Current and Emerging Therapeutic Approaches for Extracranial Malignant Rhabdoid Tumors

References

• Beckwith JB, Palmer NF. Histopathology and prognosis of Wilms tumors: results from the First National Wilms’ Tumor Study. Cancer. 1978;41(5):1937–1948. doi:10.1002/1097-0142(197805)41:5<1937::aid-cncr2820410538>3.0.co;2-u
   PubMed Web of Science ®Google Scholar
• Judkins AR, Mauger J, Ht A, Rorke LB, Biegel JA. Immunohistochemical analysis of hSNF5/INI1 in pediatric CNS neoplasms. Am J Surg Pathol. 2004;28(5):644–650. doi:10.1097/00000478-200405000-00013
   PubMed Web of Science ®Google Scholar
• Cancer statistics reports for the Germany. Available from: http://www.kinderkrebsregister.de/dkkr/ergebnisse/jahresberichte/jahresbericht-2019.html. Accessed January 11, 2022.
   Google Scholar
• Brennan B, Stiller C, Bourdeaut F. Extracranial rhabdoid tumours: what we have learned so far and future directions. Lancet Oncol. 2013;14(8):e329–e336. doi:10.1016/S1470-2045(13)70088-3
   PubMed Web of Science ®Google Scholar
• Frühwald MC, Hasselblatt M, Nemes K, et al. Age and DNA methylation subgroup as potential independent risk factors for treatment stratification in children with atypical teratoid/rhabdoid tumors. Neuro Oncol. 2020;22(7):1006–1017. doi:10.1093/neuonc/noz244
   PubMed Web of Science ®Google Scholar
• Nemes K, Bens S, Kachanov D, et al. Clinical and genetic risk factors define two risk groups of extracranial malignant rhabdoid tumours (eMRT/RTK). Eur J Cancer. 2021;142:112–122. doi:10.1016/j.ejca.2020.10.004
   PubMed Web of Science ®Google Scholar
• Nakata K, Colombet M, Stiller CA, Pritchard-Jones K, Steliarova-Foucher E. Incidence of childhood renal tumours: an international population-based study. Int J Cancer. 2020;147(12):3313–3327. doi:10.1002/ijc.33147
   PubMed Web of Science ®Google Scholar
• Tomlinson GE, Breslow NE, Dome J, et al. Rhabdoid tumor of the kidney in the National Wilms’ Tumor Study: age at diagnosis as a prognostic factor. J Clin Oncol. 2005;23(30):7641–7645. doi:10.1200/JCO.2004.00.8110
   PubMed Web of Science ®Google Scholar
• van den Heuvel-eibrink MM, van Tinteren H, Rehorst H. Malignant rhabdoid tumours of the kidney (MRTKs), registered on recent SIOP protocols from 1993 to 2005: a report of the SIOP renal tumour study group. Pediatr Blood Cancer. 2011;56(5):733–737. doi:10.1002/pbc.22922
   PubMed Web of Science ®Google Scholar
• Brennan B, De Salvo GL, Orbach D, et al. Outcome of extracranial malignant rhabdoid tumours in children registered in the European Paediatric Soft Tissue Sarcoma Study Group Non-Rhabdomyosarcoma Soft Tissue Sarcoma 2005 Study-EpSSG NRSTS 2005. Eur J Cancer. 2016;60:69–82. doi:10.1016/j.ejca.2016.02.027
   PubMed Web of Science ®Google Scholar
• Hasselblatt M, Isken S, Linge A, et al. High-resolution genomic analysis suggests the absence of recurrent genomic alterations other than SMARCB1 aberrations in atypical teratoid/rhabdoid tumors. Genes Chromosomes Cancer. 2013;52(2):185–190. doi:10.1002/gcc.22018
   PubMed Web of Science ®Google Scholar
• Kieran MW, Roberts CW, Chi SN, et al. Absence of oncogenic canonical pathway mutations in aggressive pediatric rhabdoid tumors. Pediatr Blood Cancer. 2012;59(7):1155–1157. doi:10.1002/pbc.24315
   PubMed Web of Science ®Google Scholar
• Hasselblatt M, Nagel I, Oyen F, et al. SMARCA4-mutated atypical teratoid/rhabdoid tumors are associated with inherited germline alterations and poor prognosis. Acta Neuropathol. 2014;128(3):453–456. doi:10.1007/s00401-014-1323-x
   PubMed Web of Science ®Google Scholar
• Schneppenheim R, Fruhwald MC, Gesk S, et al. Germline nonsense mutation and somatic inactivation of SMARCA4/BRG1 in a family with rhabdoid tumor predisposition syndrome. Am J Hum Genet. 2010;86(2):279–284. doi:10.1016/j.ajhg.2010.01.013
   PubMed Web of Science ®Google Scholar
• Holsten T, Bens S, Oyen F, et al. Germline variants in SMARCB1 and other members of the BAF chromatin-remodeling complex across human disease entities: a meta-analysis. Eur J Hum Genet. 2018;26(8):1083–1093. doi:10.1038/s41431-018-0143-1
   PubMed Web of Science ®Google Scholar
• Wilson BG, Roberts CW. SWI/SNF nucleosome remodellers and cancer. Nat Rev Cancer. 2011;11(7):481–492. doi:10.1038/nrc3068
