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Study of ‘Exceptional Responders’ Yields Clues to Cancer, Potential Treatments

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Genomic characterizations of cancer can uncover genetic alterations that may contribute to unexpected and long-lasting responses to treatment, according to researchers.

In a comprehensive analysis of patients with cancer who had exceptional responses to therapy, researchers have identified molecular changes in the patients’ tumors that may explain some of the exceptional responses. The results demonstrate that genomic characterizations of cancer can uncover genetic alterations that may contribute to unexpected and long-lasting responses to treatment, according to the researchers.

NCI researchers conducted the study in collaboration with investigators from other institutions, including NCI-designated cancer centers. The results appeared in Cancer Cell.

The study—which defined an exceptional responder as someone who had a partial or complete response to a treatment that would be effective in less than 10 percent of similar patients—included detailed medical histories and tumor samples from 111 patients with various types of cancer who had received standard treatments, such as chemotherapy. 

The patients had been identified by NCI’s Exceptional Responders Initiative, a national project launched in 2014 to explore the feasibility of collecting and analyzing the data and biospecimens needed to better understand the biological basis of exceptional responses in cancer. 

“The majority of patients in this study had metastatic cancers that are typically difficult to treat, yet some of the patient responses lasted for many years,” said Dr. Louis Staudt, director of NCI’s Center for Cancer Genomics, who co-led the study. “Researchers and the doctors who treat these patients have long been curious about the mechanisms underlying these rare responses to treatment. Using modern genomic tools, we can now start to solve these fascinating puzzles.”

For 26 of the 111 patients, researchers identified molecular features that could potentially explain exceptional responses to treatment, such as the co-occurrence of multiple rare genetic changes in the tumor genome or the infiltration of the tumor with certain types of immune cells.

Dr. Percy Ivy of NCI’s Division of Cancer Treatment and Diagnosis added, “The knowledge gained from studying exceptional responders can help inform how we take care of patients in the future and will help move us closer to the goal of precision oncology.”

The NIH Record

The NIH Record, founded in 1949, is the biweekly newsletter for employees of the National Institutes of Health.

Published 25 times each year, it comes out on payday Fridays.

Associate Editor: Carla Garnett
Carla.Garnett@nih.gov

Staff Writers:

Eric Bock
Eric.Bock@nih.gov

Dana Talesnik
Dana.Talesnik@nih.gov

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