Single Mouse Trials

The Single Mouse Trial (SMT) format for in vivo patient-derived xenograft (PDX) studies addresses the need for more cost-effective compound testing in the larger, more diverse tumor populations that can be targeted using our vast portfolio of highly characterized models.

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The SMT format employs a single mouse per PDX model and treatment arm, thereby enabling a cost-effective investigation of in vivo efficacy in large panels of PDX models. The SMT format reliably identifies compounds that are strongly active (or inactive) across a panel of tumor models, and can be used to investigate the most promising candidate compounds, new indications or optimal drug combinations.

Possible applications include:

  • Identification of a large number of compounds in diverse tumor populations
  • Efficient and reliable identification of treatments with broad anti-tumor activity
  • Identification of resistance mechanisms
  • Strategic exploration of new drug combinations
  • Comprehensive exploration of one histotype
  • Screening of a particular molecular characteristic
  • Expansion of clinical indications by exploring other tumor types
  • Generation of data for biomarker analysis and identification

Below, we address some of the most frequently asked questions about our single mouse trials. For answers to your specific study questions or to receive a quote, please contact us.

Can we use the SMT format to screen a panel of cancer models with a limited number of compounds?
Yes. We design all studies around each client’s unique requirements.
Would you recommend SMT for drug combinations?
What type of mouse is best for the PDX model?
Do you sell the tumor models with the mice?
Do you sell your data analysis software to accompany the SMT?
What kind of genomic data are currently available for specific PDX models (RNA-seq, whole exome seq, etc.)?
Is this a model system to screen for treatment efficacy in humans?
What types of PDX models do you have?
How are your xenograft models established from patient tumor tissue?
How do you optimize the engraftment of your PDX models?
Are extra mice implanted to get one mouse per group?
If you are planning 10 mice per tumor type implant, how do you ensure you will have the correct number of mice for our study?
Is repetition required in this kind of study?
What are your criteria to exclude any outliers?
Do you need to run the conventional model for regulatory purposes after SMTs?
Is dose selection dependent upon input from clients or do you provide guidance?
Are there any limitations on route of administration?
How many tumor lines should be tested in a single trial to get good clinical relevance for a single organ or cancer type?
How do you handle intellectual property (IP) rights when studies involve novel investigational agents?