   PubMed Web of Science ®Google Scholar
• Tegeder I, Thiel K, Erkek S, et al. Functional relevance of genes predicted to be affected by epigenetic alterations in atypical teratoid/rhabdoid tumors. J Neurooncol. 2019;141(1):43–55. doi:10.1007/s11060-018-03018-6
   PubMed Web of Science ®Google Scholar
• Koelsche C, Stichel D, Griewank KG, et al. Genome-wide methylation profiling and copy number analysis in atypical fibroxanthomas and pleomorphic dermal sarcomas indicate a similar molecular phenotype. Clin Sarcoma Res. 2019;9:2. doi:10.1186/s13569-019-0113-6
   PubMed Web of Science ®Google Scholar
• Chun HE, Lim EL, Heravi-Moussavi A, et al. Genome-wide profiles of extra-cranial malignant rhabdoid tumors reveal heterogeneity and dysregulated developmental pathways. Cancer Cell. 2016;29(3):394–406. doi:10.1016/j.ccell.2016.02.009
   PubMed Web of Science ®Google Scholar
• Koelsche C, Schrimpf D, Stichel D, Sill M, Sahm F. Sarcoma classification by DNA methylation profiling. Nat Commun. 2021;12(1):498. doi:10.1038/s41467-020-20603-4
   PubMed Web of Science ®Google Scholar
• Johann PD, Erkek S, Zapatka M, et al. Atypical teratoid/rhabdoid tumors are comprised of three epigenetic subgroups with distinct enhancer landscapes. Cancer Cell. 2016;29(3):379–393. doi:10.1016/j.ccell.2016.02.001
   PubMed Web of Science ®Google Scholar
• Torchia J, Golbourn B, Feng S, et al. Integrated (epi)-genomic analyses identify subgroup-specific therapeutic targets in CNS rhabdoid tumors. Cancer Cell. 2016;30(6):891–908. doi:10.1016/j.ccell.2016.11.003
   PubMed Web of Science ®Google Scholar
• Chun HE, Johann PD, Milne K, et al. Identification and analyses of extra-cranial and cranial rhabdoid tumor molecular subgroups reveal tumors with cytotoxic T cell infiltration. Cell Rep. 2019;29(8):2338–2354.e7. doi:10.1016/j.celrep.2019.10.013
   PubMed Web of Science ®Google Scholar
• Birks DK, Donson AM, Patel PR, et al. High expression of BMP pathway genes distinguishes a subset of atypical teratoid/rhabdoid tumors associated with shorter survival. Neuro Oncol. 2011;13(12):1296–1307. doi:10.1093/neuonc/nor140
   PubMed Web of Science ®Google Scholar
• Birks DK, Donson AM, Patel PR, et al. Pediatric rhabdoid tumors of kidney and brain show many differences in gene expression but share dysregulation of cell cycle and epigenetic effector genes. Pediatr Blood Cancer. 2013;60(7):1095–1102. doi:10.1002/pbc.24481
   PubMed Web of Science ®Google Scholar
• Ho B, Johann PD, Grabovska Y, et al. Molecular subgrouping of atypical teratoid/rhabdoid tumors-a reinvestigation and current consensus. Neuro Oncol. 2020;22(5):613–624. doi:10.1093/neuonc/noz235
   PubMed Web of Science ®Google Scholar
• Brocks D, Schmidt CR, Daskalakis M, et al. DNMT and HDAC inhibitors induce cryptic transcription start sites encoded in long terminal repeats. Nat Genet. 2017;49(11):1661. doi:10.1038/ng.3889
   PubMed Web of Science ®Google Scholar
• Andrianteranagna M, Cyrta J, Masliah-Planchon J, et al. SMARCA4-deficient rhabdoid tumours show intermediate molecular features between SMARCB1-deficient rhabdoid tumours and small cell carcinomas of the ovary, hypercalcaemic type. J Pathol. 2021;255(1):1–15. doi:10.1002/path.5705
   PubMed Web of Science ®Google Scholar
• Alimova I, Pierce A, Danis E, et al. Inhibition of MYC attenuates tumor cell self-renewal and promotes senescence in SMARCB1-deficient Group 2 atypical teratoid rhabdoid tumors to suppress tumor growth in vivo. Int J Cancer. 2019;144(8):1983–1995. doi:10.1002/ijc.31873
   PubMed Web of Science ®Google Scholar
• Bourdeaut F, Lequin D, Brugières L, et al. Frequent hSNF5/INI1 germline mutations in patients with rhabdoid tumor. Clin Cancer Res. 2011;17(1):31–38. doi:10.1158/1078-0432.CCR-10-1795
   PubMed Web of Science ®Google Scholar
• Nemes K, Bens S, Bourdeaut F, et al. Rhabdoid tumor predisposition syndrome. Adam MP, Ardinger HH, Pagon RA, et al. editors. GeneReviews(^®). Seattle: University of Washington, Seattle Copyright © 1993–2021, University of Washington, Seattle. GeneReviews is a registered trademark of the University of Washington, Seattle; 1993.
