Accelerating drug development for neuroblastoma: Summary of the
Second Neuroblastoma Drug Development Strategy forum from
Innovative Therapies for Children with Cancer and International
Society of Paediatric Oncology Europe Neuroblastoma

J 2020

Accelerating drug development for neuroblastoma: Summary of the Second Neuroblastoma Drug Development Strategy forum from Innovative Therapies for Children with Cancer and International Society of Paediatric Oncology Europe Neuroblastoma

MORENO, L., G. BARONE, S. G. DUBOIS, J. MOLENAAR, M. FISCHER et. al.

Basic information

Original name

Accelerating drug development for neuroblastoma: Summary of the Second Neuroblastoma Drug Development Strategy forum from Innovative Therapies for Children with Cancer and International Society of Paediatric Oncology Europe Neuroblastoma

Authors

MORENO, L. (724 Spain, guarantor), G. BARONE (826 United Kingdom of Great Britain and Northern Ireland), S. G. DUBOIS (840 United States of America), J. MOLENAAR (528 Netherlands), M. FISCHER (528 Netherlands), J. SCHULTE (276 Germany), A. EGGERT (276 Germany), G. SCHLEIERMACHER (276 Germany), F. SPELEMAN (250 France), L. CHESLER (56 Belgium), B. GEOERGER (826 United Kingdom of Great Britain and Northern Ireland), M. D. HOGARTY (250 France), M. S. IRWIN (840 United States of America), N. BIRD (826 United Kingdom of Great Britain and Northern Ireland), G. B. BLANCHARD (826 United Kingdom of Great Britain and Northern Ireland), S. BUCKLAND (826 United Kingdom of Great Britain and Northern Ireland), H. CARON (756 Switzerland), S. DAVIS (826 United Kingdom of Great Britain and Northern Ireland), B. DE WILDE (528 Netherlands), H. E. DEUBZER (528 Netherlands), E. DOLMAN (528 Netherlands), M. EILERS (276 Germany), R. E. GEORGE (840 United States of America), S. GEORGE (840 United States of America), Jaroslav ŠTĚRBA (203 Czech Republic, belonging to the institution), J. M. MARIS (840 United States of America), L. MARSHALL (840 United States of America), M. MERCHANT (804 Ukraine), P. MORTIMER (826 United Kingdom of Great Britain and Northern Ireland), C. OWENS (372 Ireland), A. PHILPOTT (826 United Kingdom of Great Britain and Northern Ireland), E. POON (826 United Kingdom of Great Britain and Northern Ireland), J. W. SHAY (840 United States of America), R. TONELLI (380 Italy), D. VALTEAU-COUANET (250 France), G. VASSAL (250 France), J. R. PARK (840 United States of America) and A. D. J. PEARSON (826 United Kingdom of Great Britain and Northern Ireland)

Edition

European Journal of Cancer, Oxford, Elsevier Science Inc. 2020, 0959-8049

Other information

Language

English

Type of outcome

Článek v odborném periodiku

Field of Study

30204 Oncology

Country of publisher

United Kingdom of Great Britain and Northern Ireland

Confidentiality degree

není předmětem státního či obchodního tajemství

References:

URL

Impact factor

Impact factor: 9.162

RIV identification code

RIV/00216224:14110/20:00116258

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1016/j.ejca.2020.05.010

UT WoS

000557411500009

Keywords in English

Neuroblastoma; Drug development; Phase I; Preclinical testing; Clinical trials; MYCN; Epigenetics

