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@ARTICLE{Rusert:163998,
      author       = {J. M. Rusert and E. F. Juarez and S. Brabetz$^*$ and J.
                      Jensen and A. Garancher and L. Q. Chau and S. K.
                      Tacheva-Grigorova and S. Wahab and Y. T. Udaka and D. Finlay
                      and H. Seker-Cin$^*$ and B. Reardon and S. Gröbner$^*$ and
                      J. Serrano and J. Ecker$^*$ and L. Qi and M. Kogiso and Y.
                      Du and P. A. Baxter and J. J. Henderson and M. E. Berens and
                      K. Vuori and T. Milde$^*$ and Y.-J. Cho and X.-N. Li and J.
                      M. Olson and I. Reyes and M. Snuderl and T. C. Wong and D.
                      P. Dimmock and S. A. Nahas and D. Malicki and J. R. Crawford
                      and M. L. Levy and E. M. Van Allen and S. Pfister$^*$ and P.
                      Tamayo and M. Kool$^*$ and J. P. Mesirov and R. J.
                      Wechsler-Reya},
      title        = {{F}unctional precision medicine identifies new therapeutic
                      candidates for medulloblastoma.},
      journal      = {Cancer research},
      volume       = {80},
      number       = {23},
      issn         = {1538-7445},
      address      = {Philadelphia, Pa.},
      publisher    = {AACR},
      reportid     = {DKFZ-2020-02198},
      pages        = {5393-5407},
      year         = {2020},
      note         = {2020 Dec 1;80(23):5393-5407},
      abstract     = {Medulloblastoma (MB) is among the most common malignant
                      brain tumors in children. Recent studies have identified at
                      least four subgroups of the disease that differ in terms of
                      molecular characteristics and patient outcomes. Despite this
                      heterogeneity, most MB patients receive similar therapies,
                      including surgery, radiation, and intensive chemotherapy.
                      Although these treatments prolong survival, many patients
                      still die from the disease and survivors suffer severe
                      long-term side effects from therapy. We hypothesize that
                      each MB patient is sensitive to different therapies and that
                      tailoring therapy based on the molecular and cellular
                      characteristics of patient tumors will improve outcomes. To
                      test this, we assembled a panel of orthotopic
                      patient-derived xenografts (PDX) and subjected them to DNA
                      sequencing, gene expression profiling, and high-throughput
                      drug screening. Analysis of DNA sequencing revealed that
                      most MB do not have actionable mutations that point to
                      effective therapies. In contrast, gene expression and drug
                      response data provided valuable information about potential
                      therapies for every tumor. For example, drug screening
                      demonstrated that actinomycin D, which is used for treatment
                      of sarcoma but rarely for MB, was active against PDX
                      representing Group 3 MB, the most aggressive form of the
                      disease. Functional analysis of tumor cells was successfully
                      used in a clinical setting to identify more treatment
                      options than sequencing alone. These studies suggest that it
                      should be possible to move away from a one-size-fits-all
                      approach and begin to treat each patient with therapies that
                      are effective against their specific tumor.},
      cin          = {B062 / HD01 / B310},
      ddc          = {610},
      cid          = {I:(DE-He78)B062-20160331 / I:(DE-He78)HD01-20160331 /
                      I:(DE-He78)B310-20160331},
      pnm          = {312 - Functional and structural genomics (POF3-312)},
      pid          = {G:(DE-HGF)POF3-312},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:33046443},
      doi          = {10.1158/0008-5472.CAN-20-1655},
      url          = {https://inrepo02.dkfz.de/record/163998},
}