   Google Scholar
• Sultan I, Qaddoumi I, Rodríguez-Galindo C, Nassan AA, Ghandour K, Al-Hussaini M. Age, stage, and radiotherapy, but not primary tumor site, affects the outcome of patients with malignant rhabdoid tumors. Pediatr Blood Cancer. 2010;54(1):35–40. doi:10.1002/pbc.22285
   PubMed Web of Science ®Google Scholar
• Venkatramani R, Shoureshi P, Malvar J, Zhou S, Mascarenhas L. High dose alkylator therapy for extracranial malignant rhabdoid tumors in children. Pediatr Blood Cancer. 2014;61(8):1357–1361. doi:10.1002/pbc.25093
   PubMed Web of Science ®Google Scholar
• Cheng H, Yang S, Cai S. Clinical and prognostic characteristics of 53 cases of extracranial malignant rhabdoid tumor in children. A single-institute experience from 2007 to 2017. Oncologist. 2019;24(7):e551–e558. doi:10.1634/theoncologist.2018-0416
   PubMed Web of Science ®Google Scholar
• Benesch M, Bartelheim K, Fleischhack G, et al. High-dose chemotherapy (HDCT) with auto-SCT in children with atypical teratoid/rhabdoid tumors (AT/RT): a report from the European Rhabdoid Registry (EU-RHAB). Bone Marrow Transplant. 2014;49(3):370–375. doi:10.1038/bmt.2013.208
   PubMed Web of Science ®Google Scholar
• Hoffman LM, Richardson EA, Ho B, et al. Advancing biology-based therapeutic approaches for atypical teratoid rhabdoid tumors. Neuro Oncol. 2020;22(7):944–954. doi:10.1093/neuonc/noaa046
   PubMed Web of Science ®Google Scholar
• Furtwängler R, Nourkami N, Alkassar M, et al. Update on relapses in unilateral nephroblastoma registered in 3 consecutive SIOP/GPOH studies - A report from the GPOH-nephroblastoma study group. Klin Padiatr. 2011;223(3):113–119. doi:10.1055/s-0031-1275293
   PubMed Web of Science ®Google Scholar
• Furtwängler R, Kager L, Melchior P, et al. High-dose treatment for malignant rhabdoid tumor of the kidney: no evidence for improved survival-The Gesellschaft für Pädiatrische Onkologie und Hämatologie (GPOH) experience. Pediatr Blood Cancer. 2018;65(1):e26746. doi:10.1002/pbc.26746
   Web of Science ®Google Scholar
• Melchior P, Dzierma Y, Rübe C, et al. Local stage dependent necessity of radiation therapy in rhabdoid tumors of the kidney (RTK). Int J Radiat Oncol Biol Phys. 2020;108(3):667–675. doi:10.1016/j.ijrobp.2020.04.046
   PubMed Web of Science ®Google Scholar
• Walz AL, Fernandez CV, Geller JI. Novel therapy for pediatric and adolescent kidney cancer. Cancer Metastasis Rev. 2019;38(4):643–655. doi:10.1007/s10555-019-09822-4
   PubMed Web of Science ®Google Scholar
• Unland R, Borchardt C, Clemens D, Kool M, Dirksen U, Frühwald MC. Analysis of the antiproliferative effects of 3-deazaneoplanocin A in combination with standard anticancer agents in rhabdoid tumor cell lines. Anticancer Drugs. 2015;26(3):301–311. doi:10.1097/CAD.0000000000000181
   PubMed Web of Science ®Google Scholar
• Li T, Wang J, Liu P, et al. Insulin-like growth factor 2 axis supports the serum-independent growth of malignant rhabdoid tumor and is activated by microenvironment stress. Oncotarget. 2017;8(29):47269–47283. doi:10.18632/oncotarget.17617
   PubMedGoogle Scholar
• Jeibmann A, Schulz J, Eikmeier K, et al. SMAD dependent signaling plays a detrimental role in a fly model of SMARCB1-deficiency and the biology of atypical teratoid/rhabdoid tumors. J Neurooncol. 2017;131(3):477–484. doi:10.1007/s11060-016-2326-3
   PubMed Web of Science ®Google Scholar
• Weingart MF, Roth JJ, Hutt-Cabezas M, et al. Disrupting LIN28 in atypical teratoid rhabdoid tumors reveals the importance of the mitogen activated protein kinase pathway as a therapeutic target. Oncotarget. 2015;6(5):3165–3177. doi:10.18632/oncotarget.3078
   PubMedGoogle Scholar
• Shahab S, Rubens J, Kaur H, Sweeney H, Eberhart CG, Raabe EH. MEK inhibition suppresses growth of atypical teratoid/rhabdoid tumors. J Neuropathol Exp Neurol. 2020;79(7):746–753. doi:10.1093/jnen/nlaa042
   PubMed Web of Science ®Google Scholar
• Rubens JA, Wang SZ, Price A, et al. The TORC1/2 inhibitor TAK228 sensitizes atypical teratoid rhabdoid tumors to cisplatin-induced cytotoxicity. Neuro Oncol. 2017;19(10):1361–1371. doi:10.1093/neuonc/nox067
