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Exceptional Responders

Posted 10/7/2014

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  We have all heard the miracle stories: someone who is seemingly at death's door who receives a "Hail Mary" drug and gets better. I have known a few women, maybe three or four, over the decades whose cancers were pushed back when no one thought that might happen. In one instance, a young woman had moved into a residential hospice. Her oncologist treated her with a old combination of chemotherapy drugs that were rarely used (many others had proven to be more effective), and she improved. Matter of fact, she improved so much that she moved out of the hospice and back to her apartment. Sadly, this was not a completely happy ending as her cancer did worsen again, and she moved back to the hospice and later died. However, the point here is that a drug that was not expected to be very helpful, but was known to have few side effects (so little down side in her case), bought her another nine months of pretty good life.

  Most of all, of course, patients need to believe that they could be one of these "exceptional responders", people who benefitted from drugs that, overall, help less than 10% of those people who receive them. If doctors could figure out how to identify these people, could learn what is it about their cancer cells that make the drugs effective, it would be a big leap forward. This seems to be a parallel challenge to the ongoing search for new targeted therapies; both approaches attempt to individualize treatments and better understand who can be helped with any one particular drug.

  The National Cancer Institute, the NCI, has just announced an initiative to study exceptional responders. Here is an article from Medscape that explains the project. I will then also post a link to an NCI document about it.

Keys to 'Exceptional Responders' to Cancer Drugs Sought
Janis C. Kelly

Most clinicians will have at least one anecdotal incident of a patient who showed an exceptional response to treatment. A new initiative at the National Cancer Institute (NCI) is aimed at finding out why some patients respond so dramatically.
The first-ever Exceptional Responders Initiative has been launched to examine tumors from more than 100 cancer patients who had good responses to treatments that benefited, overall, less than 10% of treated patients. Coleader Barbara A. Conley, MD, associate director of the NCI Cancer Diagnosis Program, and colleagues want to determine are molecular biomarkers in the tumor that can predict response to the drug tested or to drugs with similar mechanisms of action.
"The NCI has been interested in how current molecular analysis capability can be used to improve how we treat patients," Dr Conley told Medscape Medical News.
"NCI Director Harold E. Varmus, MD, met with experts from across the country and heard from Dr Solit and others who had found molecular reasons for a particular response," she explained.

http://www.medscape.com/viewarticle/832831_print

And here is the NCI link:

http://www.cancer.gov/newscenter/newsfromnci/2014/ExceptionalRespondersQandA/print

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