From Cell Biology to Tissue Engineering



Facilitating tailored therapeutic strategies for glioblastoma through an orthotopic patient-derived xenograft platform

Hye Won Lee1,2,3, Kyoungmin Lee2,4, Dong Geon Kim2,3,4, HeeKyoung Yang2,3 and Do-Hyun Nam2,4,5

1Research Institute for Future Medicine, Samsung Medical Center, 2Institute for Refractory Cancer Research, Samsung Medical Center, 3Samsung Biomedical Research Institute, Samsung Medical Center, 4Department of Health Sciences and Technology, SAIHST, Sungkyunkwan University and 5Departments of Neurosurgery, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Korea

Offprint requests to: Do-Hyun Nam, Department of Neurosurgery, Samsung Medical Center, Sungkyunkwan University, School of Medicine, 81 Irwon-ro, Gangnam-Gu, Seoul 135-710, Korea. e-mail: nsnam@skku.edu

Summary. Despite years of research into its pathobiology and continuing clinical trials for novel therapies, the prognosis for patients with glioblastoma (GBM) remains dismal. An important obstacle against treatment efficacy may be a high degree of intra- and inter-tumoral heterogeneity within GBMs, which may be caused by the presence of self-renewing GBM stem cells (GSCs). Recent advances in multi-omics technology introduce new possibilities for applying personalized strategies to GBM therapy. As drug discovery is accelerating with the transition from non-selective, cytotoxic therapy to a precision, targeted approach, the appropriate in vivo platform for GBM is critical for validating drug targets and prioritizing candidates for clinical studies, for co-development of companion diagnostics and, ultimately, for drug approval. Here we will describe GBM orthotopic patient-derived xenografts (PDXs) as more useful, clinically relevant resources for individually tailored strategies for GBM. Histol Histopathol 31, 269-283 (2016)

Key words: Glioblastoma, Personalized medicine, Tumor heterogeneity, Glioblastoma stem cells, Tumor microenvironment, Patient-derived xenografts, Orthotopic

DOI: 10.14670/HH-11-695