Development of a multidisciplinary research team of clinicians and scientists to provide expertise in all key disciplines to tackle MND. 30 faculty staff appointments have been made including 10 professors at SITraN with positions of influence within UK and international neuroscience.
Investment in state-of-the-art facilities as well as cutting edge equipment and technologies to accelerate the pace of therapy development, including an industrial scale drug screening facility and next generation sequencing equipment (Illumina).
Detailed characterisation of MND disease subtypes, including CHMP2B, TARDBP, FUS and C9ORF72, and molecular mechanisms of motor neuron injury based on genetics and molecular pathology; identification of novel therapeutic targets.
Preclinical evaluation of therapies using a cascade of refined in vitro and in vivo experimental model systems for MND and Parkinson's, including zebrafish, mouse, fibroblasts, iPSC stem cells; development of novel functional readouts of toxicity and therapeutic benefit e.g. mitochondrial function, axonal transport, oxidative defence, glial dysfunction.
Optimisation of drug dosing and delivery in vivo, as well as improved design and delivery of gene therapies through the blood brain barrier to the central nervous system.
Pipeline of gene therapies (SMA, SOD1 MND), as well as a number of lead pharmacological compounds for disease-modifying treatments for MND (S(+)Apomorphine) and Parkinson’s disease (ursodeoxycholic acid) with in vitro and in vivo efficacy. We have successfully obtained IP protection and Orphan drug designation for the drug S (+) Apomorphine.
Development of novel assistive technologies to improve the symptomatic care and quality of life of patients e.g. a customisable neck collar, a telehealth system (TiM) and a web resource for non-invasive ventilation (www.mymnd.org.uk/myNIV). Multicentre clinical trials led by SITraN to provide evidence base for existing symptomatic treatments to support and ensure best treatment and care guidelines for patients to improve communication, breathing, nutrition management and access to specialist care , e.g. DiPALS, ProGas.
Excellent links to Clinical Neurology and the UK Clinical Research Network; establishment of a major UK MND Care and Research Centre and the first MND patient and public advisory group (SMND-RAG). As a central resource for our neuroscience research, investigators at SITraN have generated and host a biorepository for human brain and spinal cord tissue, DNA, RNA, fibroblast, CSF banks, a large repository of lymphoblastoid cell lines for several of the core diseases and more than 2000 patient genome samples.
SITraN is part of major European MND research consortia, including ENCALS, EuroMotor and MIROCALS, as well as several EU and JPND programmes including SOPHIA and STRENGTH. Collaborations with academic and industry partners worldwide include Tongji University in Shanghai, China, The King Fahad Medical City in Riyadh, Saudi Arabia and several centres in the US including Harvard University, Boston, NIH, Bethesda, Ohio State University, Biogen Idec Ltd, AstraZeneca and ALS Worldwide.
SITraN has attracted more than £17 million of new research grant funding for Motor Neurone Disease (MND), Spinal Muscular Atrophy (SMA) and other neurodegenerative diseases. Members of the institute have in the last five years contributed to more than 400 peer-reviewed original research publications in high impact journals. SITraN was a key contributor to the successful Sheffield Medical School NIHR Experimental Medicine award (£3.1 million).