   PubMed Web of Science ®Google Scholar
• Oberlick EM, Rees MG, Seashore-Ludlow B, et al. Small-molecule and CRISPR screening converge to reveal receptor tyrosine kinase dependencies in pediatric rhabdoid tumors. Cell Rep. 2019;28(9):2331–2344.e8. doi:10.1016/j.celrep.2019.07.021
   PubMed Web of Science ®Google Scholar
• Suri A, Bailey AW, Tavares MT. Evaluation of protein kinase inhibitors with PLK4 cross-over potential in a pre-clinical model of cancer. Int J Mol Sci. 2019;20(9):2112. doi:10.3390/ijms20092112
   PubMed Web of Science ®Google Scholar
• Singh A, Lun X, Jayanthan A, et al. Profiling pathway-specific novel therapeutics in preclinical assessment for central nervous system atypical teratoid rhabdoid tumors (CNS ATRT): favorable activity of targeting EGFR-ErbB2 signaling with lapatinib. Mol Oncol. 2013;7(3):497–512. doi:10.1016/j.molonc.2013.01.001
   PubMed Web of Science ®Google Scholar
• Alimova I, Pierce AM, Harris P, et al. Targeting Polo-like kinase 1 in SMARCB1 deleted atypical teratoid rhabdoid tumor. Oncotarget. 2017;8(57):97290–97303. doi:10.18632/oncotarget.21932
   PubMedGoogle Scholar
• Sredni ST, Bailey AW, Suri A, et al. Inhibition of polo-like kinase 4 (PLK4): a new therapeutic option for rhabdoid tumors and pediatric medulloblastoma. Oncotarget. 2017;8(67):111190–111212. doi:10.18632/oncotarget.22704
   PubMedGoogle Scholar
• Sredni ST, Suzuki M, Yang JP, et al. A functional screening of the kinome identifies the Polo-like kinase 4 as a potential therapeutic target for malignant rhabdoid tumors, and possibly, other embryonal tumors of the brain. Pediatr Blood Cancer. 2017;64(11):e26551. doi:10.1002/pbc.26551
   Web of Science ®Google Scholar
• Messerli SM, Hoffman MM, Gnimpieba EZ, Bhardwaj RD. Therapeutic targeting of PTK7 is cytotoxic in atypical teratoid rhabdoid tumors. Mol Cancer Res. 2017;15(8):973–983. doi:10.1158/1541-7786.MCR-16-0432
   PubMed Web of Science ®Google Scholar
• Obaid H, Kannappan S, Gupta M, et al. In vitro investigation demonstrates IGFR/VEGFR receptor cross talk and potential of combined inhibition in pediatric central nervous system atypical teratoid rhabdoid tumors. Curr Cancer Drug Targets. 2020;20(4):295–305. doi:10.2174/1568009619666191111153049
   PubMed Web of Science ®Google Scholar
• Chakravadhanula M, Hampton CN, Chodavadia P, et al. Wnt pathway in atypical teratoid rhabdoid tumors. Neuro Oncol. 2015;17(4):526–535. doi:10.1093/neuonc/nou229
   PubMed Web of Science ®Google Scholar
• Studebaker AW, Hutzen B, Pierson CR, Shaffer TA, Raffel C, Jackson EM. Oncolytic measles virus efficacy in murine xenograft models of atypical teratoid rhabdoid tumors. Neuro Oncol. 2015;17(12):1568–1577. doi:10.1093/neuonc/nov058
   PubMed Web of Science ®Google Scholar
• Lee YE, Choi SA, Kwack PA, et al. Repositioning disulfiram as a radiosensitizer against atypical teratoid/rhabdoid tumor. Neuro Oncol. 2017;19(8):1079–1087. doi:10.1093/neuonc/now300
   PubMed Web of Science ®Google Scholar
• Golan H, Shukrun R, Caspi R, et al. In vivo expansion of cancer stemness affords novel cancer stem cell targets: malignant rhabdoid tumor as an example. Stem Cell Rep. 2018;11(3):795–810. doi:10.1016/j.stemcr.2018.07.010
   PubMed Web of Science ®Google Scholar
• Yang YP, Nguyen PNN, Ma HI, et al. Tumor mesenchymal stromal cells regulate cell migration of atypical teratoid rhabdoid tumor through exosome-mediated miR155/SMARCA4 pathway. Cancers (Basel). 2019;11(5):720. doi:10.3390/cancers11050720
   PubMed Web of Science ®Google Scholar
• Howard TP, Arnoff TE, Song MR, Giacomelli AO. MDM2 and MDM4 are therapeutic vulnerabilities in malignant rhabdoid tumors. Cancer Res. 2019;79(9):2404–2414. doi:10.1158/0008-5472.CAN-18-3066
   PubMed Web of Science ®Google Scholar
• Nakano Y, Takadera M, Miyazaki M, et al. Drug screening with a novel tumor-derived cell line identified alternative therapeutic options for patients with atypical teratoid/rhabdoid tumor. Hum Cell. 2021;34(1):271–278. doi:10.1007/s13577-020-00438-3
