Skip to main content
Top
Published in: Molecular and Cellular Pediatrics 1/2018

Open Access 01-12-2018 | Review

Precision medicine in pediatric oncology

Authors: Stefan E. G. Burdach, Mike-Andrew Westhoff, Maximilian Felix Steinhauser, Klaus-Michael Debatin

Published in: Molecular and Cellular Pediatrics | Issue 1/2018

Login to get access

Abstract

Outcome in treatment of childhood cancers has improved dramatically since the 1970s. This success was largely achieved by the implementation of cooperative clinical research trial groups that standardized and developed treatment of childhood cancer. Nevertheless, outcome in certain types of malignancies is still unfavorable. Intensification of conventional chemotherapy and radiotherapy improved outcome only marginally at the cost of acute and long-term side effects. Hence, it is necessary to develop targeted therapy strategies.
Here, we review the developments and perspectives in precision medicine in pediatric oncology with a special focus on targeted drug therapies like kinase inhibitors and inducers of apoptosis, the impact of cancer genome sequencing and immunotherapy.
Literature
10.
go back to reference Nowell PC, Hungerford DA (1960) A minute chromosome in human chronic granulocytic leukemia. Science 132:1497–1501 Nowell PC, Hungerford DA (1960) A minute chromosome in human chronic granulocytic leukemia. Science 132:1497–1501
13.
go back to reference Bartram CR, de Klein A, Hagemeijer A, van Agthoven T, Geurts van Kessel A, Bootsma D, Grosveld G, Ferguson-Smith MA, Davies T, Stone M et al (1983) Translocation of c-ab1 oncogene correlates with the presence of a Philadelphia chromosome in chronic myelocytic leukaemia. Nature 306:277–280CrossRefPubMed Bartram CR, de Klein A, Hagemeijer A, van Agthoven T, Geurts van Kessel A, Bootsma D, Grosveld G, Ferguson-Smith MA, Davies T, Stone M et al (1983) Translocation of c-ab1 oncogene correlates with the presence of a Philadelphia chromosome in chronic myelocytic leukaemia. Nature 306:277–280CrossRefPubMed
14.
go back to reference Lampert F (1967) Desoxyribonucleic acid distribution pattern in acute leukemia in childhood. Monatsschr Kinderheilkd 115:293–294PubMed Lampert F (1967) Desoxyribonucleic acid distribution pattern in acute leukemia in childhood. Monatsschr Kinderheilkd 115:293–294PubMed
16.
go back to reference Mullighan CG, Su X, Zhang J, Radtke I, Phillips LA, Miller CB, Ma J, Liu W, Cheng C, Schulman BA, Harvey RC, Chen IM, Clifford RJ, Carroll WL, Reaman G, Bowman WP, Devidas M, Gerhard DS, Yang W, Relling MV, Shurtleff SA, Campana D, Borowitz MJ, Pui CH, Smith M, Hunger SP, Willman CL, Downing JR, Children's Oncology G (2009) Deletion of IKZF1 and prognosis in acute lymphoblastic leukemia. N Engl J Med 360:470–480. https://doi.org/10.1056/NEJMoa0808253CrossRefPubMedPubMedCentral Mullighan CG, Su X, Zhang J, Radtke I, Phillips LA, Miller CB, Ma J, Liu W, Cheng C, Schulman BA, Harvey RC, Chen IM, Clifford RJ, Carroll WL, Reaman G, Bowman WP, Devidas M, Gerhard DS, Yang W, Relling MV, Shurtleff SA, Campana D, Borowitz MJ, Pui CH, Smith M, Hunger SP, Willman CL, Downing JR, Children's Oncology G (2009) Deletion of IKZF1 and prognosis in acute lymphoblastic leukemia. N Engl J Med 360:470–480. https://​doi.​org/​10.​1056/​NEJMoa0808253CrossRefPubMedPubMedCentral
18.
go back to reference Grobner SN, Worst BC, Weischenfeldt J, Buchhalter I, Kleinheinz K, Rudneva VA, Johann PD, Balasubramanian GP, Segura-Wang M, Brabetz S, Bender S, Hutter B, Sturm D, Pfaff E, Hubschmann D, Zipprich G, Heinold M, Eils J, Lawerenz C, Erkek S, Lambo S, Waszak S, Blattmann C, Borkhardt A, Kuhlen M, Eggert A, Fulda S, Gessler M, Wegert J, Kappler R, Baumhoer D, Burdach S, Kirschner-Schwabe R, Kontny U, Kulozik AE, Lohmann D, Hettmer S, Eckert C, Bielack S, Nathrath M, Niemeyer C, Richter GH, Schulte J, Siebert R, Westermann F, Molenaar JJ, Vassal G, Witt H, Project IP-S, Project IM-S, Burkhardt B, Kratz CP, Witt O, van Tilburg CM, Kramm CM, Fleischhack G, Dirksen U, Rutkowski S, Fruhwald M, von Hoff K, Wolf S, Klingebiel T, Koscielniak E, Landgraf P, Koster J, Resnick AC, Zhang J, Liu Y, Zhou X, Waanders AJ, Zwijnenburg DA, Raman P, Brors B, Weber UD, Northcott PA, Pajtler KW, Kool M, Piro RM, Korbel JO, Schlesner M, Eils R, Jones DTW, Lichter P, Chavez L, Zapatka M, Pfister SM (2018) The landscape of genomic alterations across childhood cancers. Nature. https://doi.org/10.1038/nature25480 Grobner SN, Worst BC, Weischenfeldt J, Buchhalter I, Kleinheinz K, Rudneva VA, Johann PD, Balasubramanian GP, Segura-Wang M, Brabetz S, Bender S, Hutter B, Sturm D, Pfaff E, Hubschmann D, Zipprich G, Heinold M, Eils J, Lawerenz C, Erkek S, Lambo S, Waszak S, Blattmann C, Borkhardt A, Kuhlen M, Eggert A, Fulda S, Gessler M, Wegert J, Kappler R, Baumhoer D, Burdach S, Kirschner-Schwabe R, Kontny U, Kulozik AE, Lohmann D, Hettmer S, Eckert C, Bielack S, Nathrath M, Niemeyer C, Richter GH, Schulte J, Siebert R, Westermann F, Molenaar JJ, Vassal G, Witt H, Project IP-S, Project IM-S, Burkhardt B, Kratz CP, Witt O, van Tilburg CM, Kramm CM, Fleischhack G, Dirksen U, Rutkowski S, Fruhwald M, von Hoff K, Wolf S, Klingebiel T, Koscielniak E, Landgraf P, Koster J, Resnick AC, Zhang J, Liu Y, Zhou X, Waanders AJ, Zwijnenburg DA, Raman P, Brors B, Weber UD, Northcott PA, Pajtler KW, Kool M, Piro RM, Korbel JO, Schlesner M, Eils R, Jones DTW, Lichter P, Chavez L, Zapatka M, Pfister SM (2018) The landscape of genomic alterations across childhood cancers. Nature. https://​doi.​org/​10.​1038/​nature25480
19.
