Yu Chuanfei, Wang Lan, Ni Yongbo, Wang Junzhi
Key Laboratory of the Ministry of Health for Research on Quality and Standardization of Biotech Products, National Institutes for Food and Drug Control No. 29, Huatuo Road, Daxing District Beijing 102629 China
RSC Adv. 2019 Dec 3;9(69):40196-40202. doi: 10.1039/c9ra07328k.
RANKL (receptor activator of nuclear factor κB ligand) plays a key role in the differentiation, activation and survival of osteoclasts. Denosumab, which targets RANKL, is approved for osteoporosis or bone loss that has a high risk for fracture and bone metastases from solid tumors. Bioactivity determination is essential for the safety and efficacy of therapeutic antibodies. At present, the mechanism of action (MOA) based bioassay for anti-RANKL monoclonal antibodies (mAbs) is the measurement of tartrate resistant acid phosphatase (TRAP) activity, which takes about five days and has complex operation and relatively high variation. In this study, we developed a reporter gene assay (RGA) based on a RAW264.7 cell line stably expressing luciferase reporter under the control of nuclear factor-κB (NF-κB) response elements. After optimizing the key parameters, the validation results based on ICH-Q2 not only show superior specificity, precision, linearity, accuracy and passage stability, but also a short duration and simple operation. These results demonstrate the RGA based on the RANKL-RANK-NF-κB pathway can be an excellent alternative for measuring the bioactivity of anti-RANKL mAbs.
核因子κB配体受体激活剂(RANKL)在破骨细胞的分化、激活和存活中起关键作用。靶向RANKL的地诺单抗被批准用于治疗具有高骨折风险的骨质疏松症或骨质流失以及实体瘤的骨转移。生物活性测定对于治疗性抗体的安全性和有效性至关重要。目前,基于作用机制(MOA)的抗RANKL单克隆抗体(mAb)生物测定法是测量抗酒石酸酸性磷酸酶(TRAP)活性,该方法耗时约五天,操作复杂且变异相对较大。在本研究中,我们基于在核因子κB(NF-κB)反应元件控制下稳定表达荧光素酶报告基因的RAW264.7细胞系开发了一种报告基因测定法(RGA)。优化关键参数后,基于国际人用药品注册技术协调会(ICH-Q2)的验证结果不仅显示出卓越的特异性、精密度、线性、准确性和传代稳定性,而且耗时短且操作简单。这些结果表明,基于RANKL-RANK-NF-κB途径的RGA可以成为测量抗RANKL mAb生物活性的极佳替代方法。