   PubMed Web of Science ®Google Scholar
• Morin A, Soane C, Pierce A, et al. Proteasome inhibition as a therapeutic approach in atypical teratoid/rhabdoid tumors. Neurooncol Adv. 2020;2(1):vdaa051. doi:10.1093/noajnl/vdaa051
   PubMedGoogle Scholar
• Tran HM, Wu KS, Sung SY. Upregulation of protein synthesis and proteasome degradation confers sensitivity to proteasome inhibitor bortezomib in Myc-atypical teratoid/rhabdoid tumors. Cancers (Basel). 2020;12(3):752. doi:10.3390/cancers12030752
   PubMed Web of Science ®Google Scholar
• Howard TP, Oberlick EM, Rees MG. Rhabdoid tumors are sensitive to the protein-translation inhibitor homoharringtonine. Clin Cancer Res. 2020;26(18):4995–5006. doi:10.1158/1078-0432.CCR-19-2717
   PubMed Web of Science ®Google Scholar
• Shibui Y, Kohashi K, Tamaki A, et al. The forkhead box M1 (FOXM1) expression and antitumor effect of FOXM1 inhibition in malignant rhabdoid tumor. J Cancer Res Clin Oncol. 2021;147(5):1499–1518. doi:10.1007/s00432-020-03438-w
   PubMed Web of Science ®Google Scholar
• Daifu T, Mikami M, Hiramatsu H, Iwai A, Umeda K. Suppression of malignant rhabdoid tumors through Chb-M’-mediated RUNX1 inhibition. Pediatr Blood Cancer. 2020;68(2):e28789. doi:10.1002/pbc.28789
   PubMed Web of Science ®Google Scholar
• Marsh IR, Grudzinski J, Baiu DC, et al. Preclinical pharmacokinetics and dosimetry studies of (124)I/(131) I-CLR1404 for treatment of pediatric solid tumors in murine xenograft models. J Nucl Med. 2019;60(10):1414–1420. doi:10.2967/jnumed.118.225409
   PubMed Web of Science ®Google Scholar
• Krämer KF, Moreno N, Frühwald MC, Kerl K. BRD9 inhibition, alone or in combination with cytostatic compounds as a therapeutic approach in rhabdoid tumors. Int J Mol Sci. 2017;18(7):1537. doi:10.3390/ijms18071537
   PubMed Web of Science ®Google Scholar
• Michel BC, D’Avino AR, Cassel SH, et al. A non-canonical SWI/SNF complex is a synthetic lethal target in cancers driven by BAF complex perturbation. Nat Cell Biol. 2018;20(12):1410–1420. doi:10.1038/s41556-018-0221-1
   PubMed Web of Science ®Google Scholar
• Kim KH, Roberts CW. Mechanisms by which SMARCB1 loss drives rhabdoid tumor growth. Cancer Genet. 2014;207(9):365–372. doi:10.1016/j.cancergen.2014.04.004
   PubMed Web of Science ®Google Scholar
• Carugo A, Minelli R, Sapio L, et al. p53 is a master regulator of proteostasis in SMARCB1-deficient malignant rhabdoid tumors. Cancer Cell. 2019;35(2):204–220.e9. doi:10.1016/j.ccell.2019.01.006
   PubMed Web of Science ®Google Scholar
• Perla A, Fratini L, Cardoso PS, et al. Histone deacetylase inhibitors in pediatric brain cancers: biological activities and therapeutic potential. Front Cell Dev Biol. 2020;8:546. doi:10.3389/fcell.2020.00546
   PubMed Web of Science ®Google Scholar
• Kerl K, Ries D, Unland R, et al. The histone deacetylase inhibitor SAHA acts in synergism with fenretinide and doxorubicin to control growth of rhabdoid tumor cells. BMC Cancer. 2013;13:286. doi:10.1186/1471-2407-13-286
   PubMed Web of Science ®Google Scholar
• Muscat A, Popovski D, Jayasekara WS, et al. Low-dose histone deacetylase inhibitor treatment leads to tumor growth arrest and multi-lineage differentiation of malignant rhabdoid tumors. Clin Cancer Res. 2016;22(14):3560–3570. doi:10.1158/1078-0432.CCR-15-2260
   PubMed Web of Science ®Google Scholar
• Custers L, Khabirova E, Coorens THH. Somatic mutations and single-cell transcriptomes reveal the root of malignant rhabdoid tumours. Nat Commun. 2021;12(1):1407. doi:10.1038/s41467-021-21675-6
   PubMed Web of Science ®Google Scholar
• Hoffman MM, Zylla JS, Bhattacharya S, et al. Analysis of dual class I histone deacetylase and lysine demethylase inhibitor domatinostat (4SC-202) on growth and cellular and genomic landscape of atypical teratoid/rhabdoid. Cancers (Basel). 2020;12(3):756. doi:10.3390/cancers12030756
   PubMed Web of Science ®Google Scholar
