Related Experiment Videos
Defining, Identifying, and Understanding "Exceptional Responders" in Oncology Using the Tools of Precision Medicine
Apostolia M Tsimberidou1, Rabih Said2, Louis M Staudt3
1From the Division of Cancer Medicine, Department of Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX.
Abstract:
Widely available molecular profiling technology, including next-generation sequencing has changed the landscape of drug development in cancer. An increasing number of clinical trials in early drug development require patient selection based on molecular alterations. Concurrently, efforts to identify molecular alterations in tumors that exhibited exceptional response after systemic treatment with standard or investigational agents have been published or are in progress. These discoveries may ultimately serve as predictive markers or "actionable mutations" for future therapies. To test the feasibility of collecting the archival tissues from proposed exceptional responder patients and successful subsequent molecular profiling, the National Cancer Institute opened a nationwide exceptional responder initiative protocol in 2014. In addition, an increasing number of exceptional responder cases have been identified and published from academia institutions. The Network of Enigmatic Exceptional Responders study uses crowdsourcing to identify exceptional responders and will molecularly profile tumors to discern molecular correlates with exceptional response. In this review, we discuss the potential role of exceptional responder molecular analysis in new biomarker discovery efforts to further advance precision medicine in oncology therapeutics.
Insights
Identifying molecular alterations in exceptional cancer responders can uncover actionable mutations. This research explores molecular profiling of these patients to advance precision oncology therapeutics.
Area of Science:
- Oncology
- Genomics
- Drug Development
Background:
- Molecular profiling, including next-generation sequencing, is transforming cancer drug development.
- Patient selection based on molecular alterations is increasingly crucial for clinical trials.
- Identifying molecular changes in exceptional responders may yield predictive biomarkers.
Purpose of the Study:
- To assess the feasibility of collecting archival tissues for molecular profiling of exceptional responders.
- To review current initiatives and research in identifying and analyzing exceptional responders.
- To discuss the role of exceptional responder molecular analysis in biomarker discovery for precision medicine.
Main Methods:
- Review of National Cancer Institute's exceptional responder initiative protocol.
- Discussion of academic institutions' efforts in identifying and publishing exceptional responder cases.
- Overview of the Network of Enigmatic Exceptional Responders study utilizing crowdsourcing.
Main Results:
- The National Cancer Institute established a protocol in 2014 to test tissue collection feasibility.
- Numerous exceptional responder cases have been identified and published by academic institutions.
- Ongoing studies aim to molecularly profile tumors to find correlates with exceptional response.
Conclusions:
- Molecular analysis of exceptional responders holds significant potential for discovering new biomarkers.
- These biomarkers can drive advancements in precision medicine for cancer therapeutics.
- Further research is essential to translate these findings into clinical practice.