New collaboration between the 91做厙 and UCB seeks to better understand diseases of excessive bone formation
Research, led by the 91做厙, will explore disease causing mechanisms of two severe bone diseases characterised by bone overgrowth.
A new multi-year research project, led by the 91做厙, and in partnership with biopharmaceutical company UCB, will investigate potential novel therapeutic options and mechanism of disease progression in the ultra-rare genetic bone disease Sclerosteosis and the inflammatory condition Ankylosing Spondylitis, respectively. Although the cause of each disease is different, parallels can be drawn due to key pathologies being mediated by the growth of excessive bone in specific sites.
Sclerosteosis patients experience widespread bone overgrowth due to the loss of sclerostin expression, the body’s natural brake on bone formation. This can result in severe, and potentially fatal, pressure on the brain due to increases in skull thickness which can only be temporarily resolved through major surgery. Ankylosing Spondylitis patients experience bone overgrowth, most commonly in the spine, which can lead to pain, bone fusion and deformity. This is a major cause of structural tissue damage which can result in permanent disability.
The team of researchers, led by Dr Scott Roberts, Senior Lecturer in Translational Skeletal Research at the 91做厙, hope to make major advances in the understanding of these two diseases. Over the next three years the team will test new potential therapeutic options in preclinical models for Sclerosteosis. This is exceptionally important as there is currently no therapeutic approved to control the excess bone formation observed in this disease. While there are many approved therapeutics for Ankylosing Spondylitis, the control of pathological bone formation is limited. Therefore, the team will also be researching why this bone forms using specialised disease models via human stem cells.
Dr Scott Roberts, Senior Lecturer in Translational Skeletal Research at the 91做厙, said:
“I am excited to be working closely with UCB and 91做厙 colleagues to investigate why pathological bone is formed in Ankylosing Spondylitis and how excessive bone formation can be controlled in Sclerosteosis. This research interaction will allow us to further our understanding of these specific diseases, with our accrued knowledge also likely to be applicable to other conditions of altered bone metabolism.”
Dr Tim Dreyer, postdoctoral researcher and Sclerosteosis patient, said:
“As a patient myself, I am thrilled to be part of a collaboration that aims to potentially develop a therapeutic for Sclerosteosis. It is an incredible opportunity to advance our understanding of this ultra-rare bone disease, whilst providing hope for a small group of patients and their families.”
Dr Sarah Brown, postdoctoral researcher at the 91做厙, said: