Journal Article (Review Article)

DKFZ-2025-01218

http://join2-wiki.gsi.de/foswiki/pub/Main/Artwork/join2_logo100x88.png Accelerating Drug Development for Neuroblastoma: Consensus Statement From the Third Neuroblastoma Drug Development Strategy Forum.

DuBois, S. G. ; Moreno, L. ; Anderson, J. ; Asgharzadeh, S. ; Bagatell, R. ; Beck-Popovic, M. ; Belle, J. ; Berlanga, P. ; Bird, N. J. ; Chesler, L. ; Durbin, A. ; Eggert, A. ; Eilers, M. ; Federico, S. M. ; Fischer, M. ; Gatz, S. A. ; George, R. E. ; George, S. ; Goldsmith, K. C. ; Gray, J. ; Heczey, A. ; Irwin, M. S. ; Knox, L. ; Lode, H. N. ; Ludwinski, D. ; Macy, M. E. ; Majzner, R. G. ; Maris, J. M. ; Modak, S. ; Molenaar, J. J. ; Morgenstern, D. A. ; Mossé, Y. P. ; Owens, C. ; Reynolds, C. P. ; Rossig, C. ; Schleiermacher, G. ; Scott, L. ; Sondel, P. M. ; Speleman, F. ; van Noesel, M. ; Westermann, F.DKFZ* ; Wienke, J. ; Wolpaw, A. J. ; Park, J. R. ; Pearson, A. D. J.

2025
Wiley New York, NY

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Please use a persistent id in citations: doi:10.1002/pbc.31831

Abstract: High-risk neuroblastoma is a poor prognosis cancer of the sympathetic nervous system that accounts for a disproportionate number of childhood cancer deaths. Many viable biological targets have been identified, and the number of potential combinations is even larger. Several products have attained marketing authorization for treatment of patients with neuroblastoma. Patient outcomes remain poor, with approximately 50% of children with newly diagnosed high-risk neuroblastoma cured of their disease. International, multistakeholder Neuroblastoma Drug Development Strategy (NDDS) meetings were established more than a decade ago. This third NDDS meeting included academia, industry, regulatory, and patient advocacy representatives to prioritize agents and to address key challenges in drug development in this disease. Given the central role that anti-GD2 therapy plays, novel GD2-directed combinations were a key focus, including epigenetic enzymes such as EZH2 and immunologic targets such as IL15 and TIGIT as potential combination partners. GD2-directed chimeric antigen receptor (CAR)-T cells were a top priority, along with emerging CAR-T targets such as B7-H3 and GPC2. Recognizing that combination therapies are likely to be most impactful for patients and for advancing therapies to frontline, another key focus was on high priority combinations of targeted therapies, including Aurora A kinase plus BCL2 or ATR inhibitors. Additional targets and agents were prioritized or deprioritized based upon current data. Access to drugs for clinical trials was viewed as a major barrier to progress. Strategies to overcome this challenge focused on united efforts by the international scientific and advocacy community and early engagement by industry with regulatory authorities.

Keyword(s): ALK ; ATR ; B7‐H3 ; EZH2 ; GD2 ; GPC2 ; MYCN ; clinical trials ; drug development ; neuroblastoma ; relapse

Note: 2025 Sep;72(9):e31831

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Contributing Institute(s):

1. B087 Neuroblastom Genomik (B087)
2. DKTK HD zentral (HD01)

Research Program(s):

1. 312 - Funktionelle und strukturelle Genomforschung (POF4-312) (POF4-312)

Appears in the scientific report 2025

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Database coverage:
; BIOSIS Previews ; Biological Abstracts ; Clarivate Analytics Master Journal List ; Current Contents - Clinical Medicine ; DEAL Wiley ; Essential Science Indicators ; IF < 5 ; JCR ; SCOPUS ; Science Citation Index Expanded ; Web of Science Core Collection

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