Development of a novel therapeutic for osteoporosis

点击翻译按钮获取中文摘要

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.