go back to reference Allen CE, Laetsch TW, Mody R, Irwin MS, Lim MS, Adamson PC, Seibel NL, Parsons DW, Cho YJ, Janeway K, Pediatric MT, Agent Prioritization C (2017) Target and agent prioritization for the children’s oncology Group-National Cancer Institute Pediatric MATCH Trial. J Natl Cancer Inst 109. https://doi.org/10.1093/jnci/djw274 Allen CE, Laetsch TW, Mody R, Irwin MS, Lim MS, Adamson PC, Seibel NL, Parsons DW, Cho YJ, Janeway K, Pediatric MT, Agent Prioritization C (2017) Target and agent prioritization for the children’s oncology Group-National Cancer Institute Pediatric MATCH Trial. J Natl Cancer Inst 109. https://​doi.​org/​10.​1093/​jnci/​djw274
21.
go back to reference Worst BC, van Tilburg CM, Balasubramanian GP, Fiesel P, Witt R, Freitag A, Boudalil M, Previti C, Wolf S, Schmidt S, Chotewutmontri S, Bewerunge-Hudler M, Schick M, Schlesner M, Hutter B, Taylor L, Borst T, Sutter C, Bartram CR, Milde T, Pfaff E, Kulozik AE, von Stackelberg A, Meisel R, Borkhardt A, Reinhardt D, Klusmann JH, Fleischhack G, Tippelt S, Dirksen U, Jurgens H, Kramm CM, von Bueren AO, Westermann F, Fischer M, Burkhardt B, Wossmann W, Nathrath M, Bielack SS, Fruhwald MC, Fulda S, Klingebiel T, Koscielniak E, Schwab M, Tremmel R, Driever PH, Schulte JH, Brors B, von Deimling A, Lichter P, Eggert A, Capper D, Pfister SM, Jones DT, Witt O (2016) Next-generation personalised medicine for high-risk paediatric cancer patients - the INFORM pilot study. Eur J Cancer 65:91–101. https://doi.org/10.1016/j.ejca.2016.06.009CrossRefPubMed Worst BC, van Tilburg CM, Balasubramanian GP, Fiesel P, Witt R, Freitag A, Boudalil M, Previti C, Wolf S, Schmidt S, Chotewutmontri S, Bewerunge-Hudler M, Schick M, Schlesner M, Hutter B, Taylor L, Borst T, Sutter C, Bartram CR, Milde T, Pfaff E, Kulozik AE, von Stackelberg A, Meisel R, Borkhardt A, Reinhardt D, Klusmann JH, Fleischhack G, Tippelt S, Dirksen U, Jurgens H, Kramm CM, von Bueren AO, Westermann F, Fischer M, Burkhardt B, Wossmann W, Nathrath M, Bielack SS, Fruhwald MC, Fulda S, Klingebiel T, Koscielniak E, Schwab M, Tremmel R, Driever PH, Schulte JH, Brors B, von Deimling A, Lichter P, Eggert A, Capper D, Pfister SM, Jones DT, Witt O (2016) Next-generation personalised medicine for high-risk paediatric cancer patients - the INFORM pilot study. Eur J Cancer 65:91–101. https://​doi.​org/​10.​1016/​j.​ejca.​2016.​06.​009CrossRefPubMed
22.
go back to reference Weidenbusch B, Richter GHS, Kesper MS, Guggemoos M, Gall K, Prexler C, Kazantsev I, Sipol A, Lindner L, Nathrath M, Witt O, Specht K, Beitinger F, Knebel C, Hosie S, von Eisenhardt-Rothe R, Weichert W, Teichert-von Luettichau I, Burdach S (2018) Transcriptome based individualized therapy of refractory pediatric sarcomas: feasibility, tolerability and efficacy. Oncotarget 29:20747–20760. https://doi.org/10.18632/oncotarget.25087 Weidenbusch B, Richter GHS, Kesper MS, Guggemoos M, Gall K, Prexler C, Kazantsev I, Sipol A, Lindner L, Nathrath M, Witt O, Specht K, Beitinger F, Knebel C, Hosie S, von Eisenhardt-Rothe R, Weichert W, Teichert-von Luettichau I, Burdach S (2018) Transcriptome based individualized therapy of refractory pediatric sarcomas: feasibility, tolerability and efficacy. Oncotarget 29:20747–20760. https://​doi.​org/​10.​18632/​oncotarget.​25087
23.
go back to reference Harris MH, DuBois SG, Glade Bender JL, Kim A, Crompton BD, Parker E, Dumont IP, Hong AL, Guo D, Church A, Stegmaier K, Roberts CW, Shusterman S, London WB, MacConaill LE, Lindeman NI, Diller L, Rodriguez-Galindo C, Janeway KA (2016) Multicenter feasibility study of tumor molecular profiling to inform therapeutic decisions in advanced pediatric solid tumors: the individualized cancer therapy (iCat) study. JAMA Oncol. https://doi.org/10.1001/jamaoncol.2015.5689 Harris MH, DuBois SG, Glade Bender JL, Kim A, Crompton BD, Parker E, Dumont IP, Hong AL, Guo D, Church A, Stegmaier K, Roberts CW, Shusterman S, London WB, MacConaill LE, Lindeman NI, Diller L, Rodriguez-Galindo C, Janeway KA (2016) Multicenter feasibility study of tumor molecular profiling to inform therapeutic decisions in advanced pediatric solid tumors: the individualized cancer therapy (iCat) study. JAMA Oncol. https://​doi.​org/​10.​1001/​jamaoncol.​2015.​5689
24.
go back to reference Mody RJ, Wu YM, Lonigro RJ, Cao X, Roychowdhury S, Vats P, Frank KM, Prensner JR, Asangani I, Palanisamy N, Dillman JR, Rabah RM, Kunju LP, Everett J, Raymond VM, Ning Y, Su F, Wang R, Stoffel EM, Innis JW, Roberts JS, Robertson PL, Yanik G, Chamdin A, Connelly JA, Choi S, Harris AC, Kitko C, Rao RJ, Levine JE, Castle VP, Hutchinson RJ, Talpaz M, Robinson DR, Chinnaiyan AM (2015) Integrative clinical sequencing in the management of refractory or relapsed cancer in youth. JAMA 314:913–925. https://doi.org/10.1001/jama.2015.10080CrossRefPubMedPubMedCentral Mody RJ, Wu YM, Lonigro RJ, Cao X, Roychowdhury S, Vats P, Frank KM, Prensner JR, Asangani I, Palanisamy N, Dillman JR, Rabah RM, Kunju LP, Everett J, Raymond VM, Ning Y, Su F, Wang R, Stoffel EM, Innis JW, Roberts JS, Robertson PL, Yanik G, Chamdin A, Connelly JA, Choi S, Harris AC, Kitko C, Rao RJ, Levine JE, Castle VP, Hutchinson RJ, Talpaz M, Robinson DR, Chinnaiyan AM (2015) Integrative clinical sequencing in the management of refractory or relapsed cancer in youth. JAMA 314:913–925. https://​doi.​org/​10.​1001/​jama.​2015.​10080CrossRefPubMedPubMedCentral
25.
