LAUSR

Rhabdomyosarcoma (RMS) is a cancer of skeletal muscle histogenesis and the most common soft tissue sarcoma of childhood. Despite decades of basic and clinical research, survival for patients with high-risk disease has not improved since the 1970s, remaining <30% at five years. Genomic landscape studies have identified the most frequently occurring alterations, parsing out fusion-negative RMS driven by RAS pathway mutations, and fusion-positive RMS driven by signature fusion genes including PAX3-FOXO1. However, this information has not yet translated into improved outcomes. Here, we highlight multi-institutional efforts to generate new tools for RMS precision medicine, including the NCI FusOnC2 Cancer Moonshot program to target PAX3-FOXO1, the NCI CCDI Molecular Characterization Initiative to analyze every child’s RMS tumor nucleic acids, and the application of machine learning to RMS tumor images to predict molecular subtype and improve risk stratification. Citation Format: Corinne Mary Linardic. Rhabdomyosarcoma: New research tools for precision medicine. [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2023; Part 1 (Regular and Invited Abstracts); 2023 Apr 14-19; Orlando, FL. Philadelphia (PA): AACR; Cancer Res 2023;83(7_Suppl):Abstract nr SY23-03.