• Sugimoto Y, Katsumi Y, Iehara T. The novel histone deacetylase inhibitor, OBP-801, induces apoptosis in rhabdoid tumors by releasing the silencing of NOXA. Mol Cancer Ther. 2020;19(10):1992–2000. doi:10.1158/1535-7163.MCT-20-0243
   PubMed Web of Science ®Google Scholar
• Knipstein JA, Birks DK, Donson AM, Alimova I, Foreman NK, Vibhakar R. Histone deacetylase inhibition decreases proliferation and potentiates the effect of ionizing radiation in atypical teratoid/rhabdoid tumor cells. Neuro Oncol. 2012;14(2):175–183. doi:10.1093/neuonc/nor208
   PubMed Web of Science ®Google Scholar
• Thiemann M, Oertel S, Ehemann V, et al. In vivo efficacy of the histone deacetylase inhibitor suberoylanilide hydroxamic acid in combination with radiotherapy in a malignant rhabdoid tumor mouse model. Radiat Oncol. 2012;7:52. doi:10.1186/1748-717X-7-52
   PubMed Web of Science ®Google Scholar
• Muscal JA, Thompson PA, Horton TM, et al. A phase I trial of vorinostat and bortezomib in children with refractory or recurrent solid tumors: a Children’s Oncology Group phase I consortium study (ADVL0916). Pediatr Blood Cancer. 2013;60(3):390–395. doi:10.1002/pbc.24271
   PubMed Web of Science ®Google Scholar
• Fouladi M, Park JR, Stewart CF, et al. Pediatric phase I trial and pharmacokinetic study of vorinostat: a Children’s Oncology Group phase I consortium report. J Clin Oncol. 2010;28(22):3623–3629. doi:10.1200/JCO.2009.25.9119
   PubMed Web of Science ®Google Scholar
• Su JM, Thompson P, Ou CN, et al. Phase 1 study of valproic acid in pediatric patients with refractory solid or CNS tumors: a children’s oncology group report. Clin Cancer Res. 2011;17(3):589–597. doi:10.1158/1078-0432.CCR-10-0738
   PubMed Web of Science ®Google Scholar
• Yan W, Herman JG, Guo M. Epigenome-based personalized medicine in human cancer. Epigenomics. 2016;8(1):119–133. doi:10.2217/epi.15.84
   PubMed Web of Science ®Google Scholar
• George RE, Lahti JM, Adamson PC, et al. Phase I study of decitabine with doxorubicin and cyclophosphamide in children with neuroblastoma and other solid tumors: a Children’s Oncology Group study. Pediatr Blood Cancer. 2010;55(4):629–638. doi:10.1002/pbc.22607
   PubMed Web of Science ®Google Scholar
• Margueron R, Reinberg D. The Polycomb complex PRC2 and its mark in life. Nature. 2011;469(7330):343–349. doi:10.1038/nature09784
   PubMed Web of Science ®Google Scholar
• Kim KH, Roberts CW. Targeting EZH2 in cancer. Nat Med. 2016;22(2):128–134. doi:10.1038/nm.4036
   PubMed Web of Science ®Google Scholar
• Alimova I, Birks DK, Harris PS, et al. Inhibition of EZH2 suppresses self-renewal and induces radiation sensitivity in atypical rhabdoid teratoid tumor cells. Neuro Oncol. 2013;15(2):149–160. doi:10.1093/neuonc/nos285
   PubMed Web of Science ®Google Scholar
• Kurmasheva RT, Sammons M, Favours E, et al. Initial testing (stage 1) of tazemetostat (EPZ-6438), a novel EZH2 inhibitor, by the pediatric preclinical testing program. Pediatr Blood Cancer. 2017;64(3):e26218. doi:10.1002/pbc.26218
   Web of Science ®Google Scholar
• Kurmasheva RT, Erickson SW, Earley E, Smith MA, Houghton PJ. In vivo evaluation of the EZH2 inhibitor (EPZ011989) alone or in combination with standard of care cytotoxic agents against pediatric malignant rhabdoid tumor preclinical models-A report from the Pediatric Preclinical Testing Consortium. Pediatr Blood Cancer. 2021;68(2):e28772. doi:10.1002/pbc.28772
   PubMed Web of Science ®Google Scholar
• Italiano A, Soria JC, Toulmonde M, et al. Tazemetostat, an EZH2 inhibitor, in relapsed or refractory B-cell non-Hodgkin lymphoma and advanced solid tumours: a first-in-human, open-label, phase 1 study. Lancet Oncol. 2018;19(5):649–659. doi:10.1016/S1470-2045(18)30145-1
   PubMed Web of Science ®Google Scholar
• Chi NS, Bourdeaut F, Laetsch TW et al. Phase 1 Study of Tazemetostat, an Enhancer of Zeste Homolog-2 Inhibitor, Pediatric Patients With Relapsed/Refractory Integrase Interacor 1-Negative Tumors. ASCO 2020. Available from: https://www.epizyme.com/wp-content/uploads/2021/06/EZH-102_ASCO-2020_Poster_Final.pdf. Accessed January 11, 2022.