go back to reference Parsons DW, Roy A, Yang Y, Wang T, Scollon S, Bergstrom K, Kerstein RA, Gutierrez S, Petersen AK, Bavle A, Lin FY, Lopez-Terrada DH, Monzon FA, Hicks MJ, Eldin KW, Quintanilla NM, Adesina AM, Mohila CA, Whitehead W, Jea A, Vasudevan SA, Nuchtern JG, Ramamurthy U, McGuire AL, Hilsenbeck SG, Reid JG, Muzny DM, Wheeler DA, Berg SL, Chintagumpala MM, Eng CM, Gibbs RA, Plon SE (2016) Diagnostic yield of clinical tumor and germline whole-exome sequencing for children with solid tumors. JAMA Oncol. https://doi.org/10.1001/jamaoncol.2015.5699 Parsons DW, Roy A, Yang Y, Wang T, Scollon S, Bergstrom K, Kerstein RA, Gutierrez S, Petersen AK, Bavle A, Lin FY, Lopez-Terrada DH, Monzon FA, Hicks MJ, Eldin KW, Quintanilla NM, Adesina AM, Mohila CA, Whitehead W, Jea A, Vasudevan SA, Nuchtern JG, Ramamurthy U, McGuire AL, Hilsenbeck SG, Reid JG, Muzny DM, Wheeler DA, Berg SL, Chintagumpala MM, Eng CM, Gibbs RA, Plon SE (2016) Diagnostic yield of clinical tumor and germline whole-exome sequencing for children with solid tumors. JAMA Oncol. https://​doi.​org/​10.​1001/​jamaoncol.​2015.​5699
26.
go back to reference Harttrampf AC, Lacroix L, Deloger M, Deschamps F, Puget S, Auger N, Vielh P, Varlet P, Balogh Z, Abbou S, Allorant A, Valteau-Couanet D, Sarnacki S, Gamiche-Rolland L, Meurice G, Minard-Colin V, Grill J, Brugieres L, Dufour C, Gaspar N, Michiels S, Vassal G, Soria JC, Geoerger B (2017) Molecular screening for cancer treatment optimization (MOSCATO-01) in pediatric patients: a single-institutional prospective molecular stratification trial. Clin Cancer Res 23:6101–6112. https://doi.org/10.1158/1078-0432.CCR-17-0381CrossRefPubMed Harttrampf AC, Lacroix L, Deloger M, Deschamps F, Puget S, Auger N, Vielh P, Varlet P, Balogh Z, Abbou S, Allorant A, Valteau-Couanet D, Sarnacki S, Gamiche-Rolland L, Meurice G, Minard-Colin V, Grill J, Brugieres L, Dufour C, Gaspar N, Michiels S, Vassal G, Soria JC, Geoerger B (2017) Molecular screening for cancer treatment optimization (MOSCATO-01) in pediatric patients: a single-institutional prospective molecular stratification trial. Clin Cancer Res 23:6101–6112. https://​doi.​org/​10.​1158/​1078-0432.​CCR-17-0381CrossRefPubMed
27.
go back to reference Geoerger B, Schleiermacher G, Pierron G, Lacroix L, Deloger M, Bessoltane N, Harttrampf AC, Michiels S, Scoazec JY, Freneaux P, Paoletti X, Delattre O, Hoog-Labouret N, Vassal G (2017) Abstract CT004: European pediatric precision medicine program in recurrent tumors: first results from MAPPYACTS molecular profiling trial towards AcSe-ESMART proof-of-concept study. Cancer Research, American Association for Cancer Research, p CT004. https://doi.org/10.1158/1538-7445.AM2017-CT004 Geoerger B, Schleiermacher G, Pierron G, Lacroix L, Deloger M, Bessoltane N, Harttrampf AC, Michiels S, Scoazec JY, Freneaux P, Paoletti X, Delattre O, Hoog-Labouret N, Vassal G (2017) Abstract CT004: European pediatric precision medicine program in recurrent tumors: first results from MAPPYACTS molecular profiling trial towards AcSe-ESMART proof-of-concept study. Cancer Research, American Association for Cancer Research, p CT004. https://​doi.​org/​10.​1158/​1538-7445.​AM2017-CT004
29.
37.
go back to reference Zhang J, Walsh MF, Wu G, Edmonson MN, Gruber TA, Easton J, Hedges D, Ma X, Zhou X, Yergeau DA, Wilkinson MR, Vadodaria B, Chen X, McGee RB, Hines-Dowell S, Nuccio R, Quinn E, Shurtleff SA, Rusch M, Patel A, Becksfort JB, Wang S, Weaver MS, Ding L, Mardis ER, Wilson RK, Gajjar A, Ellison DW, Pappo AS, Pui CH, Nichols KE, Downing JR (2015) Germline mutations in predisposition genes in pediatric cancer. N Engl J Med 373:2336–2346. https://doi.org/10.1056/NEJMoa1508054CrossRefPubMedPubMedCentral Zhang J, Walsh MF, Wu G, Edmonson MN, Gruber TA, Easton J, Hedges D, Ma X, Zhou X, Yergeau DA, Wilkinson MR, Vadodaria B, Chen X, McGee RB, Hines-Dowell S, Nuccio R, Quinn E, Shurtleff SA, Rusch M, Patel A, Becksfort JB, Wang S, Weaver MS, Ding L, Mardis ER, Wilson RK, Gajjar A, Ellison DW, Pappo AS, Pui CH, Nichols KE, Downing JR (2015) Germline mutations in predisposition genes in pediatric cancer. N Engl J Med 373:2336–2346. https://​doi.​org/​10.​1056/​NEJMoa1508054CrossRefPubMedPubMedCentral
38.
go back to reference Huether R, Dong L, Chen X, Wu G, Parker M, Wei L, Ma J, Edmonson MN, Hedlund EK, Rusch MC, Shurtleff SA, Mulder HL, Boggs K, Vadordaria B, Cheng J, Yergeau D, Song G, Becksfort J, Lemmon G, Weber C, Cai Z, Dang J, Walsh M, Gedman AL, Faber Z, Easton J, Gruber T, Kriwacki RW, Partridge JF, Ding L, Wilson RK, Mardis ER, Mullighan CG, Gilbertson RJ, Baker SJ, Zambetti G, Ellison DW, Zhang J, Downing JR (2014) The landscape of somatic mutations in epigenetic regulators across 1,000 paediatric cancer genomes. Nat Commun 5:3630. https://doi.org/10.1038/ncomms4630CrossRefPubMed Huether R, Dong L, Chen X, Wu G, Parker M, Wei L, Ma J, Edmonson MN, Hedlund EK, Rusch MC, Shurtleff SA, Mulder HL, Boggs K, Vadordaria B, Cheng J, Yergeau D, Song G, Becksfort J, Lemmon G, Weber C, Cai Z, Dang J, Walsh M, Gedman AL, Faber Z, Easton J, Gruber T, Kriwacki RW, Partridge JF, Ding L, Wilson RK, Mardis ER, Mullighan CG, Gilbertson RJ, Baker SJ, Zambetti G, Ellison DW, Zhang J, Downing JR (2014) The landscape of somatic mutations in epigenetic regulators across 1,000 paediatric cancer genomes. Nat Commun 5:3630. https://​doi.​org/​10.​1038/​ncomms4630CrossRefPubMed
39.
