← 1 2 →