Exceptional responders may hold genetic keys to better cancer treatments

The National Cancer Institute has mounted a search for people who are dramatic outliers in their response to cancer therapies in an effort to improve treatments for common malignancies.

People with breast, bladder and other common cancers could one day be treated with new drugs based on a novel genetic paradigm made possible by studies that look for how these patients were able to respond better than their average counterparts. A number of studies have already opened the door for researchers like Barbara Conley, MD, associate director of the Cancer Diagnosis Program (CDP) in the Division of Cancer Treatment and Diagnosis (DCTD), National Cancer Institute; and David B. Solit, MD, the principal investigator in a study being conducted at Memorial Sloan-Kettering Cancer Center (MSKCC).

In the MSKCC study assessing the effectveness of the drug everolimus for bladder cancer, 45 patients were administered the drug—only two responded.

“The verdict was, ‘O.K., I guess everolimus does not work in bladder cancer,’” said Solit in this New York Times report. However, one of those two patients had metastases to the abdomen and had been given less than a year to live, yet she still responded to the therapy. “I was at a clinical meeting, and everyone was saying this drug did not work...I said, ‘It worked for her.’"

This and other studies could provide fodder for deeper genetic research into the mechanisms by which these patients are able to respond so favorably. Such insight could be turned into drug or gene therapies that could potentially improve health outcomes for cancer patients.