go back to reference Northcott PA, Lee C, Zichner T, Stutz AM, Erkek S, Kawauchi D, Shih DJ, Hovestadt V, Zapatka M, Sturm D, Jones DT, Kool M, Remke M, Cavalli FM, Zuyderduyn S, Bader GD, VandenBerg S, Esparza LA, Ryzhova M, Wang W, Wittmann A, Stark S, Sieber L, Seker-Cin H, Linke L, Kratochwil F, Jager N, Buchhalter I, Imbusch CD, Zipprich G, Raeder B, Schmidt S, Diessl N, Wolf S, Wiemann S, Brors B, Lawerenz C, Eils J, Warnatz HJ, Risch T, Yaspo ML, Weber UD, Bartholomae CC, von Kalle C, Turanyi E, Hauser P, Sanden E, Darabi A, Siesjo P, Sterba J, Zitterbart K, Sumerauer D, van Sluis P, Versteeg R, Volckmann R, Koster J, Schuhmann MU, Ebinger M, Grimes HL, Robinson GW, Gajjar A, Mynarek M, von Hoff K, Rutkowski S, Pietsch T, Scheurlen W, Felsberg J, Reifenberger G, Kulozik AE, von Deimling A, Witt O, Eils R, Gilbertson RJ, Korshunov A, Taylor MD, Lichter P, Korbel JO, Wechsler-Reya RJ, Pfister SM (2014) Enhancer hijacking activates GFI1 family oncogenes in medulloblastoma. Nature 511:428–434. https://doi.org/10.1038/nature13379CrossRefPubMedPubMedCentral Northcott PA, Lee C, Zichner T, Stutz AM, Erkek S, Kawauchi D, Shih DJ, Hovestadt V, Zapatka M, Sturm D, Jones DT, Kool M, Remke M, Cavalli FM, Zuyderduyn S, Bader GD, VandenBerg S, Esparza LA, Ryzhova M, Wang W, Wittmann A, Stark S, Sieber L, Seker-Cin H, Linke L, Kratochwil F, Jager N, Buchhalter I, Imbusch CD, Zipprich G, Raeder B, Schmidt S, Diessl N, Wolf S, Wiemann S, Brors B, Lawerenz C, Eils J, Warnatz HJ, Risch T, Yaspo ML, Weber UD, Bartholomae CC, von Kalle C, Turanyi E, Hauser P, Sanden E, Darabi A, Siesjo P, Sterba J, Zitterbart K, Sumerauer D, van Sluis P, Versteeg R, Volckmann R, Koster J, Schuhmann MU, Ebinger M, Grimes HL, Robinson GW, Gajjar A, Mynarek M, von Hoff K, Rutkowski S, Pietsch T, Scheurlen W, Felsberg J, Reifenberger G, Kulozik AE, von Deimling A, Witt O, Eils R, Gilbertson RJ, Korshunov A, Taylor MD, Lichter P, Korbel JO, Wechsler-Reya RJ, Pfister SM (2014) Enhancer hijacking activates GFI1 family oncogenes in medulloblastoma. Nature 511:428–434. https://​doi.​org/​10.​1038/​nature13379CrossRefPubMedPubMedCentral
40.
go back to reference Peifer M, Hertwig F, Roels F, Dreidax D, Gartlgruber M, Menon R, Kramer A, Roncaioli JL, Sand F, Heuckmann JM, Ikram F, Schmidt R, Ackermann S, Engesser A, Kahlert Y, Vogel W, Altmuller J, Nurnberg P, Thierry-Mieg J, Thierry-Mieg D, Mariappan A, Heynck S, Mariotti E, Henrich KO, Gloeckner C, Bosco G, Leuschner I, Schweiger MR, Savelyeva L, Watkins SC, Shao C, Bell E, Hofer T, Achter V, Lang U, Theissen J, Volland R, Saadati M, Eggert A, de Wilde B, Berthold F, Peng Z, Zhao C, Shi L, Ortmann M, Buttner R, Perner S, Hero B, Schramm A, Schulte JH, Herrmann C, O'Sullivan RJ, Westermann F, Thomas RK, Fischer M (2015) Telomerase activation by genomic rearrangements in high-risk neuroblastoma. Nature 526:700–704. https://doi.org/10.1038/nature14980CrossRefPubMedPubMedCentral Peifer M, Hertwig F, Roels F, Dreidax D, Gartlgruber M, Menon R, Kramer A, Roncaioli JL, Sand F, Heuckmann JM, Ikram F, Schmidt R, Ackermann S, Engesser A, Kahlert Y, Vogel W, Altmuller J, Nurnberg P, Thierry-Mieg J, Thierry-Mieg D, Mariappan A, Heynck S, Mariotti E, Henrich KO, Gloeckner C, Bosco G, Leuschner I, Schweiger MR, Savelyeva L, Watkins SC, Shao C, Bell E, Hofer T, Achter V, Lang U, Theissen J, Volland R, Saadati M, Eggert A, de Wilde B, Berthold F, Peng Z, Zhao C, Shi L, Ortmann M, Buttner R, Perner S, Hero B, Schramm A, Schulte JH, Herrmann C, O'Sullivan RJ, Westermann F, Thomas RK, Fischer M (2015) Telomerase activation by genomic rearrangements in high-risk neuroblastoma. Nature 526:700–704. https://​doi.​org/​10.​1038/​nature14980CrossRefPubMedPubMedCentral
41.
go back to reference Bouffet E, Larouche V, Campbell BB, Merico D, de Borja R, Aronson M, Durno C, Krueger J, Cabric V, Ramaswamy V, Zhukova N, Mason G, Farah R, Afzal S, Yalon M, Rechavi G, Magimairajan V, Walsh MF, Constantini S, Dvir R, Elhasid R, Reddy A, Osborn M, Sullivan M, Hansford J, Dodgshun A, Klauber-Demore N, Peterson L, Patel S, Lindhorst S, Atkinson J, Cohen Z, Laframboise R, Dirks P, Taylor M, Malkin D, Albrecht S, Dudley RW, Jabado N, Hawkins CE, Shlien A, Tabori U (2016) Immune checkpoint inhibition for hypermutant glioblastoma multiforme resulting from germline Biallelic mismatch repair deficiency. J Clin Oncol 34:2206–2211. https://doi.org/10.1200/JCO.2016.66.6552CrossRefPubMed Bouffet E, Larouche V, Campbell BB, Merico D, de Borja R, Aronson M, Durno C, Krueger J, Cabric V, Ramaswamy V, Zhukova N, Mason G, Farah R, Afzal S, Yalon M, Rechavi G, Magimairajan V, Walsh MF, Constantini S, Dvir R, Elhasid R, Reddy A, Osborn M, Sullivan M, Hansford J, Dodgshun A, Klauber-Demore N, Peterson L, Patel S, Lindhorst S, Atkinson J, Cohen Z, Laframboise R, Dirks P, Taylor M, Malkin D, Albrecht S, Dudley RW, Jabado N, Hawkins CE, Shlien A, Tabori U (2016) Immune checkpoint inhibition for hypermutant glioblastoma multiforme resulting from germline Biallelic mismatch repair deficiency. J Clin Oncol 34:2206–2211. https://​doi.​org/​10.​1200/​JCO.​2016.​66.​6552CrossRefPubMed
43.
