Maurie Markman, MD
The era of precision medicine is upon us. Doubters should note the acceleration in regulatory agency approvals for novel antineoplastic agents for which clinical activity is defined by a specific molecular abnormality or at least a well-characterized constellation of biological events (eg, microsatellite instability–high). These identify patients with a probability of attaining clinical benefit from particular approaches to disease management.
The essential component that permits the process of precision medicine to achieve a successful outcome is the discovery and subsequent validation of a biomarker that, by its presence or absence, has been demonstrated to be predictive of a particular strategy’s utility.
Today, the focus of biomarker discovery is on abnormalities present within the cancer itself or germline events (eg, BRCA
mutations) that will predict for the biological and clinical activity of a given antineoplastic agent. It is reasonable to speculate that, in the future, unique signatures found within a given patient’s germline will also be used to identify individuals who may have a meaningfully greater probability of experiencing a clinically relevant toxicity (eg, neuropathy and stomatitis) from a particular drug or class of drugs. This may permit the selection of an equally active regimen that does not cause the toxicity. Unfortunately, such testing remains largely an investigative strategy with still-undefined utility in routine oncology practice.
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