Preoperative treatment is a mainstay of clinical care and research for women with breast cancer. Unique among solid tumors, the use of neoadjuvant treatment has been extensively studied in breast cancer patients, with many beneficial and provocative clinical results. It is an outstanding model for translational research, because of the accessibility of tumor tissue, and the opportunity to obtain tissue at serial time points before, during, and after treatment.
The largest challenge confronting the use of neoadjuvant therapy as a definitive model for treatment of early stage disease remains the difficulty in convincingly relating the short-term surrogate (response, pCR) with the long-term endpoint of tumor control or recurrence. In the meanwhile, the use of response/pCR as endpoints should allow novel strategies to be quickly evaluated in the neoadjuvant setting, identified approaches that clearly warrant (or not) large-scale study in randomized phase 3 adjuvant trials. Preoperative treatment also holds promise for identifying biological pathways and biological effects of new cancer drugs. Insights from such translational studies may allow for accelerated recognition of biologically relevant compounds, or pairs of compounds, that can verify or refute other laboratory-based models of cancer treatment. This information—uniquely available from preoperative studies because of the availability of tumor samples—should dramatically inform evolving clinical practice in the years ahead.
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