50 whys to look for genes: 22. Exceptional responders to drug treatment

Sometimes experimental treatments for cancers are effective for only a small number of patients—”exceptional responders.”  Sequencing of their cancers may reveal a genetic mutation that explains why the drug was effective (Kolata 2014).  Future patients can have their cancer genomes analyzed to determine whether an effective drug is known for their profile of mutations.

Complications

  • Drug companies discontinue production of the drug because it works for so few.
  • “Researchers… see hundreds of mutations in a cancer and none will explain a patient’s response to a drug,” molecular oncologist quoted in Kolata (2014)
  • Cancer centers lure patients using the possibility that they are the rare exceptional responders.

Reference
Kolata, G. (2014) Finding Clues in Genes of ‘Exceptional Responders’ New York Times, 8 October

(Introduction to this series of posts)

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