BEYOND MOONSHOTS AND METASTATIC DISEASE – Expanded Atypical Responders Analysis

Researchers in clinical oncology and other domains generally focus on results encompassing a statistical mean with little attention to patients undergoing investigational or standard therapies who respond considerably better or worse than the norm. An enormous opportunity exists to explore the reasons underlying an unusual (“atypical”) response, which would increase understanding of the mechanisms involved in cancer progression and treatment resistance, accelerate biomarker identification, and improve personalized medicine by allowing clinicians to prospectively select optimal treatments while avoiding therapies that would otherwise prove ineffective.

Understanding the reasons underlying atypical responses and exceptional survival is critical to advancing the success of personalized medicine. Therefore, the Metastatic Breast Cancer Alliance calls for:

  1. Unified categories of, and criteria for, exceptional responders, rapid progressors, and exceptional survivors
  2. Enhancing clinical trial design to study exceptional responders and rapid progressors relative to treatment outcomes. Exceptional survivors who enroll in clinical trials can be readily identified by the date of their MBC diagnosis, and specific methodologies to study these patients are needed
  3. Funding studies that expand upon the role of genetics in atypical responses by integrating other factors including CIM, co-morbidities, and the entire patient, without limiting the study to genetics
  4. Sharing standardized, de-identified patient information that is generated from these studies in a secure centralized data repository that is accessible to authorized scientists

This paper – from the same authors as the peer-reviewed opinion piece which appeared in the journal npj Breast Cancer on March 16, 2017 – summarizes published studies examining the reasons for an atypical response, new research initiatives that are exploring this topic, the strengths and limitations of these endeavors, and a call-to-action for strategic direction.