go back to reference Kwak EL, Bang YJ, Camidge DR, Shaw AT, Solomon B, Maki RG, Ou SH, Dezube BJ, Janne PA, Costa DB, Varella-Garcia M, Kim WH, Lynch TJ, Fidias P, Stubbs H, Engelman JA, Sequist LV, Tan W, Gandhi L, Mino-Kenudson M, Wei GC, Shreeve SM, Ratain MJ, Settleman J, Christensen JG, Haber DA, Wilner K, Salgia R, Shapiro GI, Clark JW, Iafrate AJ (2010) Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer. N Engl J Med 363:1693–1703. https://doi.org/10.1056/NEJMoa1006448CrossRefPubMedPubMedCentral Kwak EL, Bang YJ, Camidge DR, Shaw AT, Solomon B, Maki RG, Ou SH, Dezube BJ, Janne PA, Costa DB, Varella-Garcia M, Kim WH, Lynch TJ, Fidias P, Stubbs H, Engelman JA, Sequist LV, Tan W, Gandhi L, Mino-Kenudson M, Wei GC, Shreeve SM, Ratain MJ, Settleman J, Christensen JG, Haber DA, Wilner K, Salgia R, Shapiro GI, Clark JW, Iafrate AJ (2010) Anaplastic lymphoma kinase inhibition in non-small-cell lung cancer. N Engl J Med 363:1693–1703. https://​doi.​org/​10.​1056/​NEJMoa1006448CrossRefPubMedPubMedCentral
44.
45.
go back to reference Geoerger B, Schulte J, Zwaan CM, Casanova M, Fischer M, Moreno L, Trahair T, Jimenez I, Kang HJ, Pappo AS, Schafer E, Weiss BD, Healy ME, Li K, Lin T, Boral A, Pearson ADJ (2015) Phase I study of ceritinib in pediatric patients (Pts) with malignancies harboring a genetic alteration in ALK (ALK plus): safety, pharmacokinetic (PK), and efficacy results. J Clin Oncol 33:10005–10005. https://doi.org/10.1200/jco.2015.33.15_suppl.10005 Geoerger B, Schulte J, Zwaan CM, Casanova M, Fischer M, Moreno L, Trahair T, Jimenez I, Kang HJ, Pappo AS, Schafer E, Weiss BD, Healy ME, Li K, Lin T, Boral A, Pearson ADJ (2015) Phase I study of ceritinib in pediatric patients (Pts) with malignancies harboring a genetic alteration in ALK (ALK plus): safety, pharmacokinetic (PK), and efficacy results. J Clin Oncol 33:10005–10005. https://​doi.​org/​10.​1200/​jco.​2015.​33.​15_​suppl.​10005
47.
go back to reference Roberts KG, Li Y, Payne-Turner D, Harvey RC, Yang YL, Pei D, McCastlain K, Ding L, Lu C, Song G, Ma J, Becksfort J, Rusch M, Chen SC, Easton J, Cheng J, Boggs K, Santiago-Morales N, Iacobucci I, Fulton RS, Wen J, Valentine M, Cheng C, Paugh SW, Devidas M, Chen IM, Reshmi S, Smith A, Hedlund E, Gupta P, Nagahawatte P, Wu G, Chen X, Yergeau D, Vadodaria B, Mulder H, Winick NJ, Larsen EC, Carroll WL, Heerema NA, Carroll AJ, Grayson G, Tasian SK, Moore AS, Keller F, Frei-Jones M, Whitlock JA, Raetz EA, White DL, Hughes TP, Guidry Auvil JM, Smith MA, Marcucci G, Bloomfield CD, Mrozek K, Kohlschmidt J, Stock W, Kornblau SM, Konopleva M, Paietta E, Pui CH, Jeha S, Relling MV, Evans WE, Gerhard DS, Gastier-Foster JM, Mardis E, Wilson RK, Loh ML, Downing JR, Hunger SP, Willman CL, Zhang J, Mullighan CG (2014) Targetable kinase-activating lesions in Ph-like acute lymphoblastic leukemia. N Engl J Med 371:1005–1015. https://doi.org/10.1056/NEJMoa1403088CrossRefPubMedPubMedCentral Roberts KG, Li Y, Payne-Turner D, Harvey RC, Yang YL, Pei D, McCastlain K, Ding L, Lu C, Song G, Ma J, Becksfort J, Rusch M, Chen SC, Easton J, Cheng J, Boggs K, Santiago-Morales N, Iacobucci I, Fulton RS, Wen J, Valentine M, Cheng C, Paugh SW, Devidas M, Chen IM, Reshmi S, Smith A, Hedlund E, Gupta P, Nagahawatte P, Wu G, Chen X, Yergeau D, Vadodaria B, Mulder H, Winick NJ, Larsen EC, Carroll WL, Heerema NA, Carroll AJ, Grayson G, Tasian SK, Moore AS, Keller F, Frei-Jones M, Whitlock JA, Raetz EA, White DL, Hughes TP, Guidry Auvil JM, Smith MA, Marcucci G, Bloomfield CD, Mrozek K, Kohlschmidt J, Stock W, Kornblau SM, Konopleva M, Paietta E, Pui CH, Jeha S, Relling MV, Evans WE, Gerhard DS, Gastier-Foster JM, Mardis E, Wilson RK, Loh ML, Downing JR, Hunger SP, Willman CL, Zhang J, Mullighan CG (2014) Targetable kinase-activating lesions in Ph-like acute lymphoblastic leukemia. N Engl J Med 371:1005–1015. https://​doi.​org/​10.​1056/​NEJMoa1403088CrossRefPubMedPubMedCentral
54.
go back to reference Roberts AW, Davids MS, Pagel JM, Kahl BS, Puvvada SD, Gerecitano JF, Kipps TJ, Anderson MA, Brown JR, Gressick L, Wong S, Dunbar M, Zhu M, Desai MB, Cerri E, Heitner Enschede S, Humerickhouse RA, Wierda WG, Seymour JF (2016) Targeting BCL2 with venetoclax in relapsed chronic lymphocytic leukemia. N Engl J Med 374:311–322. https://doi.org/10.1056/NEJMoa1513257CrossRefPubMed Roberts AW, Davids MS, Pagel JM, Kahl BS, Puvvada SD, Gerecitano JF, Kipps TJ, Anderson MA, Brown JR, Gressick L, Wong S, Dunbar M, Zhu M, Desai MB, Cerri E, Heitner Enschede S, Humerickhouse RA, Wierda WG, Seymour JF (2016) Targeting BCL2 with venetoclax in relapsed chronic lymphocytic leukemia. N Engl J Med 374:311–322. https://​doi.​org/​10.​1056/​NEJMoa1513257CrossRefPubMed
56.
