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Clinical challenges and therapeutic strategies in chordoma: Translating molecular insights into patient care
Beatrice Campilan1, Weston C de Lomba1, Madison J Michles1
1Department of Neurosurgery, Brown University, The Warren Alpert Medical School, Providence, RI, United States.
Abstract:
Chordoma is a rare, locally aggressive bone tumor originating from notochordal remnants. Despite advances in surgical and radiation-based approaches, effective systemic therapies remain limited, and recurrence rates are high. This chapter explores the current clinical landscape of chordoma management with an emphasis on the integration of molecular insights into therapeutic decision-making. In particular, we highlight emerging strategies targeting brachyury and receptor tyrosine kinases, as well as promising immunotherapies, including PD-1/PD-L1 immune checkpoint inhibitors, cancer vaccines, and adoptive cell therapies. We further underscore the pivotal role of institutional and multi-center biobanking efforts in enabling biomarker discovery, enhancing diagnostic accuracy, and facilitating patient prognostication. Alongside these efforts, clinical trial development and public health considerations provide a comprehensive overview of the present scope and future potential of precision medicine in chordoma. By emphasizing the translational potential of molecular profiling together with advances in the clinical, institutional, and public health infrastructures that support its implementation, this chapter outlines a pathway toward more personalized and effective care for patients with chordoma.
Insights
Chordoma, a rare bone cancer, has limited systemic therapies. Integrating molecular insights and precision medicine offers a promising pathway toward more personalized and effective patient care.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Chordoma is a rare, locally aggressive bone tumor arising from notochordal remnants.
- Current treatments like surgery and radiation have limitations, with high recurrence rates and few effective systemic therapies.
- Advances in understanding chordoma's molecular landscape are crucial for developing novel therapeutic strategies.
Purpose of the Study:
- To explore the current clinical management of chordoma, emphasizing molecular insights.
- To highlight emerging therapeutic strategies targeting key molecular pathways and immunotherapies.
- To underscore the role of biobanking and clinical infrastructure in advancing precision medicine for chordoma.
Main Methods:
- Review of current clinical landscape and molecular insights in chordoma management.
- Identification and discussion of emerging targeted therapies (brachyury, receptor tyrosine kinases) and immunotherapies (PD-1/PD-L1 inhibitors, cancer vaccines, adoptive cell therapies).
- Emphasis on the role of biobanking, biomarker discovery, diagnostic accuracy, prognostication, and clinical trial development.
Main Results:
- Limited effective systemic therapies exist for chordoma, necessitating novel treatment approaches.
- Emerging strategies targeting brachyury and receptor tyrosine kinases show promise.
- Immunotherapies, including checkpoint inhibitors and cell-based therapies, represent a significant area of development.
- Biobanking and multi-center collaborations are essential for biomarker discovery and advancing personalized medicine.
Conclusions:
- Integrating molecular profiling and precision medicine holds significant potential for improving chordoma patient care.
- Advancements in targeted therapies and immunotherapies are crucial for overcoming current treatment limitations.
- Robust clinical, institutional, and public health infrastructures are vital for translating research into effective patient outcomes.
