Exceptional Responders: The “N of 1” Paradigm

November 5, 2014
Exceptional Responders: The “N of 1” Paradigm
The NCI is funding research to determine whether outliers from failed clinical trials can reveal hidden drug development possibilities. [iStock/francisblack]

Patricia Fitzpatrick Dimond, Ph.D.

We have all heard stories about the single patient who survives a deadly cancer in response to a particular drug, in contrast to other patients receiving the same drug who succumb to the disease.

These patients may include heavily pretreated cancer patients for whom other drugs have failed, but then who respond to a therapy not typically used to treat their type of cancer. Now, with novel genomics tools at their disposal, investigators say that by analyzing the drug responses of “n-of-1,” patients who are single outliers in clinical studies, new insights into cancer mechanisms and more effective treatments can be gained. 

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