Zhang Wenchao, Qi Lin, Xu Haodong, Yin Chi, Yu Zhuowen, Xu Ruiling, Feng Chengyao, Ren Xiaolei, Tu Chao, Li Zhihong
Department of Orthopedics, The Second Xiangya Hospital, Central South University, Changsha, China.
Hunan Key Laboratory of Tumor Models and Individualized Medicine, The Second Xiangya Hospital, Changsha, China.
Oncogene. 2025 Mar;44(7):427-438. doi: 10.1038/s41388-024-03205-y. Epub 2024 Nov 19.
Osteosarcoma is a common primary malignant bone tumor in children and young adults, with limited progress in improving survival rates for metastatic or recurrent cases. Kinase inhibitors have emerged as potential treatments for osteosarcoma due to the critical role kinases play in regulating cellular networks. However, single-agent kinase inhibitors often face challenges due to the activation of compensatory oncogenic signaling pathways, which can undermine treatment efficacy. In this study, a combination screening of FDA-approved kinase inhibitors was conducted in osteosarcoma cells. We identified the combination of ALK inhibitor and FLT3 inhibitor as a potent kinase-based therapeutic strategy for osteosarcoma. Our results showed that the combinatorial treatment synergistically suppressed osteosarcoma in cell lines, patient-derived organoids, and xenograft models. Mechanistically, the inhibition of FLT3 significantly promoted the activation of ALK, which subsequently enhanced its downstream PI3K/Akt and MAPK signaling pathways. The combinatorial use of an ALK inhibitor could reverse this process. Thus, our study demonstrates that the cooperative blockade of FLT3 and ALK synergistically suppresses osteosarcoma, providing a potential alternative for its treatment.
骨肉瘤是儿童和青少年常见的原发性恶性骨肿瘤,在提高转移性或复发性病例的生存率方面进展有限。由于激酶在调节细胞网络中起关键作用,激酶抑制剂已成为骨肉瘤的潜在治疗方法。然而,单药激酶抑制剂由于代偿性致癌信号通路的激活,往往面临挑战,这可能会削弱治疗效果。在本研究中,我们在骨肉瘤细胞中进行了FDA批准的激酶抑制剂的联合筛选。我们确定ALK抑制剂和FLT3抑制剂的联合使用是一种有效的基于激酶的骨肉瘤治疗策略。我们的结果表明,联合治疗在细胞系、患者来源的类器官和异种移植模型中协同抑制骨肉瘤。从机制上讲,抑制FLT3显著促进ALK的激活,随后增强其下游PI3K/Akt和MAPK信号通路。ALK抑制剂的联合使用可以逆转这一过程。因此,我们的研究表明,联合阻断FLT3和ALK可协同抑制骨肉瘤,为其治疗提供了一种潜在的替代方案。