Abstract

V originále

Only one class of targeted agents (anti-GD2 antibodies) has been incorporated into front-line therapy for neuroblastoma since the 1980s. The Neuroblastoma New Drug Development Strategy (NDDS) initiative commenced in 2012 to accelerate the development of new drugs for neuroblastoma. Advances have occurred, with eight of nine high-priority targets being evaluated in paediatric trials including anaplastic lymphoma kinase inhibitors being investigated in front-line, but significant challenges remain. This article reports the conclusions of the second NDDS forum, which expanded across the Atlantic to further develop the initiative. Pre-clinical and clinical data for 40 genetic targets and mechanisms of action were prioritised and drugs were identified for early-phase trials. Strategies to develop drugs targeting TERT, telomere maintenance, ATRX, alternative lengthening of telomeres (ALT), BRIP1 and RRM2 as well as direct targeting of MYCN are high priority and should be championed for drug discovery. Promising pre-clinical data suggest that targeting of ALT by ATM or PARP inhibition may be potential strategies. Drugs targeting CDK2/9, CDK7, ATR and telomere maintenance should enter paediatric clinical development rapidly. Optimising the response to anti-GD2 by combinations with chemotherapy, targeted agents and other immunological targets are crucial. Delivering this strategy in the face of small patient cohorts, genomically defined subpopulations and a large number of permutations of combination trials, demands even greater international collaboration. In conclusion, the NDDS provides an internationally agreed, biologically driven selection of prioritised genetic targets and drugs. Improvements in the strategy for conducting trials in neuroblastoma will accelerate bringing these new drugs more rapidly to front-line therapy. (C) 2020 Elsevier Ltd. All rights reserved.

Citovat

MORENO, L., G. BARONE, S. G. DUBOIS, J. MOLENAAR, M. FISCHER, J. SCHULTE, A. EGGERT, G. SCHLEIERMACHER, F. SPELEMAN, L. CHESLER, B. GEOERGER, M. D. HOGARTY, M. S. IRWIN, N. BIRD, G. B. BLANCHARD, S. BUCKLAND, H. CARON, S. DAVIS, B. DE WILDE, H. E. DEUBZER, E. DOLMAN, M. EILERS, R. E. GEORGE, S. GEORGE, Jaroslav ŠTĚRBA, J. M. MARIS, L. MARSHALL, M. MERCHANT, P. MORTIMER, C. OWENS, A. PHILPOTT, E. POON, J. W. SHAY, R. TONELLI, D. VALTEAU-COUANET, G. VASSAL, J. R. PARK and A. D. J. PEARSON. Accelerating drug development for neuroblastoma: Summary of the Second Neuroblastoma Drug Development Strategy forum from Innovative Therapies for Children with Cancer and International Society of Paediatric Oncology Europe Neuroblastoma. European Journal of Cancer. Oxford: Elsevier Science Inc., 2020, vol. 136, SEP 2020, p. 52-68. ISSN 0959-8049. Available from: https://dx.doi.org/10.1016/j.ejca.2020.05.010.

@article{1674867,
   author = {Moreno, L. and Barone, G. and DuBois, S. G. and Molenaar, J. and Fischer, M. and Schulte, J. and Eggert, A. and Schleiermacher, G. and Speleman, F. and Chesler, L. and Geoerger, B. and Hogarty, M. D. and Irwin, M. S. and Bird, N. and Blanchard, G. B. and Buckland, S. and Caron, H. and Davis, S. and De Wilde, B. and Deubzer, H. E. and Dolman, E. and Eilers, M. and George, R. E. and George, S. and Štěrba, Jaroslav and Maris, J. M. and Marshall, L. and Merchant, M. and Mortimer, P. and Owens, C. and Philpott, A. and Poon, E. and Shay, J. W. and Tonelli, R. and ValteauandCouanet, D. and Vassal, G. and Park, J. R. and Pearson, A. D. J.},
   article_location = {Oxford},
   article_number = {SEP 2020},
   doi = {http://dx.doi.org/10.1016/j.ejca.2020.05.010},
   keywords = {Neuroblastoma; Drug development; Phase I; Preclinical testing; Clinical trials; MYCN; Epigenetics},
   language = {eng},
   issn = {0959-8049},
   journal = {European Journal of Cancer},
   title = {Accelerating drug development for neuroblastoma: Summary of the Second Neuroblastoma Drug Development Strategy forum from Innovative Therapies for Children with Cancer and International Society of Paediatric Oncology Europe Neuroblastoma},
   url = {https://www.sciencedirect.com/science/article/pii/S0959804920302768?via%3Dihub},
   volume = {136},
   year = {2020}
}

