Neoadjuvant Immunotherapy: Leveraging the Immune System to Treat Early-Stage Disease

Abstract

Given the success of immunotherapy in treating patients with metastatic disease in a variety of tumor types, there is tremendous enthusiasm for expanding the use of immunotherapy to those with early-stage cancer. Administering immunotherapy in the neoadjuvant, preoperative setting is a biologically sound approach because preclinical studies have shown that stronger and broader immune responses can be generated if immunotherapy is administered while the tumor and/or draining lymph nodes are intact. It is therefore likely that administering immunotherapy preoperatively will generate optimal immune responses, leading to high rates of pathologic response as well as improved long-term survival. Although neoadjuvant immunotherapy is currently only approved for use in combination with chemotherapy in triple-negative breast cancer and non–small cell lung cancer, it is anticipated that ongoing and future clinical trials will further define the role of neoadjuvant immunotherapy in many cancer types. These trials should be designed with appropriate survival endpoints and rigorous correlative studies to include imaging and biospecimen-based analyses to address currently unanswered questions that must be resolved to optimize the use of immunotherapy in early-stage disease.