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Experimental Therapeutics Research

There is a large range of research questions that fall into the category of experimental therapeutics in mice.  The investigations span studies designed to discover potential targets and test for their role in cancer initiation, tumor progression, metastatic spread, and response or resistance to therapy to pre-clinical and co-clinical testing of drug candidates and clinical agents. A sort of conceptual "mouse hospital" model to maximize the comparisons to human therapeutics is being developed. This will help us move towards in vivo real time stratification of responses, and the identification of novel biomarkers of response and resistance, with the ultimate goal of accelerating drug development and approval.

Translational researchers employ mice to reflect the tumor situation in patients using a variety of types of models.  A well described example of this involves the numerous mouse models for BRCA1 that have been used for many investigations. Strategies sometimes called “chemical genomics” rely on screening protocols to identify genes and compounds that demonstrate evidence of alterations in the tumor status of mouse models. New mouse models that enable visualization help to assess drug responses in screening protocols.

Engineered mice and xenograft models have been used to study many aspects of drug development, including to validate possible drug targets, assess tumor responses, evaluate mechanisms of action, and to understand possible toxicity problems with new drugs or with treatments such as radiation.  Potential more general strategies for interfering with cell proliferation mechanisms or angiogenesis mechanisms are continuously being evaluated in mouse models as novel methods of intervention.  Vaccine strategies can also be explored in mouse models, which may lead to useful additional therapeutic routes.

In addition to screening for effective compounds in primary tumor situations, certain tumors become resistant to therapies and scientists are challenged to understand this problem.  Mouse models have been used to pursue those avenues as well. Multidrug resistant tumors have been studied in mice, for both intrinsic and acquired resistanceModulation of immune system effects has been attempted using mouse models.