NCI Seeking So-Called Exceptional Responders for Study


Sometimes, despite overwhelming odds, a patient who has no other options or who is considered terminal will suddenly respond to treatment. The National Cancer Institute wants to know why.

To do so they have launched a study into the phenomena of “Exceptional Responders” – cancer patients who have a unique response to treatments (primarily chemotherapy) that have not been effective for most other patients:

For this initiative, exceptional responders will be identified among patients enrolled in early-phase clinical trials in which fewer than 10 percent of the patients responded to the treatments being studied; patients who were treated with drugs not found to be generally effective for their disease; patients who were treated in later-phase clinical trials of single agents or combinations; and even patients who were treated with established therapies. In this pilot study, malignant tissue (and normal tissue, when possible) and clinical data will be obtained from a group of exceptional responders and analyzed in detail. The goal is to determine whether certain molecular features of the malignant tissue can predict responses to the same or similar drugs.

They're seeking 100 such exceptional responders, and they'll want tissue samples. With them, they will compare DNA sequences and RNA transcript levels and other molecular measurements to try to understand why these patients were such outliers in their response to treatment.

There is hope: an exceptional responder with bladder cancer led researchers to discover a new molecular pathway involved in the development of the disease, and suggested new therapeutic approaches for other similar patients.

If you or someone you know might qualify for the study, read on:

Patients who believe they may be exceptional responders should contact their physicians or clinical trialists to see if they can assist in submitting tissue for consideration. [...] Investigators who have tissue from a potential exceptional responder should send an email to [email protected]. The email should include a short description of the case, without patient identifiers; information about whether tissue collected before the exceptional response is available; whether informed consent was given to use tissue for research; and the patient’s vital status.

Source: NCI

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