Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, United States.
Developmental Therapeutics Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, MD, United States.
Front Endocrinol (Lausanne). 2021 Aug 19;12:708949. doi: 10.3389/fendo.2021.708949. eCollection 2021.
Medullary thyroid carcinoma (MTC) is a rare neuroendocrine tumor that accounts for 2-4% of all thyroid cancers. All inherited MTC and approximately 50% of sporadic cases are driven by mutations in the arranged during ransfection ( proto-oncogene. The recent expansion of the armamentarium of RET-targeting tyrosine kinase inhibitors (TKIs) has provided effective options for systemic therapy for patients with metastatic and progressive disease. However, patients that develop resistant disease as well as those with other molecular drivers such as RAS have limited options. An improved understanding of mechanisms of resistance to TKIs as well as identification of novel therapeutic targets is needed to improve outcomes for patients with MTC.
甲状腺髓样癌（MTC）是一种罕见的神经内分泌肿瘤，约占所有甲状腺癌的 2-4%。所有遗传性 MTC 和大约 50%的散发性病例都是由转染（原癌基因排列引起的。最近，RET 靶向酪氨酸激酶抑制剂（TKI）的治疗手段不断增加，为转移性和进展性疾病患者的系统治疗提供了有效的选择。然而，对于那些出现耐药性疾病的患者以及那些存在其他分子驱动因素（如 RAS）的患者，选择有限。为了改善 MTC 患者的预后，需要更好地了解 TKI 耐药的机制，并确定新的治疗靶点。
