Mining the Outliers

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

Written byJef Akst

| 4 min read

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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
Go to The Scientist's LinkedIn Page
Jef (an unusual nickname for Jennifer) got her master’s degree from Indiana University in April 2009 studying the mating behavior of seahorses. After four years of diving off the Gulf Coast of Tampa and performing behavioral experiments at the Tennessee Aquarium in Chattanooga, she left research to pursue a career in science writing. As The Scientist's managing editor, Jef edited features and oversaw the production of the TS Digest and quarterly print magazine. In 2022, her feature on uterus transplantation earned first place in the trade category of the Awards for Excellence in Health Care Journalism. She is a member of the National Association of Science Writers.
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