   Google Scholar
• Kohashi K, Oda Y. Oncogenic roles of SMARCB1/INI1 and its deficient tumors. Cancer Sci. 2017;108(4):547–552. doi:10.1111/cas.13173
   PubMed Web of Science ®Google Scholar
• Cimica V, Smith ME, Zhang Z, Mathur D, Mani S, Kalpana GV. Potent inhibition of rhabdoid tumor cells by combination of flavopiridol and 4OH-tamoxifen. BMC Cancer. 2010;10:634. doi:10.1186/1471-2407-10-634
   PubMed Web of Science ®Google Scholar
• Hashizume R, Zhang A, Mueller S, et al. Inhibition of DNA damage repair by the CDK4/6 inhibitor palbociclib delays irradiated intracranial atypical teratoid rhabdoid tumor and glioblastoma xenograft regrowth. Neuro Oncol. 2016;18(11):1519–1528. doi:10.1093/neuonc/now106.euro-oncology
   PubMed Web of Science ®Google Scholar
• Geoerger B, Bourdeaut F, DuBois SG, et al. A phase I study of the CDK4/6 inhibitor ribociclib (LEE011) in pediatric patients with malignant rhabdoid tumors, neuroblastoma, and other solid tumors. Clin Cancer Res. 2017;23(10):2433–2441. doi:10.1158/1078-0432.CCR-16-2898
   PubMed Web of Science ®Google Scholar
• Maris JM, Morton CL, Gorlick R, et al. Initial testing of the Aurora kinase A inhibitor MLN8237 by the Pediatric Preclinical Testing Program (PPTP). Pediatr Blood Cancer. 2010;55(1):26–34. doi:10.1002/pbc.22430
   PubMed Web of Science ®Google Scholar
• Venkataraman S, Alimova I, Tello T, et al. Targeting Aurora Kinase A enhances radiation sensitivity of atypical teratoid rhabdoid tumor cells. J Neurooncol. 2012;107(3):517–526. doi:10.1007/s11060-011-0795-y
   PubMed Web of Science ®Google Scholar
• Wetmore C, Boyett J, Li S, et al. Alisertib is active as single agent in recurrent atypical teratoid rhabdoid tumors in 4 children. Neuro Oncol. 2015;17(6):882–888. doi:10.1093/neuonc/nov017
   PubMed Web of Science ®Google Scholar
• Mossé YP, Fox E, Teachey DT. A phase II study of alisertib in children with recurrent/refractory solid tumors or leukemia: children’s oncology group phase I and pilot consortium (ADVL0921). Clin Cancer Res. 2019;25(11):3229–3238. doi:10.1158/1078-0432.CCR-18-2675
   PubMed Web of Science ®Google Scholar
• Davis SL, Ionkina AA, Bagby SM, Orth JD, Gittleman B. Preclinical and dose-finding phase I trial results of combined treatment with a TORC1/2 inhibitor (TAK-228) and Aurora A kinase inhibitor (alisertib) in solid tumors. Clin Cancer Res. 2020;26(17):4633–4642. doi:10.1158/1078-0432.CCR-19-3498
   PubMed Web of Science ®Google Scholar
• Upadhyaya S, Campagne O, Robinson GW. Phase II study of alisertib as a single agent in recurrent or progressive atypical teratoid rhabdoid tumors. J Clin Oncol. 2020;38(15_suppl):10542. doi:10.1200/JCO.2020.38.15_suppl.10542
   Web of Science ®Google Scholar
• Wedekind MF, Denton NL, Chen CY, Cripe TP. Pediatric cancer immunotherapy: opportunities and challenges. Paediatr Drugs. 2018;20(5):395–408. doi:10.1007/s40272-018-0297-x
   PubMed Web of Science ®Google Scholar
• Michot JM, Bigenwald C, Champiat S, et al. Immune-related adverse events with immune checkpoint blockade: a comprehensive review. Eur J Cancer. 2016;54:139–148. doi:10.1016/j.ejca.2015.11.016
   PubMed Web of Science ®Google Scholar
• Binnewies M, Roberts EW, Kersten K, et al. Understanding the tumor immune microenvironment (TIME) for effective therapy. Nat Med. 2018;24(5):541–550. doi:10.1038/s41591-018-0014-x
   PubMed Web of Science ®Google Scholar
• Hirata E, Sahai E. Tumor microenvironment and differential responses to therapy. Cold Spring Harb Perspect Med. 2017;7(7):a026781. doi:10.1101/cshperspect.a026781
   PubMed Web of Science ®Google Scholar
• Grabovska Y, Mackay A, O’Hare P, Crosier S, Finetti M. Pediatric pan-central nervous system tumor analysis of immune-cell infiltration identifies correlates of antitumor immunity. Nat Commun. 2020;11(1):4324. doi:10.1038/s41467-020-18070-y
   PubMed Web of Science ®Google Scholar
• Petralia F, Tignor N, Reva B, et al. Integrated proteogenomic characterization across major histological types of pediatric brain cancer. Cell. 2020;183(7):1962–1985.e31. doi:10.1016/j.cell.2020.10.044
   PubMed Web of Science ®Google Scholar
• Samstein RM, Lee CH, Shoushtari AN. Tumor mutational load predicts survival after immunotherapy across multiple cancer types. Nat Genet. 2019;51(2):202–206. doi:10.1038/s41588-018-0312-8
