Deshmukh Shriya, Kelly Ciara, Tinoco Gabriel
Department of Internal Medicine, The Ohio State University, Columbus, OH, USA.
Memorial Sloan Kettering Cancer Center, New York, NY, USA.
Target Oncol. 2025 Jan;20(1):13-25. doi: 10.1007/s11523-024-01115-3. Epub 2024 Nov 15.
Chondrosarcomas, a rare form of bone sarcomas with multiple subtypes, pose a pressing clinical challenge for patients with advanced or metastatic disease. The lack of US Food and Drug Administration (FDA)-approved medications underscores the urgent need for further research and development in this area. Patients and their families face challenges as there are no systemic therapeutic options available with substantial effectiveness. A significant number (50-80%) of chondrosarcomas have a mutation in the isocitrate dehydrogenase (IDH) genes. This review focuses on IDH-mediated pathogenesis and recent pharmacological advances with novel IDH inhibitors, explores their potential therapeutic value, and proposes potential future avenues for clinical trials combining IDH inhibitors with other systemic agents for chondrosarcomas.
软骨肉瘤是一种罕见的骨肉瘤，有多种亚型，对患有晚期或转移性疾病的患者构成了紧迫的临床挑战。美国食品药品监督管理局（FDA）批准的药物的缺乏凸显了该领域进一步研发的迫切需求。由于没有具有显著疗效的全身治疗选择，患者及其家属面临诸多挑战。相当一部分（50-80%）软骨肉瘤存在异柠檬酸脱氢酶（IDH）基因突变。本综述重点关注IDH介导的发病机制以及新型IDH抑制剂的最新药理学进展，探讨其潜在治疗价值，并提出将IDH抑制剂与其他全身药物联合用于软骨肉瘤临床试验的潜在未来途径。
