Research ArticleNEUROIMAGING

Learning to identify CNS drug action and efficacy using multistudy fMRI data

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Science Translational Medicine  11 Feb 2015:
Vol. 7, Issue 274, pp. 274ra16
DOI: 10.1126/scitranslmed.3008438

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Brain patterns determine drug efficacy

There are many drugs out there that affect the central nervous system (CNS), from drugs for chronic pain to schizophrenia to obesity. A brain imaging technique called functional magnetic resonance imaging (fMRI) has shown promise for distinguishing an effective compound from an ineffective one, but the real unmet need is to be able to predict whether a new CNS drug will have clinical efficacy. To this end, Duff et al. evaluated existing fMRI data sets for patients who were exposed to painful stimuli (such as heat or a squeeze) and given either an analgesic compound or a placebo. From these brain “maps,” or neural signatures, the authors were able to create a general “stop/go” decision-making framework—which included quality assurance, pharmacodynamic effect, and evidence for clinical efficacy steps—that allowed them to determine whether the signature of a new compound provided evidence for analgesic properties. Other than evaluating potential drug efficacy, the authors revealed insights into pain response mechanisms. This multistudy approach by Duff et al. may translate to a powerful tool in synthesizing and learning from neuroimaging data to improve—and perhaps speed up—CNS drug discovery and repurposing.

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