NIH launches study of 'exceptional responders' to cancer therapies

The National Cancer Institute has launched a three- to four-year broad-based study of exceptional responders--patients who have had particularly good outcomes on cancer therapies as compared to their peers. The initiative was first considered a few years ago when it started to become easier to determine the molecular basis of why a particular patient's cancer responds to a molecularly targeted drug, but it is only being undertaken now.

Investigators are collecting tumor specimens from patients in clinical trials who achieved a better response versus other trial participants, as well as from patients who do so on approved therapies.

The NCI plans to collect samples and data from NCI-supported clinical trials, as well as other clinical trials, standard therapy settings such as community practice and from pharma industry trials. It's currently sending out letters of solicitation to cancer centers and other clinicians nationally to locate suitable samples.

"The increasing ability of molecular technologies to stratify tumor types by prognosis or response to treatment will result in many common cancers being separated into specific subtypes that may respond to drugs in very different ways," Dr. Barbara Conley of the Division of Cancer Treatment and Diagnosis at NCI and a co-lead investigator for the study said in a statement. NCI is part of the National Institutes of Health.

"The ability to identify molecular markers that are able to predict a clinical response in these subsets of patients will provide us with the tools to further advance our ability to conduct studies consistent with the principles of precision medicine," she added.

The DNA and RNA from tissue samples will be isolated at the Biospecimen Core Resource at Nationwide Children's Hospital in Columbus, OH. Those isolates will be shipped for DNA sequencing and analysis to Baylor University in Houston, TX.

This is an exploratory study intended to scope out the feasibility of a larger exceptional responder study. The hope is that researchers in the current study may produce a list of relevant mutations in exceptional responder cases.

Investigators expect this dataset could enable scientists to test any resulting hypotheses in a trial for a particular drug or by comparing their own data to it. They anticipate putting the full genomic annotation of 100 cases of exceptional responders in the public domain.

The study is expected to help identify patients who may be responders to particular agents with a certain mechanism of action. It could also allow for the advancement of treatments to which only a very small portion of the population responds. The researchers warn that relevant mutations may be present in less than 5% of tumors, making them difficult to find.

- here's the release

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