NCI launches “exceptional responders” initiative

It is well understood that some patients exhibit exceptionally good responses to investigational and to standard forms of treatment for cancer — well beyond the responses exhibited by the majority of patients treated with the same type of investigational or standard treatment.

Although this has been known for some time, and there has always been interest in why such patients have good and unusual responses, there has never (until today) been a deliberate and coordinated determination to actually study why some patients have these unusual types of response.

However, the National Cancer Institute has now launched the Exceptional Reponders Initiative (click here for a detailed media release) that is designed to look at such exceptional responders to therapy in a coordinated and thorough manner.

Patients who appear to have had exceptional responses to treatment for their prostate cancer might want to talk to their doctors about whether their data and  whatever biological specimens are available could be submitted for analysis, although we should be clear that it will only be useful to the NCI research group if really reliable and high quality data are available for patients treated in the community setting and outside of randomized clinical trials.

What might constitute an “exceptional response” to treatment for prostate cancer? Here are two possible examples:

  • 20-year survival (or longer) after first-line therapy only in a man initially diagnosed with a Gleason score of 10 and a PSA of > 20 ng/ml
  • 10-year survival (or longer) after the onset of castration resistance in any patient with prior, evident, metastatic disease

One of the primary goals of this study is to be able to identify molecular markers that predict highly positive responses to certain therapies, even in a small subset of patients. Identification of such biomarkers might make it possible to more effectively choose treatment programs for individual patients who exhibit such biomarkers.

Additional details about this initiative can be found on the web site.

One Response

  1. It is so encouraging that such a study is being launched.

    Having read the news release and the text at, Here is one observation on a limitation of this study, probably a limitation that is unavoidable given the pilot nature of the work and the current levels of cancer and genetic technology: both the outcome measures and the eligibility description refer to patients’ reactions to “the drug”, rather than to a combined approach involving several agents. There have been cases where a single agent, such as Gleevec, has accomplished a profound effect, but increasingly often it appears that success against cancer involves simultaneous attacks against several cancer survival pathways.

    While the description points toward genetic analysis playing a key role, I’m thinking that complementary research on the epigenomic and proteomic environments may also come into play in analysis.

    Cool stuff!

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