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@ARTICLE{Lou:144570,
      author       = {E. Lou and A. C. Nelson and M. Kool$^*$},
      title        = {{D}ifferential response of {SHH}-expressing adult
                      medulloblastomas to the sonic hedgehog inhibitor vismodegib:
                      whole-genome analysis.},
      journal      = {Cancer biology $\&$ therapy},
      volume       = {20},
      number       = {11},
      issn         = {1555-8576},
      address      = {Georgetown, Tex.},
      publisher    = {Landes Bioscience},
      reportid     = {DKFZ-2019-02013},
      pages        = {1398-1402},
      year         = {2019},
      note         = {20(11):1398-1402.},
      abstract     = {Medulloblastoma is an aggressive primitive neuroectodermal
                      tumor of the cerebellum that is more common in children than
                      in adults. In the past decade, advances in understanding the
                      molecular drivers of medulloblastoma have identified four
                      molecular subgroups defined by experimental gene expression
                      profiles: the WNT pathway, sonic hedgehog (SHH) pathway, and
                      subgroups 3 and 4 (non-SHH/WNT).  Medulloblastoma of adults
                      belong primarily to the SHH category. Vismodegib, an
                      SHH-pathway inhibitor, FDA-approved in 2012 for treatment of
                      basal cell carcinoma, has been used successfully in the
                      setting of chemorefractory medulloblastoma, but not as a
                      first-line therapy. In 2016, we reported a case of an adult
                      patient with a sustained response of an unresectable
                      multifocal form of adult medulloblastoma to vismodegib.
                      Molecular analysis in that case revealed mutations in TP53
                      and a cytogenetic abnormality, i17q, that is prevalent and
                      most often associated with subgroup 4 rather than the
                      SHH-activated form of medulloblastoma. Here, we report
                      further whole-genome analysis of that patient (designated
                      Patient A) as well as an additional adult patient (Patient
                      B) whose tumor harbored the SHH molecular subgroup but which
                      was unresponsive to visgmodegib therapy. Comparison of these
                      disparate responses highlights the challenges to tailoring
                      SHH-targeted treatment in individual patients with adult
                      medulloblastoma.},
      cin          = {B062 / L101},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)L101-20160331},
      pnm          = {312 - Functional and structural genomics (POF3-312)},
      pid          = {G:(DE-HGF)POF3-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:31423907},
      doi          = {10.1080/15384047.2019.1647057},
      url          = {https://inrepo02.dkfz.de/record/144570},
}