Precision Medicine in Sarcoma: Integrating Genomics, Patient Advocacy, and Regulatory Innovation

Sarcomas represent a heterogeneous group of rare malignancies arising from mesenchymal tissues, accounting for approximately 1% of adult cancers and 15% of pediatric cancers. Despite advances in chemotherapy and radiation, survival outcomes remain suboptimal, with 5-year survival rates stagnating at 60–70% in many sarcoma subtypes. Precision medicine—leveraging genomic profiling, molecular biomarkers, and individualized treatment strategies—offers transformative potential for sarcoma management. Recent discoveries in sarcoma biology, including recurrent translocations (e.g., EWSR1-FLI1 in Ewing sarcoma) and actionable mutations, have enabled targeted therapies and immunotherapies. However, translating these advances into clinical practice remains challenging, particularly in rare entities. Equally important is ensuring that precision medicine initiatives center patient needs, incorporate patient advocacy input, and navigate the evolving regulatory landscape to accelerate drug approvals and equitable access to precision therapies.This Research Topic aims to advance precision medicine in sarcoma by addressing the clinical, translational, and stakeholder dimensions necessary for meaningful implementation. We seek to showcase how genomic profiling can stratify patients, predict treatment response, and improve outcomes across sarcoma subtypes—from well-characterized entities (Ewing sarcoma, rhabdomyosarcoma) to orphan tumors. Beyond the science, this Research Topic emphasizes the critical voice of patient advocacy and lived experience in shaping research priorities and treatment decision-making. Patients and advocacy organizations bring invaluable perspective on burden of illness, treatment tolerability, and what outcomes matter most. Simultaneously, we address regulatory pathways—including accelerated approval mechanisms, breakthrough designations, and adaptive trial designs—that can expedite patient access to precision therapies while maintaining rigorous safety and efficacy standards.The overarching goal is to create a multidisciplinary dialogue that integrates genomic science, patient-centered perspectives, and regulatory innovation, demonstrating how precision medicine can be implemented equitably and responsively in the sarcoma community.We welcome Original Research, Reviews, Clinical Trials, Perspectives, and Case Reports addressing:• Genomic and molecular advances: Whole genome/exome sequencing, transcriptomics, and biomarker discovery in sarcoma subtypes; liquid biopsy applications and minimal residual disease monitoring.• Targeted and immunotherapies: Efficacy of CDK4/6 inhibitors, tyrosine kinase inhibitors, immune checkpoint inhibitors, and CAR-T cell therapies in precision medicine frameworks.• Patient perspectives and advocacy: Qualitative research on patient priorities; co-design of trials with patient advocates; patient-reported outcomes in precision oncology.• Regulatory pathways: Real-world evidence, adaptive trial designs, pediatric and rare disease regulatory incentives, and health equity considerations.• Implementation science: Barriers to precision medicine adoption; cost-effectiveness analyses; strategies for equitable access in low- and middle-income settings. Topic editors: Alice Chen, Denise K Reinke, Kilian Salerno Source: https://www.frontiersin.org/research-topics/83308/precision-medicine-in-sarcoma-integrating-genomics-patient-advocacy-and-regulatory-innovation