Date: Saturday, 15th June 2024, 10:20-10:45 HKT
Topic 3: Novel systemic therapeutic strategies for (even) rarer sarcomas
Speaker: Dr. Tom Wei-Wu CHEN, National Taiwan University Hospital, Taiwan
Abstract:
Systemic cytotoxic agents remain the cornerstone of soft tissue sarcoma(STS) tr eatment. However, the advent of molecularly guided therapies marks a new era in STS management. Dermatofibrosarcoma protuberans, characterized by the pathognomonic COL1A1::PDGFB fusion gene, responds well to imatinib, which inhibits the PDGF-PDGFR signaling axis. Inflammatory myofibroblastic tumors exhibit distinct gene fusions with ALK, ROS1, and NTRK, and their respective targeted therapies demonstrated successful outcomes. Epithelioid sarcoma, defined by the loss of INI-1/SMARCB1 protein, shows sensitivity to the EZH2 inhibitor tazemetostat in advanced stage patients. Additionally, the aberrant activation of the mammalian target of rapamycin (mTOR) pathway in perivascular epithelioid cell tumors (PEComa) has been addressed through mTOR inhibitors like sirolimus and everolimus. The AMPECT study further substantiates the efficacy of nab-sirolimus, a nanoparticle, albumin-bound, intravenously administered sirolimus, providing excellent outcomes for malignant PEComa patients. This lecture will delve into these molecularly targeted approaches, highlighting their clinical implications and integration into contemporary STS treatment paradigms