go back to reference Kool M, Jones DT, Jager N, Northcott PA, Pugh TJ, Hovestadt V, Piro RM, Esparza LA, Markant SL, Remke M, Milde T, Bourdeaut F, Ryzhova M, Sturm D, Pfaff E, Stark S, Hutter S, Seker-Cin H, Johann P, Bender S, Schmidt C, Rausch T, Shih D, Reimand J, Sieber L, Wittmann A, Linke L, Witt H, Weber UD, Zapatka M, Konig R, Beroukhim R, Bergthold G, van Sluis P, Volckmann R, Koster J, Versteeg R, Schmidt S, Wolf S, Lawerenz C, Bartholomae CC, von Kalle C, Unterberg A, Herold-Mende C, Hofer S, Kulozik AE, von Deimling A, Scheurlen W, Felsberg J, Reifenberger G, Hasselblatt M, Crawford JR, Grant GA, Jabado N, Perry A, Cowdrey C, Croul S, Zadeh G, Korbel JO, Doz F, Delattre O, Bader GD, McCabe MG, Collins VP, Kieran MW, Cho YJ, Pomeroy SL, Witt O, Brors B, Taylor MD, Schuller U, Korshunov A, Eils R, Wechsler-Reya RJ, Lichter P, Pfister SM (2014) Genome sequencing of SHH medulloblastoma predicts genotype-related response to smoothened inhibition. Cancer Cell 25:393–405. https://doi.org/10.1016/j.ccr.2014.02.004CrossRefPubMedPubMedCentral Kool M, Jones DT, Jager N, Northcott PA, Pugh TJ, Hovestadt V, Piro RM, Esparza LA, Markant SL, Remke M, Milde T, Bourdeaut F, Ryzhova M, Sturm D, Pfaff E, Stark S, Hutter S, Seker-Cin H, Johann P, Bender S, Schmidt C, Rausch T, Shih D, Reimand J, Sieber L, Wittmann A, Linke L, Witt H, Weber UD, Zapatka M, Konig R, Beroukhim R, Bergthold G, van Sluis P, Volckmann R, Koster J, Versteeg R, Schmidt S, Wolf S, Lawerenz C, Bartholomae CC, von Kalle C, Unterberg A, Herold-Mende C, Hofer S, Kulozik AE, von Deimling A, Scheurlen W, Felsberg J, Reifenberger G, Hasselblatt M, Crawford JR, Grant GA, Jabado N, Perry A, Cowdrey C, Croul S, Zadeh G, Korbel JO, Doz F, Delattre O, Bader GD, McCabe MG, Collins VP, Kieran MW, Cho YJ, Pomeroy SL, Witt O, Brors B, Taylor MD, Schuller U, Korshunov A, Eils R, Wechsler-Reya RJ, Lichter P, Pfister SM (2014) Genome sequencing of SHH medulloblastoma predicts genotype-related response to smoothened inhibition. Cancer Cell 25:393–405. https://​doi.​org/​10.​1016/​j.​ccr.​2014.​02.​004CrossRefPubMedPubMedCentral
58.
go back to reference Kovac M, Blattmann C, Ribi S, Smida J, Mueller NS, Engert F, Castro-Giner F, Weischenfeldt J, Kovacova M, Krieg A, Andreou D, Tunn PU, Durr HR, Rechl H, Schaser KD, Melcher I, Burdach S, Kulozik A, Specht K, Heinimann K, Fulda S, Bielack S, Jundt G, Tomlinson I, Korbel JO, Nathrath M, Baumhoer D (2015) Exome sequencing of osteosarcoma reveals mutation signatures reminiscent of BRCA deficiency. Nat Commun 6:8940. https://doi.org/10.1038/ncomms9940CrossRefPubMed Kovac M, Blattmann C, Ribi S, Smida J, Mueller NS, Engert F, Castro-Giner F, Weischenfeldt J, Kovacova M, Krieg A, Andreou D, Tunn PU, Durr HR, Rechl H, Schaser KD, Melcher I, Burdach S, Kulozik A, Specht K, Heinimann K, Fulda S, Bielack S, Jundt G, Tomlinson I, Korbel JO, Nathrath M, Baumhoer D (2015) Exome sequencing of osteosarcoma reveals mutation signatures reminiscent of BRCA deficiency. Nat Commun 6:8940. https://​doi.​org/​10.​1038/​ncomms9940CrossRefPubMed
59.
go back to reference Ma X, Edmonson M, Yergeau D, Muzny DM, Hampton OA, Rusch M, Song G, Easton J, Harvey RC, Wheeler DA, Ma J, Doddapaneni H, Vadodaria B, Wu G, Nagahawatte P, Carroll WL, Chen IM, Gastier-Foster JM, Relling MV, Smith MA, Devidas M, Guidry Auvil JM, Downing JR, Loh ML, Willman CL, Gerhard DS, Mullighan CG, Hunger SP, Zhang J (2015) Rise and fall of subclones from diagnosis to relapse in pediatric B-acute lymphoblastic leukaemia. Nat Commun 6:6604. https://doi.org/10.1038/ncomms7604CrossRefPubMed Ma X, Edmonson M, Yergeau D, Muzny DM, Hampton OA, Rusch M, Song G, Easton J, Harvey RC, Wheeler DA, Ma J, Doddapaneni H, Vadodaria B, Wu G, Nagahawatte P, Carroll WL, Chen IM, Gastier-Foster JM, Relling MV, Smith MA, Devidas M, Guidry Auvil JM, Downing JR, Loh ML, Willman CL, Gerhard DS, Mullighan CG, Hunger SP, Zhang J (2015) Rise and fall of subclones from diagnosis to relapse in pediatric B-acute lymphoblastic leukaemia. Nat Commun 6:6604. https://​doi.​org/​10.​1038/​ncomms7604CrossRefPubMed
60.
go back to reference Eleveld TF, Oldridge DA, Bernard V, Koster J, Daage LC, Diskin SJ, Schild L, Bentahar NB, Bellini A, Chicard M, Lapouble E, Combaret V, Legoix-Ne P, Michon J, Pugh TJ, Hart LS, Rader J, Attiyeh EF, Wei JS, Zhang S, Naranjo A, Gastier-Foster JM, Hogarty MD, Asgharzadeh S, Smith MA, Guidry Auvil JM, Watkins TB, Zwijnenburg DA, Ebus ME, van Sluis P, Hakkert A, van Wezel E, van der Schoot CE, Westerhout EM, Schulte JH, Tytgat GA, Dolman ME, Janoueix-Lerosey I, Gerhard DS, Caron HN, Delattre O, Khan J, Versteeg R, Schleiermacher G, Molenaar JJ, Maris JM (2015) Relapsed neuroblastomas show frequent RAS-MAPK pathway mutations. Nat Genet 47:864–871. https://doi.org/10.1038/ng.3333CrossRefPubMedPubMedCentral Eleveld TF, Oldridge DA, Bernard V, Koster J, Daage LC, Diskin SJ, Schild L, Bentahar NB, Bellini A, Chicard M, Lapouble E, Combaret V, Legoix-Ne P, Michon J, Pugh TJ, Hart LS, Rader J, Attiyeh EF, Wei JS, Zhang S, Naranjo A, Gastier-Foster JM, Hogarty MD, Asgharzadeh S, Smith MA, Guidry Auvil JM, Watkins TB, Zwijnenburg DA, Ebus ME, van Sluis P, Hakkert A, van Wezel E, van der Schoot CE, Westerhout EM, Schulte JH, Tytgat GA, Dolman ME, Janoueix-Lerosey I, Gerhard DS, Caron HN, Delattre O, Khan J, Versteeg R, Schleiermacher G, Molenaar JJ, Maris JM (2015) Relapsed neuroblastomas show frequent RAS-MAPK pathway mutations. Nat Genet 47:864–871. https://​doi.​org/​10.​1038/​ng.​3333CrossRefPubMedPubMedCentral
63.
