Patients with cancer, who have achieved an unexpectedly favorable and
long-term clinical response are commonly known as exceptional responders (ER).
Such patients have often experienced extraordinary responses to some oncology
therapies, which have been ineffective for other individuals with similar malignancies.
These unusually positive responses may be partially due to some unique genetic and
molecular mechanisms, which can be further studied. This, in turn, could provide some
directions to a better understanding of why the specific therapy works for only a small
number of patients with cancer, but not for everybody. To further elucidate these
issues, the National Cancer Institute (NCI) has been conducting various research
projects to explain biological processes, which can be responsible for these remarkable
responses.
A recent pilot study, known as the Exceptional Responders Initiative (ERI), has
evaluated the feasibility of identifying exceptional responders retrospectively, by
obtaining pre-exceptional response treatment tumor tissues and analyzing them with
modern molecular tools. The promising findings of this study can inspire many women
with breast cancer (BC) and their medical teams.
This chapter presents a synopsis of the ERI. It suggests some possibilities to adjust this
concept for patients with breast cancer (BC) (e.g., advanced or metastatic triplenegative breast cancer (TNBC)).
Keywords: Actionable mutations, Exceptional responders, Exceptional responses, Integrated studies, Next-generation sequencing (NGS), Precision oncology.
