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@ARTICLE{Rechberger:285048,
      author       = {J. S. Rechberger and S. M. Bouchal and E. A. Power and L.
                      Nonnenbroich$^*$ and C. L. Nesvick and D. J. Daniels},
      title        = {{B}ench-to-bedside investigations of {H}3 {K}27-altered
                      diffuse midline glioma: drug targets and potential
                      pharmacotherapies.},
      journal      = {Expert opinion on therapeutic targets},
      volume       = {27},
      number       = {11},
      issn         = {1460-0412},
      address      = {Abingdon, Oxon},
      publisher    = {Routledge, Taylor $\&$ Francis},
      reportid     = {DKFZ-2023-02191},
      pages        = {1071-1086},
      year         = {2023},
      note         = {2023 Jul-Dec;27(11):1071-1086},
      abstract     = {H3 K27-altered diffuse midline glioma (DMG) is the most
                      common malignant brainstem tumor in the pediatric
                      population. Despite enormous preclinical and clinical
                      efforts, the prognosis remains dismal, with fewer than
                      $10\%$ of patients surviving for two years after diagnosis.
                      Fractionated radiation remains the only standard treatment
                      options for DMG. Developing novel treatments and therapeutic
                      delivery methods is critical to improving outcomes in this
                      devastating disease.This review addresses recent advances in
                      molecularly targeted pharmacotherapy and immunotherapy in
                      DMG. The clinical presentation, diagnostic workup, unique
                      pathological challenges, and current clinical trials are
                      highlighted throughout.Promising pharmacotherapies targeting
                      various components of DMG pathology and the application of
                      immunotherapies have the potential to improve patient
                      outcomes. However, novel approaches are needed to truly
                      revolutionize treatment for this tumor. First, combinational
                      therapy should be employed, as DMG can develop resistance to
                      single-agent approaches and many therapies are susceptible
                      to rapid clearance from the brain. Second, drug-tumor
                      residence time, i.e. the time for which a therapeutic is
                      present at efficacious concentrations within the tumor, must
                      be maximized to facilitate a durable treatment response.
                      Engineering extended drug delivery methods with minimal
                      off-tumor toxicity should be a focus of future studies.},
      subtyp        = {Review Article},
      keywords     = {Diffuse midline glioma (Other) / H3 K27-altered (Other) /
                      H3 K27M (Other) / ONC201 (Other) / blood-brain barrier
                      (Other) / convection-enhanced delivery (Other) / diffuse
                      intrinsic pontine glioma (Other) / histone (Other) /
                      immunotherapy (Other) / targeted therapy (Other)},
      cin          = {B310 / HD01},
      ddc          = {610},
      cid          = {I:(DE-He78)B310-20160331 / 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:37897190},
      doi          = {10.1080/14728222.2023.2277232},
      url          = {https://inrepo02.dkfz.de/record/285048},
}