go back to reference Coley WB (1991) The treatment of malignant tumors by repeated inoculations of erysipelas. With a report of ten original cases. Clin Orthop Relat Res 1893:3–11 Coley WB (1991) The treatment of malignant tumors by repeated inoculations of erysipelas. With a report of ten original cases. Clin Orthop Relat Res 1893:3–11
64.
go back to reference Mathe G, Schwarzenberg L, Amiel JL, Schneider M, Cattan A, Schlumberger JR (1967) Immunological approaches to the treatment of human cancers. Bull Cancer 54:33–54PubMed Mathe G, Schwarzenberg L, Amiel JL, Schneider M, Cattan A, Schlumberger JR (1967) Immunological approaches to the treatment of human cancers. Bull Cancer 54:33–54PubMed
65.
go back to reference Kohler G, Milstein C (1975) Continuous cultures of fused cells secreting antibody of predefined specificity. Nature 256:495–497CrossRefPubMed Kohler G, Milstein C (1975) Continuous cultures of fused cells secreting antibody of predefined specificity. Nature 256:495–497CrossRefPubMed
66.
go back to reference Rosenberg SA (1984) Immunotherapy of cancer by systemic administration of lymphoid cells plus interleukin-2. J Biol Response Mod 3:501–511PubMed Rosenberg SA (1984) Immunotherapy of cancer by systemic administration of lymphoid cells plus interleukin-2. J Biol Response Mod 3:501–511PubMed
67.
go back to reference Shiloni E, Lafreniere R, Mule JJ, Schwarz SL, Rosenberg SA (1986) Effect of immunotherapy with allogeneic lymphokine-activated killer cells and recombinant interleukin 2 on established pulmonary and hepatic metastases in mice. Cancer Res 46:5633–5640PubMed Shiloni E, Lafreniere R, Mule JJ, Schwarz SL, Rosenberg SA (1986) Effect of immunotherapy with allogeneic lymphokine-activated killer cells and recombinant interleukin 2 on established pulmonary and hepatic metastases in mice. Cancer Res 46:5633–5640PubMed
68.
go back to reference Kolb HJ, Mittermuller J, Clemm C, Holler E, Ledderose G, Brehm G, Heim M, Wilmanns W (1990) Donor leukocyte transfusions for treatment of recurrent chronic myelogenous leukemia in marrow transplant patients. Blood 76:2462–2465CrossRefPubMed Kolb HJ, Mittermuller J, Clemm C, Holler E, Ledderose G, Brehm G, Heim M, Wilmanns W (1990) Donor leukocyte transfusions for treatment of recurrent chronic myelogenous leukemia in marrow transplant patients. Blood 76:2462–2465CrossRefPubMed
69.
go back to reference Eshhar Z, Waks T, Gross G, Schindler DG (1993) Specific activation and targeting of cytotoxic lymphocytes through chimeric single chains consisting of antibody-binding domains and the gamma or zeta subunits of the immunoglobulin and T-cell receptors. Proc Natl Acad Sci U S A 90:720–724CrossRefPubMedPubMedCentral Eshhar Z, Waks T, Gross G, Schindler DG (1993) Specific activation and targeting of cytotoxic lymphocytes through chimeric single chains consisting of antibody-binding domains and the gamma or zeta subunits of the immunoglobulin and T-cell receptors. Proc Natl Acad Sci U S A 90:720–724CrossRefPubMedPubMedCentral
70.
go back to reference Leach DR, Krummel MF, Allison JP (1996) Enhancement of antitumor immunity by CTLA-4 blockade. Science 271:1734–1736CrossRefPubMed Leach DR, Krummel MF, Allison JP (1996) Enhancement of antitumor immunity by CTLA-4 blockade. Science 271:1734–1736CrossRefPubMed
71.
go back to reference Rossig C, Bollard CM, Nuchtern JG, Merchant DA, Brenner MK (2001) Targeting of G(D2)-positive tumor cells by human T lymphocytes engineered to express chimeric T-cell receptor genes. Int J Cancer 94:228–236CrossRefPubMed Rossig C, Bollard CM, Nuchtern JG, Merchant DA, Brenner MK (2001) Targeting of G(D2)-positive tumor cells by human T lymphocytes engineered to express chimeric T-cell receptor genes. Int J Cancer 94:228–236CrossRefPubMed
75.
go back to reference Pollack IF, Jakacki RI, Butterfield LH, Hamilton RL, Panigrahy A, Normolle DP, Connelly AK, Dibridge S, Mason G, Whiteside TL, Okada H (2016) Antigen-specific immunoreactivity and clinical outcome following vaccination with glioma-associated antigen peptides in children with recurrent high-grade gliomas: results of a pilot study. J Neuro-Oncol 130:517–527. https://doi.org/10.1007/s11060-016-2245-3CrossRef Pollack IF, Jakacki RI, Butterfield LH, Hamilton RL, Panigrahy A, Normolle DP, Connelly AK, Dibridge S, Mason G, Whiteside TL, Okada H (2016) Antigen-specific immunoreactivity and clinical outcome following vaccination with glioma-associated antigen peptides in children with recurrent high-grade gliomas: results of a pilot study. J Neuro-Oncol 130:517–527. https://​doi.​org/​10.​1007/​s11060-016-2245-3CrossRef
76.
79.
go back to reference Sawada A, Inoue M, Kondo O, Yamada-Nakata K, Ishihara T, Kuwae Y, Nishikawa M, Ammori Y, Tsuboi A, Oji Y, Koyama-Sato M, Oka Y, Yasui M, Sugiyama H, Kawa K (2016) Feasibility of cancer immunotherapy with WT1 peptide vaccination for solid and hematological malignancies in children. Pediatr Blood Cancer 63:234–241. https://doi.org/10.1002/pbc.25792CrossRefPubMed Sawada A, Inoue M, Kondo O, Yamada-Nakata K, Ishihara T, Kuwae Y, Nishikawa M, Ammori Y, Tsuboi A, Oji Y, Koyama-Sato M, Oka Y, Yasui M, Sugiyama H, Kawa K (2016) Feasibility of cancer immunotherapy with WT1 peptide vaccination for solid and hematological malignancies in children. Pediatr Blood Cancer 63:234–241. https://​doi.​org/​10.​1002/​pbc.​25792CrossRefPubMed
81.
