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Current status of biomarker testing in historically rare, high-unmet-need tumors: soft tissue sarcomas and thyroid
Bridgette Schroader1, Sheldon Kong2, Sibyl Anderson2
1Oncology Medical Communications, Xcenda, LLC, Palm Harbor, FL, USA.
Abstract:
Introduction: The development of precision medicine and targeted therapies have revolutionized cancer treatment. Historically, treatment was chosen based on the tumor-histology, but can now be tailored to patient-specific biomarkers. Investigations have shown up to 40% of cancer patients who undergo molecular testing have an actionable biomarker with a drug currently available, and that patients benefit from these drugs. In 2018, larotrectinib became the first drug developed and approved exclusively as a tumor-agnostic therapy. Because of advancement in precision medicine, biomarker testing has become a standard of care in a variety of tumors and its use is increasing.Areas covered: We discuss the landscape of biomarker testing in the context of soft tissue sarcomas and thyroid cancer, two rare diseases with historically high unmet needs in their subpopulations. We summarize historical data and contemporary applications.Expert opinion: The paradigm shift in oncology treatment toward precision medicine, including tumor-agnostic agents, is experiencing substantial momentum but still facing challenges. A gap exists between guideline recommendations for biomarker testing and clinical application, resulting in compromised access and suboptimal outcomes. Future progress will require routine access to testing and expanding treatment options coupled with awareness, predictability, and strategies to address resistance mechanisms.
Insights
Precision medicine revolutionizes cancer care by tailoring treatments to patient biomarkers. Biomarker testing, crucial for rare cancers like soft tissue sarcomas and thyroid cancer, faces challenges in clinical application despite proven benefits.
Area of Science:
- Oncology
- Precision Medicine
- Molecular Diagnostics
Background:
- Cancer treatment has shifted from histology-based to biomarker-driven precision medicine.
- Targeted therapies and tumor-agnostic drugs offer significant patient benefits.
- Biomarker testing is increasingly standard of care, with up to 40% of tested patients having actionable targets.
Purpose of the Study:
- To review biomarker testing in rare cancers: soft tissue sarcomas and thyroid cancer.
- To summarize historical data and current applications of biomarker testing.
- To discuss the paradigm shift towards precision oncology and tumor-agnostic therapies.
Main Methods:
- Literature review of biomarker testing in soft tissue sarcomas and thyroid cancer.
- Analysis of precision medicine advancements and their impact on treatment selection.
- Discussion of clinical application challenges and future directions.
Main Results:
- Biomarker testing identifies actionable targets in a significant portion of cancer patients.
- Tumor-agnostic therapies, like larotrectinib, represent a major advancement.
- Rare cancers, including soft tissue sarcomas and thyroid cancer, have specific subpopulations benefiting from targeted approaches.
Conclusions:
- Precision medicine and biomarker testing are transforming oncology, especially for rare diseases.
- A gap persists between biomarker testing guidelines and clinical practice, impacting patient outcomes.
- Future progress requires improved access to testing, expanded treatment options, and strategies to overcome resistance.
