By obtaining clinical specimens from participants with high-grade bone and soft tissue sarcomas to establish and profile as freshly implanted tumors in mice, the aim of this study is to identify agents with predicted activity in the host patient while also potentially providing them with individualized cancer treatment options
Patient-derived xenografts (PDX) are increasingly used as tools for drug development in pre-clinical settings, and have been shown to recapitulate the histology and behavior of the cancers from which they are derived. Although, they have been commonly used productively as pre-clinical disease models to study disease biology and drug response, they have not been used prospectively to inform clinical management. PDX have been employed to inform clinical decision-making in small studies, which have shown high concordance between individual PDX and patient responses to therapy. While encouraging, the role of this approach in bone and soft tissue sarcomas and in the context of genomic drug matching strategies remains undefined. This has created an opportunity to evaluate the utility of PDX as clinical predictors to direct the use of chemo- and targeted therapies in combination with comprehensive genomic and epigenetic analysis for patients with bone and soft tissue sarcomas.
Study Type
OBSERVATIONAL
Enrollment
20
Molecular profiling of host tumour sample and PDX will be performed and analyzed by an expert panel. In vitro drug testing using organoid culture generation may also be performed if sufficient fresh tissue is available. Matched treatment recommendation based on profiling and in vivo PDX drug testing results will be made, if available.
Measure of drug sensitive PDX to a panel of drugs as a predictor of clinical response in matched host
Sensitivity measured by tumor growth inhibition (\>80%) or objective tumor response (regression) as per Response Evaluation Criteria In Solid Tumors (RECIST) criteria.
Time frame: up to 2 years
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