82.
go back to reference Trappe R, Oertel S, Leblond V, Mollee P, Sender M, Reinke P, Neuhaus R, Lehmkuhl H, Horst HA, Salles G, Morschhauser F, Jaccard A, Lamy T, Leithauser M, Zimmermann H, Anagnostopoulos I, Raphael M, Riess H, Choquet S, German PSG, European PN (2012) Sequential treatment with rituximab followed by CHOP chemotherapy in adult B-cell post-transplant lymphoproliferative disorder (PTLD): the prospective international multicentre phase 2 PTLD-1 trial. Lancet Oncol 13:196–206. https://doi.org/10.1016/S1470-2045(11)70300-XCrossRefPubMed Trappe R, Oertel S, Leblond V, Mollee P, Sender M, Reinke P, Neuhaus R, Lehmkuhl H, Horst HA, Salles G, Morschhauser F, Jaccard A, Lamy T, Leithauser M, Zimmermann H, Anagnostopoulos I, Raphael M, Riess H, Choquet S, German PSG, European PN (2012) Sequential treatment with rituximab followed by CHOP chemotherapy in adult B-cell post-transplant lymphoproliferative disorder (PTLD): the prospective international multicentre phase 2 PTLD-1 trial. Lancet Oncol 13:196–206. https://​doi.​org/​10.​1016/​S1470-2045(11)70300-XCrossRefPubMed
84.
go back to reference Ladenstein R, Poetschger U, Gray J, Valteau-Couanet D, Luksch R, Castel V, Yaniv I, Laureys G, Elliott M, Michon JM, Owens C, Trahair T, Chan GCF, Ruud E, Schroeder H, Popovic MB, Glogova E, Schreier G, Loibner H and Lode HN (2016) Toxicity and outcome of anti-GD(2) antibody ch14.18/CHO in front-line, high-risk patients with neuroblastoma: final results of the phase III immunotherapy randomisation (HR-NBL1/SIOPEN trial). J Clin Oncol 34. doi: https://doi.org/10.1200/JCO.2016.34.15_suppl.10500 Ladenstein R, Poetschger U, Gray J, Valteau-Couanet D, Luksch R, Castel V, Yaniv I, Laureys G, Elliott M, Michon JM, Owens C, Trahair T, Chan GCF, Ruud E, Schroeder H, Popovic MB, Glogova E, Schreier G, Loibner H and Lode HN (2016) Toxicity and outcome of anti-GD(2) antibody ch14.18/CHO in front-line, high-risk patients with neuroblastoma: final results of the phase III immunotherapy randomisation (HR-NBL1/SIOPEN trial). J Clin Oncol 34. doi: https://​doi.​org/​10.​1200/​JCO.​2016.​34.​15_​suppl.​10500
85.
go back to reference Topp MS, Gokbuget N, Stein AS, Zugmaier G, O'Brien S, Bargou RC, Dombret H, Fielding AK, Heffner L, Larson RA, Neumann S, Foa R, Litzow M, Ribera JM, Rambaldi A, Schiller G, Bruggemann M, Horst HA, Holland C, Jia C, Maniar T, Huber B, Nagorsen D, Forman SJ, Kantarjian HM (2015) Safety and activity of blinatumomab for adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia: a multicentre, single-arm, phase 2 study. Lancet Oncol 16:57–66. https://doi.org/10.1016/S1470-2045(14)71170-2CrossRefPubMed Topp MS, Gokbuget N, Stein AS, Zugmaier G, O'Brien S, Bargou RC, Dombret H, Fielding AK, Heffner L, Larson RA, Neumann S, Foa R, Litzow M, Ribera JM, Rambaldi A, Schiller G, Bruggemann M, Horst HA, Holland C, Jia C, Maniar T, Huber B, Nagorsen D, Forman SJ, Kantarjian HM (2015) Safety and activity of blinatumomab for adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia: a multicentre, single-arm, phase 2 study. Lancet Oncol 16:57–66. https://​doi.​org/​10.​1016/​S1470-2045(14)71170-2CrossRefPubMed
86.
go back to reference Rettinger E, Willasch AM, Kreyenberg H, Borkhardt A, Holter W, Kremens B, Strahm B, Woessmann W, Mauz-Koerholz C, Gruhn B, Burdach S, Albert MH, Schlegel PG, Klingebiel T, Bader P (2011) Preemptive immunotherapy in childhood acute myeloid leukemia for patients showing evidence of mixed chimerism after allogeneic stem cell transplantation. Blood 118:5681–5688. https://doi.org/10.1182/blood-2011-04-348805CrossRefPubMed Rettinger E, Willasch AM, Kreyenberg H, Borkhardt A, Holter W, Kremens B, Strahm B, Woessmann W, Mauz-Koerholz C, Gruhn B, Burdach S, Albert MH, Schlegel PG, Klingebiel T, Bader P (2011) Preemptive immunotherapy in childhood acute myeloid leukemia for patients showing evidence of mixed chimerism after allogeneic stem cell transplantation. Blood 118:5681–5688. https://​doi.​org/​10.​1182/​blood-2011-04-348805CrossRefPubMed
93.
go back to reference Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA (2018) Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med 378:439–448. https://doi.org/10.1056/NEJMoa1709866CrossRefPubMedPubMedCentral Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, Bader P, Verneris MR, Stefanski HE, Myers GD, Qayed M, De Moerloose B, Hiramatsu H, Schlis K, Davis KL, Martin PL, Nemecek ER, Yanik GA, Peters C, Baruchel A, Boissel N, Mechinaud F, Balduzzi A, Krueger J, June CH, Levine BL, Wood P, Taran T, Leung M, Mueller KT, Zhang Y, Sen K, Lebwohl D, Pulsipher MA, Grupp SA (2018) Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med 378:439–448. https://​doi.​org/​10.​1056/​NEJMoa1709866CrossRefPubMedPubMedCentral
95.
go back to reference Burdach S (2016) Coping with T-cell promiscuity in genomics-based immunotherapy - allorestricted T-cell receptor transgenic T-cells (ATRs) vs. chimeric antigen receptor T-cells (CARs). Oncol Res Treat 39:30–30 Burdach S (2016) Coping with T-cell promiscuity in genomics-based immunotherapy - allorestricted T-cell receptor transgenic T-cells (ATRs) vs. chimeric antigen receptor T-cells (CARs). Oncol Res Treat 39:30–30
102.
go back to reference Horvat TZ, Adel NG, Dang TO, Momtaz P, Postow MA, Callahan MK, Carvajal RD, Dickson MA, D'Angelo SP, Woo KM, Panageas KS, Wolchok JD, Chapman PB (2015) Immune-related adverse events, need for systemic immunosuppression, and effects on survival and time to treatment failure in patients with melanoma treated with ipilimumab at Memorial Sloan Kettering Cancer Center. J Clin Oncol 33:3193–3198. https://doi.org/10.1200/JCO.2015.60.8448CrossRefPubMedPubMedCentral Horvat TZ, Adel NG, Dang TO, Momtaz P, Postow MA, Callahan MK, Carvajal RD, Dickson MA, D'Angelo SP, Woo KM, Panageas KS, Wolchok JD, Chapman PB (2015) Immune-related adverse events, need for systemic immunosuppression, and effects on survival and time to treatment failure in patients with melanoma treated with ipilimumab at Memorial Sloan Kettering Cancer Center. J Clin Oncol 33:3193–3198. https://​doi.​org/​10.​1200/​JCO.​2015.​60.​8448CrossRefPubMedPubMedCentral
105.
go back to reference Silverman E (2018) Kymriah: a sign of more difficult decisions to come. Manag Care 27:17PubMed Silverman E (2018) Kymriah: a sign of more difficult decisions to come. Manag Care 27:17PubMed
Metadata
Title
Precision medicine in pediatric oncology
Authors
Stefan E. G. Burdach
Mike-Andrew Westhoff
Maximilian Felix Steinhauser
Klaus-Michael Debatin
Publication date
01-12-2018
Publisher
Springer International Publishing
Published in
Molecular and Cellular Pediatrics / Issue 1/2018
Electronic ISSN: 2194-7791
DOI
https://doi.org/10.1186/s40348-018-0084-3

Other articles of this Issue 1/2018

Molecular and Cellular Pediatrics 1/2018 Go to the issue