NCI's New Approach With The Exceptional Responder Initiative

With the aim of understanding why some patients respond better to a particular drug, NCI has launched the Exceptional Responders Initiative, to generate molecular profiles of tumors that respond well to a particular treatment.

The Exceptional Responders Initiative, a study to investigate the molecular factors of tumors associated with exceptional treatment responses of cancer patients to drug therapies, was launched today by the National Cancer Institute (NCI), part of the National Institutes of Health. Scientists will attempt to identify the molecular features of tumors that predict whether or not a particular drug or class of drugs will be beneficial. Investigators will examine tumor specimens from patients in clinical trials who achieved an exceptional response relative to other trial participants, or other patients who achieved an exceptional and unexpected response to a non-investigational therapy.

This initiative was initially considered two years ago when, through the use of advanced DNA sequencing methods, the molecular basis for the prolonged remission of bladder cancer in a patient following treatment with a molecularly targeted drug in a clinical trial was determined. Researchers thought it might be possible to understand the mechanism of drug response for a relatively small number of patients in early phase clinical trials who benefit dramatically from therapy. In the past, trials in which up to only 10 percent of patients had significant and prolonged responses were considered unsuccessful because it was not possible to understand why some patients benefitted from treatment and others did not.

NIH press release: