Neurology Residency Program, Medical College of Georgia, 1120 15th Street, Augusta, GA, 30912, USA.
Hereditary Cancer Clinic, Georgia Cancer Center, Medical College of Georgia, Augusta University, 1411 Laney Walker Blvd, Augusta, GA, 30912, USA.
Curr Treat Options Oncol. 2021 Jul 2;22(9):80. doi: 10.1007/s11864-021-00876-7.
Nervous system tumors arising in the setting of monogenic, hereditary cancer predisposition syndromes are unique in that the initiating genetic event in tumor formation is known. This knowledge provides a powerful treatment approach if the alteration or pathway can be targeted with a therapeutic agent. A reasonable argument can be made for the use of targeted agents in these tumor patients, even though many of them have FDA approval only for other tumor types. It is our practice to use and employ targeted therapy when standard treatments have failed or represent an unattractive option. Over time, however, targeted therapies will likely become first-line options.
神经系统肿瘤发生于单基因遗传性癌症易感综合征时具有独特性，因为肿瘤形成中的起始遗传事件是已知的。如果可以用治疗药物针对这种改变或通路进行治疗，那么这一知识为治疗提供了强有力的方法。尽管许多药物仅获得针对其他肿瘤类型的 FDA 批准，但可以合理地认为这些肿瘤患者可以使用靶向药物。我们的做法是，在标准治疗失败或不具吸引力时使用和采用靶向治疗。然而，随着时间的推移，靶向治疗很可能成为一线治疗选择。
