Available Resources

Preclinical models of childhood solid tumors (GEMMs)

GEMM-derived tissue samples developed at St. Jude are available for a variety of pediatric solid tumors, including neuroblastoma, retinoblastoma and osteosarcoma. Available strains are detailed on the request form.

Protocols (ChIPSeq)

Protocols with various antibodies, targets and tissue types have been developed and validated at St. Jude and are available to be shared. Available ChIPSeq protocols are detailed on the request form under “Protocols & Reagents”.

Patient-derived orthotopic xenografts

Orthotopic patient derived xenografts (O-PDXs) have been created from a wide variety of pediatric solid tumors collected under the Molecular Analysis of Solid Tumors (MAST) protocol at St. Jude Children’s Research Hospital (ClinicalTrials.gov: NCT01050296). Most O-PDX models are available with a luciferase reporter gene integrated into the genome to enable monitoring of tumor engraftment and growth in vivo. Cryopreserved cells, fresh frozen tissue/cells, and formalin-fixed paraffin-embedded tissue blocks are available. See below for the full list of tumor types and samples. Extensive molecular and drug sensitivity data are available for the O-PDX models. For each of the O-PDX models, a characterization sheet will be provided when the samples are shipped (see example for SJNBL046_X or visual descriptions of the clonal evolution groups.) Characterization sheets can also be requested to help investigators select the appropriate O-PDX models for their studies. For the samples that have been added most recently, characterization is pending and the completed characterization sheets will be sent to requestors as soon as they are available.

The list of available tumors is updated every 6 months (January and July) after the samples have engrafted and sufficient vials have been cryopreserved for distribution.

Neuroblastoma samples

Symbols/Abbreviations: – = not available, mo = months, N = no, Tx = treatment, Y = yes, yr = years.
Pending = characterization is ongoing.

Osteosarcoma samples

Symbols/Abbreviations: F = female, M = male, Tx = treatment, yr = years.
Pending = characterization is ongoing.

Rhabdomyosarcoma samples

Symbols/Abbreviations: ARMS = alveolar rhabdomyosarcoma, ERMS = embryonal rhabdomyosarcoma, F = female, M = male, mo = months, Tx = treatment, yr = years.
Pending = characterization is ongoing.

Retinoblastoma Samples

Please note that retinoblastoma sample size is small so tissue distribution may be limited.
Symbols/Abbreviations: F = female, M = male, mo = months, Tx = treatment, yr = years.
Pending = characterization is ongoing.

Other samples

Symbols/Abbreviations: F = female, M = male, mo = months, Tx = treatment, yr = years.
Pending = characterization is ongoing.

Patient derived iPSCs

Induced pluripotent stem cells (iPSCs) have been created from 15 patients with retinoblastoma that have germline RB1 mutations collected under the RETCELL (A Study of Children with Heritable Retinoblastoma) protocol at St Jude Children’s Research Hospital (ClinicalTrials.gov NCT02193724). The clinical spectrum of disease varies from early diagnosis (Group A and/or B) to advanced bilateral disease (Group D and/or E). In addition, some participants were found to be unaffected or spontaneously regressed carriers of the heritable mutation or chromosomal abnormality. The RB1 mutations have been validated in each line and they have undergone complete characterization including trilineage differentiation, whole genome sequencing, karyotyping and 3D retinal organoid differentiation. Vials of cryopreserved cells are available upon request. Additional iPSC lines are being developed for other pediatric solid tumor types and the list of available lines will be updated every 6 months after they have been validated and characterized.

1: OU, oculus uterque (both eyes); OS, oculus sinister (left eye); OD, oculus destrus (right eye), trilateral, OU disease with tumor in the pineal gland at diagnosis 
2: Reese-Ellsworth staging (1-5) and International Classification of Intraocular Retinoblastoma (A-E)

Genomic, drug sensitivity and pharmacokinetic data

We offer molecular and drug sensitivity data of diverse pediatric solid tumors, patient-derived orthotopic xenografts and solid tumor cell lines. (Some cell lines are available; see the request form for details.) High-throughput drug screening (HTS) data are available for many pediatric solid tumor cell lines and orthotopic xenografts, including Ewing sarcoma, neuroblastoma, osteosarcoma, retinoblastoma and rhabdomyosarcoma.

Access Drug Sensitivity Data

While comprehensive raw datasets are not made available prior to publication, we are happy to accommodate many types of data requests to answer specific research questions.

Genomic sequencing and integrated epigenetic profiling have been performed on pediatric solid tumors and patient-derived orthotopic xenografts as part of the St. Jude-Washington University Pediatric Cancer Genome Project and is actively ongoing in St. Jude investigator-led labs. Available data may include:

• Whole genome sequencing (WGS)
• Whole exome sequencing (WES)
• RNA-seq
• Whole-genome bisulfite sequencing (WGBS)
• ChIP-seq

High-throughput drug screening (HTS) data are available for many pediatric solid tumor cell lines and orthotopic xenografts, including Ewing sarcoma, neuroblastoma, osteosarcoma, retinoblastoma and rhabdomyosarcoma. In addition, we offer preclinical pharmacokinetic data for a number of high-priority drugs that are being explored for activity against pediatric solid tumors. Standard of Care regimens used in current preclinical trials can be found in this appendix. Data from completed preclinical Phase I (tolerability), Phase II (small pilot efficacy study) and Phase III (randomized, placebo controlled) trials are available by request. For more details and descriptions of Phase I, II and III, please see Stewart et al, 2014 Cell Reports.