As many as 1 in 10 patients respond well in clinical trials of experimental medicines that U.S. regulators end up rejecting, according to the National Cancer Institute (NCI). To understand why these patients had such a response, researchers are beginning to use DNA sequencing technology to determine if the patients they call “exceptional responders” carry gene variations that can lead to better targeted therapies, including new treatments and the reconsideration of others.
Traditional treatments such as chemotherapy kill healthy cells along with malignant ones, but targeted therapies are designed to leave healthy cells unscathed and home in on cancer cells that make tumors grow and spread. The catch is that they don’t work for everyone, and even patients who find them helpful tend to develop resistance over time. The NCI and academic medical centers including Memorial Sloan Kettering Cancer Center in New York, the Dana-Farber Cancer Institute and Massachusetts General Hospital in Boston, and the Broad Institute in Cambridge, Mass., are creating a national database of exceptional responders to aid research. “What was yesterday’s miracle event is today becoming a subject of scientific inquiry,” says Leonard Lichtenfeld, an oncologist and the deputy chief medical officer of the American Cancer Society.
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