   PubMed Web of Science ®Google Scholar
• Gröbner SN, Worst BC, Weischenfeldt J, et al. The landscape of genomic alterations across childhood cancers. Nature. 2018;555(7696):321–327. doi:10.1038/nature25480
   PubMed Web of Science ®Google Scholar
• Yarmarkovich M, Maris JM. When cold is hot: immune checkpoint inhibition therapy for rhabdoid tumors. Cancer Cell. 2019;36(6):575–576. doi:10.1016/j.ccell.2019.11.006
   PubMed Web of Science ®Google Scholar
• Leruste A, Tosello J, Ramos RN, et al. Clonally expanded T cells reveal immunogenicity of rhabdoid tumors. Cancer Cell. 2019;36(6):597–612.e8. doi:10.1016/j.ccell.2019.10.008
   PubMed Web of Science ®Google Scholar
• Bourdeaut F, Thaku MD, Bergthold G, Karski E. ATRT-11. Marked response to Atezolizumab in a patient with rhabdoid tumor: a case study from the imatrix-atezolizumab trial. Neuro-Oncology. 2017;19(Suppl4):iv3. doi:10.1093/neuonc/nox083.010
   Google Scholar
• Geoerger B, Zwaan CM, Marshall LV, et al. Atezolizumab for children and young adults with previously treated solid tumours, non-Hodgkin lymphoma, and Hodgkin lymphoma (iMATRIX): a multicentre phase 1–2 study. Lancet Oncol. 2020;21(1):134–144. doi:10.1016/S1470-2045(19)30693-X
   PubMed Web of Science ®Google Scholar
• Geoerger B, Kang HJ, Yalon-Oren M, et al. Pembrolizumab in paediatric patients with advanced melanoma or a PD-L1-positive, advanced, relapsed, or refractory solid tumour or lymphoma (KEYNOTE-051): interim analysis of an open-label, single-arm, phase 1–2 trial. Lancet Oncol. 2020;21(1):121–133. doi:10.1016/S1470-2045(19)30671-0
   PubMed Web of Science ®Google Scholar
• Hoppmann A, Williams AP, Coleman A, et al. Partial response to carboplatin, etoposide phosphate, and atezolizumab in a pediatric patient with high-grade metastatic tumor with rhabdoid and focal neuroendocrine features. Pediatr Blood Cancer. 2020;67(2):e28048. doi:10.1002/pbc.28048
   PubMed Web of Science ®Google Scholar
• Morrissey KM, Yuraszeck TM, Li CC, Zhang Y, Kasichayanula S. Immunotherapy and novel combinations in oncology: current landscape, challenges, and opportunities. Clin Transl Sci. 2016;9(2):89–104. doi:10.1111/cts.12391
   PubMed Web of Science ®Google Scholar
• Wang D, Quiros J, Mahuron K, et al. Targeting EZH2 reprograms intratumoral regulatory T cells to enhance cancer immunity. Cell Rep. 2018;23(11):3262–3274. doi:10.1016/j.celrep.2018.05.050
   PubMed Web of Science ®Google Scholar
• Goel S, DeCristo MJ, Watt AC, et al. CDK4/6 inhibition triggers anti-tumour immunity. Nature. 2017;548(7668):471–475. doi:10.1038/nature23465
   PubMed Web of Science ®Google Scholar
• Leruste A, Chauvin C, Pouponnot C, Bourdeaut F, Waterfall JJ. Immune responses in genomically simple SWI/SNF-deficient cancers. Nature. 2017;548(7668):471–475. doi:10.1038/nature23465
   PubMed Web of Science ®Google Scholar
• Majzner RG, Theruvath JL, Nellan A, et al. CAR T cells targeting B7-H3, a pan-cancer antigen, demonstrate potent preclinical activity against pediatric solid tumors and brain tumors. Clin Cancer Res. 2019;25(8):2560–2574. doi:10.1158/1078-0432.CCR-18-0432
   PubMed Web of Science ®Google Scholar
• Du H, Hirabayashi K, Ahn S, et al. Antitumor responses in the absence of toxicity in solid tumors by targeting B7-H3 via chimeric antigen receptor T cells. Cancer Cell. 2019;35(2):221–237.e8. doi:10.1016/j.ccell.2019.01.002
   PubMed Web of Science ®Google Scholar
• Theruvath J, Sotillo E, Mount CW, et al. Locoregionally administered B7-H3-targeted CAR T cells for treatment of atypical teratoid/rhabdoid tumors. Nat Med. 2020;26(5):712–719. doi:10.1038/s41591-020-0821-8
   PubMed Web of Science ®Google Scholar
• Chauvin C, Leruste A, Tauziede-Espariat A, et al. High-throughput drug screening identifies pazopanib and clofilium tosylate as promising treatments for malignant rhabdoid tumors. Cell Rep. 2017;21(7):1737–1745. doi:10.1016/j.celrep.2017.10.076
   PubMed Web of Science ®Google Scholar
• Wong JP, Todd JR, Finetti MA, et al. Dual targeting of PDGFRα and FGFR1 displays synergistic efficacy in malignant rhabdoid tumors. Cell Rep. 2016;17(5):1265–1275. doi:10.1016/j.celrep.2016.10.005
   PubMed Web of Science ®Google Scholar