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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},
}