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@ARTICLE{DuBois:302020,
author = {S. G. DuBois and L. Moreno and J. Anderson and S.
Asgharzadeh and R. Bagatell and M. Beck-Popovic and J. Belle
and P. Berlanga and N. J. Bird and L. Chesler and A. Durbin
and A. Eggert and M. Eilers and S. M. Federico and M.
Fischer and S. A. Gatz and R. E. George and S. George and K.
C. Goldsmith and J. Gray and A. Heczey and M. S. Irwin and
L. Knox and H. N. Lode and D. Ludwinski and M. E. Macy and
R. G. Majzner and J. M. Maris and S. Modak and J. J.
Molenaar and D. A. Morgenstern and Y. P. Mossé and C. Owens
and C. P. Reynolds and C. Rossig and G. Schleiermacher and
L. Scott and P. M. Sondel and F. Speleman and M. van Noesel
and F. Westermann$^*$ and J. Wienke and A. J. Wolpaw and J.
R. Park and A. D. J. Pearson},
title = {{A}ccelerating {D}rug {D}evelopment for {N}euroblastoma:
{C}onsensus {S}tatement {F}rom the {T}hird {N}euroblastoma
{D}rug {D}evelopment {S}trategy {F}orum.},
journal = {Pediatric blood $\&$ cancer},
volume = {72},
number = {9},
issn = {1545-5009},
address = {New York, NY},
publisher = {Wiley},
reportid = {DKFZ-2025-01218},
pages = {e31831},
year = {2025},
note = {2025 Sep;72(9):e31831},
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.},
subtyp = {Review Article},
keywords = {ALK (Other) / ATR (Other) / B7‐H3 (Other) / EZH2 (Other)
/ GD2 (Other) / GPC2 (Other) / MYCN (Other) / clinical
trials (Other) / drug development (Other) / neuroblastoma
(Other) / relapse (Other)},
cin = {B087 / HD01},
ddc = {610},
cid = {I:(DE-He78)B087-20160331 / I:(DE-He78)HD01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40509548},
doi = {10.1002/pbc.31831},
url = {https://inrepo02.dkfz.de/record/302020},
}