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How a rare bone disorder inspired osteoporosis researchers

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    By any measure, the story behind UCB’s potential new medication for osteoporosis is remarkable. It features a rare genetic bone disorder, a major collaborative research effort, a space flight and – we hope – will ultimately help relieve some of the burden of bone fractures suffered by millions of people.

    Let’s start at the very beginning. In 1958, a research paper described the cases of two South African girls with rare bone disorder. Their condition, which led them to have large, abnormally dense bones and other symptoms, became known as ‘sklerosteose’ or sclerosteosis.

    Several decades later, with medical research techniques having improved dramatically, the SOST gene was discovered. This gene, which codes for sclerostin, was connected to the sclerosteosis reported in the South African cases decades earlier. It transpired that those were cases of insufficient sclerostin production.

    Further research linked sclerostin to bone fragility: when too much sclerostin is produced by the body, bones are more fragile and the risk of fracture is greater. This means that if the action of sclerostin could be reduced, bones could become stronger.

    Collaboration and clinical trials
    UCB and Amgen have been collaborating to explore the science behind bone biology and to test a new monocolonal antibody which essentially  binds to, and inhibits, sclerostin.

    Bones are constantly being remodelled, balancing bone building and bone resorption. Most existing drugs for osteoporosis seek to reduce bone resorption. The antibody we have been working on with Amgen seeks to increase bone formation and decreases bone resorption.

    This antibody – which saw UCB and Amgen partner with NASA to explore its role in redressing the bone effects of space flight – has been in clinical trials for several years. The latest results, reported at the ENDO 2016 conference and covered in a recent edition of Nature, are encouraging.

    There is more work to be done before this antibody could become available to patients with osteoporosis – a disease which causes 9 million bone fractures per year. However, this area of research stands as an excellent example of UCB’s willingness to learn from nature and partner with others on a fresh approach to tackling severe diseases.

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