Biomarkers and Precision Medicine in Oncology Practice and Clinical Trials

Biomarker testing, which is now often the standard of care for patients diagnosed with cancer, can be used by physicians to assess cancer risk, diagnose a particular cancer, select treatment, and/or assess treatment response. In this summary of a keynote address, Edith Perez, Professor of Medicine at the Mayo Clinic, discusses basic concepts and issues of biomarker-based precision medicine in clinical trials and oncology practice. She describes the general features of well-designed biomarker-driven clinical trials and offers specific suggestions for designing clinical trials to support FDA approval. Perez predicts that in the near future, tumor sequencing will become standard clinical practice; liquid biopsies will become available to sample circulating tumor DNA (ctDNA); tumor classification will become molecular-based, and tumor-agnostic biomarker strategies may be used to manage patients; and clinical trials in oncology will use sequencing at both enrollment and follow-up. Additionally, she describes some basic concepts and challenges in the use of cancer immunotherapy (CIT) biomarkers, which are revolutionizing oncology. A new Program for Accelerated Cancer Therapies (PACT) was cited as an example of a multidisciplinary collaboration with NIH, NCI, and biopharma that aims to support the development of standardized biomarkers for immunoprofiling and exploratory biomarkers of high relevance to patient care. Finally, Perez explains why bringing biomarker-based trials to patients is challenging and predicts that large collaboratives such as PACT will move precision medicine and oncology forward by linking clinical retrospective and prospective cancer genomic and proteomic data with longitudinal clinical outcomes.