LifeArc and CSO work together to drive Scottish research into rare diseases
LifeArc and the Chief Scientist Office (CSO) have today jointly awarded a total of £300,000 to researchers at The University of Edinburgh to progress their exciting research towards helping people living with rare and complex health conditions.
Three research grants have been awarded to help tackle some of the biggest health challenges, boosting Scottish life science research. The projects include research into new treatments for motor neurone disease (MND), a group of conditions that affect nerves in the brain and spinal cord; malignant pleural mesothelioma, a rare cancer of the lining of the chest cavity linked to asbestos exposure; and Duchenne muscular dystrophy (DMD), a genetic disease that usually affects boys in early childhood and causes progressive muscle wasting and weakness.
Professor Tom Gillingwater, who is leading the MND project, is aiming to develop new drugs for treating certain types of MND: amyotrophic lateral sclerosis (ALS) that causes rapid loss of muscle control and eventual paralysis – and spinal muscular atrophy (SMA), a genetic disease that affects children. The research team’s approach involves targeting an enzyme called UBA1, which has previously been shown to have considerable potential for treating these conditions.
Dr Carsten Hansen and his team are searching for new drug compounds that could be developed into new personalised treatments for mesothelioma. Sadly, the disease is very difficult to treat, and more than half of patients will lose their lives within one year of diagnosis. They are aiming to find potential drug compounds that target a key cellular communication cascade – the Hippo pathway – which is faulty in around four out of ten mesotheliomas.
Professor Colin Farquharson is carrying out laboratory experiments to investigate whether a new drug can help prevent bone fractures in young people with DMD and explore the best strategies for using it in combination with existing medicines.
Around one-half of children with the condition lose their ability to walk after their first long-bone fracture. Spinal fractures are also common and can lead to ongoing back pain that affects their quality of life. The results of the study will set the stage for future clinical trials to find out whether this medicine can provide a safe, effective, and convenient new treatment for children with DMD.