Development of an artificial ligament for knee repair
Professor Molly Stevens, Imperial College London
Ligaments are bands of tissue, that hold bones or cartilage together, to ensure joints are stable. Damage to ligaments can be caused by sports injuries, or osteoarthritis. The problem with ligament injury is that they can’t repair very well, and may require surgical reconstruction to fix it. Often, this requires taking a ligament from another part of the body to repair the damaged one, which is not ideal. The development of artificial ligaments for repair would provide a much better solution. In this project, Professor Steven’s team are trying to develop an artificial ligament that could be implanted in to the body with minimal side effects.
Investigating the effects of Arimoclomol on muscle, brain and spinal cord pathology in Inclusion Body Myopathy
Professor Linda Greensmith, University College London
Inclusion Body Myositis (IBM) is an untreatable and disabling condition that is the commonest acquired muscle disease affecting people over 50 years old. Part of the reason for limited success in treating IBM to date is a poor understanding of the disease mechanism. Previous work by Professor Greensmith’s team investigated the use of a drug called Arimoclomol in model of IBM with frontal temporal dementia, and found it to be highly effective reducing loss of muscle strength and other symptoms of IBM. In light of these findings, the research team are now trying to understand the mechanism by which Arimoclomol improves IBM-like symptoms in order to move the study closer to the clinic. As the mouse ages it also displays characteristics of Frontotemporal Dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS) in the brain and spinal cord. This study therefore aims to assess the effects of Arimoclomol on ALS and FTD like-characteristics of the mouse in the brain and spinal cord, and to further understand the mechanism underlying IBM. This study is crucial to moving arimoclomal to the clinic.
Development of new therapies for fascioscapulohumeral muscular dystrophy (FSHD)
Professor George Dickson, Royal Holloway University of London
FSH-MD is one of the most common hereditary muscle disorders, affecting one individual in 14,000. Symptoms include progressive weakening and loss of muscles in the face, shoulders and arms, with patients also experiencing difficulty walking. At present there is no cure or effective treatment to relieve the symptoms of FSHD.
The aim of Professor George Dickson from the Royal Holloway, University of London, is to develop a drug to target the gene responsible for FSH-MD. The DUX4 gene is usually switched off in humans, but is switched on in people with FSH-MD, producing a protein that is toxic to muscles. Professor Dickson will develop an ‘antisense oligonucleotide’ (AO) drug, that produces its therapeutic effect by targeting and suppressing products of the DUX4 gene, switching it back to the healthy, inactive state. The patient benefit from such a drug could be life-changing with the potential to suppress and even reverse muscle wasting.
Parathyroid Hormone Enhances Bone Formation Around a Stem Cell Coated Implant
Professor Gordon Blunn & Liza Osagie, University College London
The aim of Liza Osagie’s PhD project is to improve outcomes for patients receiving hip replacements, so that there is less need for revision surgery afterwards. She will assess the benefit of coating implants using a novel technique, to improve their adherence at the bone interface and by coating them with stem cells enable natural bone cells to develop to anchor the implant. With the average age of hip replacement of 68 and a life expectancy of 80+, 2 or 3 follow-up replacements are often needed. This research should reduce the number of revisions needed.
Generating cartilage from human embryonic stem cells to treat osteoarthritis
Dr Franchesca Houghton, University of Southampton
Osteoarthritis (OA) affects around 8. 5million people in the UK, and is due to damage to the cartilage covering joints, which leads to inflammation, pain and destruction of the joint. In severe conditions, joint replacement is the final treatment option; however, surgery is not always successful, highlighting the need for alternative treatment strategies. One possible treatment option is to develop new cartilage from human stem cells. Harvesting stem cells from the bone marrow of adults with OA is painful and limited by the few cells that can be collected as well as the inferior quality of the cartilage grown from these cells. Therefore, it is important to develop an alternative source of stem cells. In this study, Dr Houghton’s team are investigating human embryonic stem cells derived from the amniotic fluid. These cells can grow indefinitely, and do not cause an immune response – reducing the likelihood of adverse effects. The researchers hope to develop and test cartilage generated from these stem cells, which could lead to cartilage grafts, reducing the need for joint replacement surgery.