Exceptional Responders Inspire Change: Lessons for Drug Development From the Bedside to the Bench and Back

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 Targeted therapies have changed the landscape of cancer treatment. Unfortunately, the promise of “targeted therapy” still fails too many patients with advanced cancer. Oncologists should ask why and how can we rapidly develop novel strategies. In medicine, exciting and novel occurrences are often first publicized as a case report. This quick yet detailed description of the clinical presentation, diagnosis, treatment, and outcome of an individual patient remains an important part of medical progress. However, although case reports serve as anecdotal clinical evidence that may be shared for educational, scientific, or medical purposes, the amount of information that can be generalized to a wider group of cancer patients is typically limited. Can case reports extend knowledge beyond the single patient experience and offer mechanistic insight or lead to novel therapeutic strategies?.....