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Orthotopic Patient-Derived Xenografts of Pediatric Solid Tumors

Pediatric solid tumors arise from endodermal, ectodermal, or mesodermal lineages. Although the overall survival of children with solid tumors is 75%-80%, that of children with recurrent disease is below 30%. To capture the complexity and diversity of pediatric solid tumors and establish new models of recurrent disease, we developed a protocol to produce orthotopic patient-derived xenografts (O-PDXs) at diagnosis, recurrence, and autopsy. Tumor specimens were received from 168 patients, and 64 O-PDXs were established for 12 types of pediatric solid tumors. The origins of the O-PDX tumors were reflected in their gene-expression profiles and epigenomes. Genomic profiling of the tumors, including detailed clonal analysis, was performed to determine whether the clonal population in the xenograft recapitulated the patient’s tumor. We identified several drug vulnerabilities and showed that the combination of a WEE1 inhibitor (AZD1775), irinotecan, and vincristine can lead to complete response in multiple rhabdomyosarcoma O-PDX tumors in vivo.

Click on a Dataset ID in the table below to learn more, and to find out who to contact about access to these data

Dataset ID Description Technology Samples
EGAD00001003432 Illumina HiSeq 2000 20
EGAD00001003433 Illumina HiSeq 2000 98
EGAD00001003434 Illumina HiSeq 2000 149
EGAD00001003435 Illumina HiSeq 2000 150
Publications Citations
Orthotopic patient-derived xenografts of paediatric solid tumours.
Nature 549: 2017 96-100
Barriers to accessing public cancer genomic data.
Sci Data 6: 2019 98
VCF2CNA: A tool for efficiently detecting copy-number alterations in VCF genotype data and tumor purity.
Sci Rep 9: 2019 10357
MYCN amplification and ATRX mutations are incompatible in neuroblastoma.
Nat Commun 11: 2020 913
CAR T cells targeting tumor-associated exons of glypican 2 regress neuroblastoma in mice.
Cell Rep Med 2: 2021 100297