TY  - JOUR
ID  - 1674867
AU  - Moreno, L. - Barone, G. - DuBois, S. G. - Molenaar, J. - Fischer, M. - Schulte, J. - Eggert, A. - Schleiermacher, G. - Speleman, F. - Chesler, L. - Geoerger, B. - Hogarty, M. D. - Irwin, M. S. - Bird, N. - Blanchard, G. B. - Buckland, S. - Caron, H. - Davis, S. - De Wilde, B. - Deubzer, H. E. - Dolman, E. - Eilers, M. - George, R. E. - George, S. - Štěrba, Jaroslav - Maris, J. M. - Marshall, L. - Merchant, M. - Mortimer, P. - Owens, C. - Philpott, A. - Poon, E. - Shay, J. W. - Tonelli, R. - Valteau-Couanet, D. - Vassal, G. - Park, J. R. - Pearson, A. D. J.
PY  - 2020
TI  - Accelerating drug development for neuroblastoma: Summary of the Second Neuroblastoma Drug Development Strategy forum from Innovative Therapies for Children with Cancer and International Society of Paediatric Oncology Europe Neuroblastoma
JF  - European Journal of Cancer
VL  - 136
IS  - SEP 2020
SP  - 52-68
EP  - 52-68
PB  - Elsevier Science Inc.
SN  - 09598049
KW  - Neuroblastoma
KW  - Drug development
KW  - Phase I
KW  - Preclinical testing
KW  - Clinical trials
KW  - MYCN
KW  - Epigenetics
UR  - https://www.sciencedirect.com/science/article/pii/S0959804920302768?via%3Dihub
L2  - https://www.sciencedirect.com/science/article/pii/S0959804920302768?via%3Dihub
N2  - Only one class of targeted agents (anti-GD2 antibodies) has been incorporated into front-line therapy for neuroblastoma since the 1980s. The Neuroblastoma New Drug Development Strategy (NDDS) initiative commenced in 2012 to accelerate the development of new drugs for neuroblastoma. Advances have occurred, with eight of nine high-priority targets being evaluated in paediatric trials including anaplastic lymphoma kinase inhibitors being investigated in front-line, but significant challenges remain. This article reports the conclusions of the second NDDS forum, which expanded across the Atlantic to further develop the initiative. Pre-clinical and clinical data for 40 genetic targets and mechanisms of action were prioritised and drugs were identified for early-phase trials. Strategies to develop drugs targeting TERT, telomere maintenance, ATRX, alternative lengthening of telomeres (ALT), BRIP1 and RRM2 as well as direct targeting of MYCN are high priority and should be championed for drug discovery. Promising pre-clinical data suggest that targeting of ALT by ATM or PARP inhibition may be potential strategies. Drugs targeting CDK2/9, CDK7, ATR and telomere maintenance should enter paediatric clinical development rapidly. Optimising the response to anti-GD2 by combinations with chemotherapy, targeted agents and other immunological targets are crucial. Delivering this strategy in the face of small patient cohorts, genomically defined subpopulations and a large number of permutations of combination trials, demands even greater international collaboration. In conclusion, the NDDS provides an internationally agreed, biologically driven selection of prioritised genetic targets and drugs. Improvements in the strategy for conducting trials in neuroblastoma will accelerate bringing these new drugs more rapidly to front-line therapy. (C) 2020 Elsevier Ltd. All rights reserved.
ER  -

MORENO, L., G. BARONE, S. G. DUBOIS, J. MOLENAAR, M. FISCHER, J. SCHULTE, A. EGGERT, G. SCHLEIERMACHER, F. SPELEMAN, L. CHESLER, B. GEOERGER, M. D. HOGARTY, M. S. IRWIN, N. BIRD, G. B. BLANCHARD, S. BUCKLAND, H. CARON, S. DAVIS, B. DE WILDE, H. E. DEUBZER, E. DOLMAN, M. EILERS, R. E. GEORGE, S. GEORGE, Jaroslav ŠTĚRBA, J. M. MARIS, L. MARSHALL, M. MERCHANT, P. MORTIMER, C. OWENS, A. PHILPOTT, E. POON, J. W. SHAY, R. TONELLI, D. VALTEAU-COUANET, G. VASSAL, J. R. PARK and A. D. J. PEARSON. Accelerating drug development for neuroblastoma: Summary of the Second Neuroblastoma Drug Development Strategy forum from Innovative Therapies for Children with Cancer and International Society of Paediatric Oncology Europe Neuroblastoma. \textit{European Journal of Cancer}. Oxford: Elsevier Science Inc., 2020, vol.~136, SEP 2020, p.~52-68. ISSN~0959-8049. Available from: https://dx.doi.org/10.1016/j.ejca.2020.05.010.

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