The mouse as a model for neuropsychiatric drug development (Howe et al., 2018)

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Much has been written about the validity of mice as a preclinical model for brain disorders. Critics cite numerous examples of apparently effective treatments in mouse models that failed in human clinical trials, raising the possibility that the two species’ neurobiological differences could explain the high translational failure rate in psychiatry and neurology (neuropsychiatry). However, every stage of translation is plagued by complex problems unrelated to neurobiological conservation. Therefore, although these case studies are intriguing, they cannot alone determine whether these differences observed account for translation failures. Our analysis of the literature indicates that most neuropsychiatric treatments used in humans are at least partially effective in mouse models, suggesting that neurobiological differences are unlikely to be the main cause of neuropsychiatric translation failures.

In Plain English:

The failure to develop many effective drugs to treat neuropsychiatric diseases has been blamed in part on the inadequacy of the mouse model. In this essay, using a back-translational approach to provide evidence, we argue that the mouse should continue to serve an important role in neuropsychiatric drug development.


This publication was the basis of a presentation I gave at UCLA on Mar. 1, 2019. The slides from this presentation can be accessed here.