Mining the Outliers

Even when a clinical trial fails, some patients improve. What can researchers learn from these exceptional responders?

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TREATABLE: Exceptional responders are patients whose therapeutic experience diverges from that of the majority of clinical trial participants.© RHODA BAER/NATIONAL CANCER INSTITUTE

Last year, Harvard’s Jochen Lorch and colleagues took a leap of faith by enrolling a handful of patients with a deadly form of thyroid cancer in a clinical trial involving patients with a much more tractable form of the disease. The study was designed to test Novartis’s Afinitor (everolimus) on patients with differentiated thyroid cancer, typically considered a curable disease. Lorch and his colleagues didn’t have high hopes for the anaplastic thyroid cancer patients, who tend to survive less than five months after diagnosis, but with few treatment options, they figured it was worth a shot. Amazingly, the second anaplastic thyroid cancer patient they enrolled responded to the drug: the mass in her chest began to shrink almost immediately.

For about 18 months, the patient’s ...

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Meet the Author

  • Jef Akst

    Jef Akst was managing editor of The Scientist, where she started as an intern in 2009 after receiving a master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses.

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