Development of a novel therapeutic for osteoporosis

骨质疏松症影响很大比例的老年人口，由于不受控制的骨质流失，经常导致骨折；50岁以上的女性中有1 / 2和男性中有1 / 5会经历骨质疏松相关的骨折。这严重影响了生活质量，20-30%的髋部骨折患者在12个月内死亡。由于副作用和长期使用相关的问题，例如非典型髋部骨折和导致感染的免疫抑制，目前尚无理想的骨质疏松症治疗方法。依从性差（由于这些副作用和诊断不足）是一个重大问题，因此全世界的治疗率不到20%。最重要的是，骨质疏松症是一种长期的疾病，需要长期的治疗，即长达几十年。因此，临床越来越需要开发新的治疗方法，提供额外的治疗选择，以便更好地管理这种常见的、使人衰弱和可能致命的疾病。我们已经确定了一种分子，它是一种有效的骨质流失抑制剂，我们建议开发一种新的治疗骨质疏松症的方法。该候选药物来源于一种在正常骨转换调节中起作用的人类蛋白质。我们预计这种蛋白质将具有有限的不良副作用（与现有疗法不同），从而使其能够长期使用（并提高依从性）。该项目将以我们的初步数据为基础，旨在建立一个经批准的绝经后骨质疏松症实验模型的最佳治疗方案；初步的安全测试也将进行。这项临床前评估将为包括人体试验在内的未来研究提供信息和促进，并帮助我们找到一个商业合作伙伴，共同开发我们的技术。

Osteoporosis, which affects a large proportion of the elderly population, frequently results in bone fractures due to uncontrolled bone loss; 1 in 2 women and 1 in 5 men over the age of 50 will experience an osteoporosis-related fracture during their lifetime. This severely impacts on quality of life and 20-30% of hip fractures lead to death within 12 months. There is currently no ideal treatment for osteoporosis due to side effects and problems associated with their long-term use, e.g. atypical fractures of the hip and immune suppression resulting in infections. Poor compliance (due to these side effects and under diagnosis) is a significant problem and consequently treatment rates are less than 20% throughout the world. Most importantly osteoporosis is a long-term condition that requires long-term therapy, i.e. up to several decades. Therefore, there is a growing clinical need for the development of new therapies to provide additional treatment options to allow better management of this common, debilitating and potentially fatal condition.We have identified a molecule that is a potent inhibitor of bone loss, which we propose to develop as a novel treatment for osteoporosis. This drug candidate is derived from a human protein that has a role in the normal regulation of bone turnover. We anticipate that this protein will have limited undesirable side effects (unlike existing therapies), thus enabling its long-term use (and improving compliance). This project will build on our preliminary data and is aimed at establishing an optimal treatment protocol in an approved experimental model of post-menopausal osteoporosis; initial safety testing will also be carried out. This preclinical evaluation will inform and facilitate future studies, including trials in humans, and help us obtain a commercial partner with whom to co-develop our technology.