King's College London researchers discover cancer treatment drug could also help rescue weakened muscles in those with muscular dystrophy
Facioscapulohumeral Dystrophy (FSHD) is a progressive skeletal muscle disorder that leads to weakness in muscles of the face, shoulders and upper arms. Resulting in an impaired quality of life, the disorder affects around 1 in around 9000 of the UK population. There is no cure for this debilitating condition and despite extensive research it is still not clear what causes muscle weakness in those affected.
However a multi-faculty team between King’s College London and University College London have recently discovered a novel application of the drug Sunitinib to help reduce the weakness in the muscles affected by the condition. Their research has shown that the drug could help rescue abnormal muscle formation in cells from FSHD patients.
The team led by Dr Robert Knight from the Craniofacial Development and Stem Cell Biology Division at the KCL Dental Institute and Professor Peter Zammit of The Randall Division of Cell & Molecular Biophysics in the Faculty of Life Sciences & Medicine have found that Sunitinib, which is approved for human use in treating a variety of cancers, also has therapeutic potential in the treatment of Facioscapulohumeral Dystrophy.
Dr Robert Knight explains: “Muscle dystrophies are an untreatable collection of diseases that result in progressive loss of mobility and often premature death, and affect millions of people worldwide. The discovery that an approved anti-cancer treatment may prove useful for enhancing the ability of stem cells to repair muscle in a type of muscle dystrophy affecting face and shoulder muscles offers a new route for putative therapies for many patients.”
The results of the research have been outlined in a recently published paper on eLife. Building on this exciting find, Professor Zammit and Dr Knight are now collaborating to explore how Sunitinib can improve the ability of muscle stem cells from FSHD patients to form muscle. The research has support from King's Health Partners through the Challenge Fund.
Dr Robert Knight is a Principal Investigator at the Division of Craniofacial Development and Stem Cell Biology at King’s College London’s Dental Institute. His laboratory aims to understand the molecular and cellular events that direct stem cells during musculoskeletal development and regeneration.
Professor Zammit is a Principle Investigator in the Randall Division of Cell & Molecular Biophysics who currently investigating the control of muscle stem cells, examining mechanisms of disease and potential therapies for Emery-Dreifuss muscular dystrophy, Fascioscapulohumeral muscular dystrophy and rhabdomyosarcoma, and testing biomaterials for their ability to support muscle function.
The full paper on the research can be read on eLife here: https://elifesciences.org/content/5/e11405
More information about the Knight and Zammit research groups can be found here: