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We translate mechanistic insights to first in human trials of disease-modifying therapies and on to later phase trials and to clinical practice. Multi-disciplinary research programmes covering genetics, molecular cell biology, transgenic and knock in mouse models, neuroimaging, biomarkers, neuropathology, systems neuroscience and experimental medicine provide an interrelated bench-to-bedside research portfolio.

We are centrally involved in the major global consortia for genetic analysis, including the International PD Genetic Consortium and the European Alzheimer’s disease Sequencing Consortium.

Our research on prion diseases takes advantage of nationally unique specialised facilities for studying human and animal prions and pathogenic protein assemblies in other neurodegenerative diseases, notably in Alzheimer’s and other dementias.

Major future strategic priorities for our research on prion diseases include:

• atomic resolution structures of prion strains and deducing the molecular basis of prion strain diversity;
• characterisation of neurotoxic PrP species in prion disease;
• determination of the role of epigenetic factors in prion disease;
• development of sensitive cell-based bioassays for human prion strains.

In 2018 the MRC Prion Unit became incorporated into Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø as the Institute of Prion Diseases and relocated to the newly refurbished Courtauld Building (a joint £30M investment by the MRC and Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø) providing a national centre of excellence with all necessary facilities to pursue a major long-term research strategy in prion and related diseases and protein misfolding research. The Institute operates at the clinical interface, running the internationally unique NHS National Prion Clinic and National Prion Cohort study, with major translational programmes developing new diagnostic tests, clinical scales, blood, CSF and imaging biomarkers and rational therapeutics (both small molecules and immunotherapeutics).

Our research in neuroinflammation is focused on neuroinflammatory disease, principally multiple sclerosis (MS), but including Alzheimer’s disease, diabetic neuropathy, and iron insufficiency. Our overall strategy is to understand the pathophysiology of neuroinflammatory diseases and develop novel therapeutic strategies in our basic science, which are then examined in investigator-led clinical trials.

One major line of research explores sodium channel blocking agents as neuroprotective therapies for MS and the second investigates therapies to restore oxygenation of the inflamed CNS, focussing on the use of vasodilating agents to achieve protection from demyelination. Another important research avenue is an investigation of human neuroinflammatory and neurodegenerative diseases using human iPSC-derived microglia.

Our strategic plan for the next five years is to bring expertise in immunology, inflammation and innate immunity to Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø’s wider community of neuroscientists.

Our basic and clinical research in neuromuscular disease combines genetic, functional cell biology and neuropathological methods to understand and develop novel therapies.

Research ranges from spinal cord motor circuits and motor neuron physiology, systems neuroscience, muscle disorders including Inclusion Body Myositis, mitochondrial disease and muscle ion channel disorders, peripheral nerve disease, and a number of neurodegenerative diseases including ataxia. We have particular strength in motor neuron diseases – including amyotrophic lateral sclerosis and Kennedy’s Disease.

Our research centres include the new MRC International Centre for Genomic Medicine in Neuromuscular Diseases, which aims to create a transcontinental genomics research capacity in neuromuscular diseases, building partnership between the UK and Brazil, India, South Africa, Turkey and Zambia.

2019 saw the launch of the Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø Queen Square Centre for Motor Neuron Disease, which brings together world-leading Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø research groups spanning the spectrum of basic discovery science, experimental medicine and clinical research.

Our research portfolio in this area covers the complete pipeline from basic science discovery to clinical trials as well as population studies and health service research. The major goal of our experimental research is the elucidation of the cellular and molecular mechanisms underlying paroxysmal brain disorders leading to the development of new treatments.

Key infrastructure for this work area includes a range of cutting edge experimental techniques, such as time-resolved fluorescence imaging, tri-dimensional, direct stochastic optical reconstruction microscopy, optogenetics and the full clinical phenotyping tool including MRI, fMRI PET, SPECT, Stereo-EEG, OCT, EEG-video telemetry, TMS, visual field perimetry, genomic analysis, facial stereography and therapeutic drug monitoring.

Key strategic priorities over the next period include several major lines of research on Parkinson disease:

• genetic causes and pathogenesis identification of novel biochemical targets for disease modification and neuroprotection;
• clinical trials of novel disease modifying therapies;
• identification of prodromal features in genetically stratified and idiopathic forms of the disease;
• and characterisation of its non-motor features and their measurement, evolution and treatment.

Our research in brain repair and rehabilitation covers the clinical themes of stroke, neuroradiology, neurorehabilitation, spinal cord repair, autonomic neurology, uro-neurology, neurosurgery, headache, and high-dimensional neurology. We aim to translate neuroscience and mechanistic findings into precision medicine to improve clinical care (diagnosis, treatment, neurorehabilitation).

Key future ambitions are:

• to develop the application of high-dimensional modelling with products deployed in clinical use, strengthening digital infrastructure within our partner hospitals and facilitating clinical research at previously impossible data scales;
• to increase the provision and effectiveness of practice-based therapy for patients with cognitive impairments due to acquired or progressive brain injury;
• to continue to deliver practice-changing randomised controlled trials and observational studies in stroke treatment/prevention;
• to strengthen strategic collaboration with Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø Computer Science to expedite machine learning technology applied to neuroradiology.

Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø is making a major investment (~£280M) in the creation of a new state-of-the-art neuroscience facility on Grays Inn Road for the Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø Queen Square Institute of Neurology, the UK Dementia Research Institute headquarters and the Ïã¸ÛÁùºÏ²ÊÖÐÌØÍøH National Hospital for Neurology and Neurosurgery. The facility will bring together research scientists, clinicians and patients to create a world-leading hub for dementia and neurological disease research. The building has been designed to encourage interdisciplinary and collaborative research through the sharing of space, equipment, core facilities and laboratory processes. It will foster new models of open innovation and will support effective engagement with industry, patient organisations, patients, the public, policy makers and funders. 

In 2016, Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø was chosen as the headquarters of the UK Dementia Research Institute (UK DRI), which is the single biggest investment the UK has ever made in dementia thanks to £290M from founding funders the Medical Research Council, Alzheimer’s Society and Alzheimer’s Research UK. The UK DRI brings together world-leading expertise in biomedical, care and translational dementia research in a national institute currently made up of over 350 researchers, plus over 150 students and a support team of over 50, seeking to improve early diagnosis and prevention. The Institute carries out research relevant to all dementias, including Alzheimer’s disease, Parkinson’s disease, frontotemporal dementia, vascular dementia, Huntington’s disease and beyond.

The Sainsbury Wellcome Centre for Neural Circuits and Behaviour, funded by the Gatsby Charitable Foundation and the Wellcome Trust, opened in 2016 and substantially strengthened Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø research in systems-level neuroscience. Neuroscientists working in the new facility use state-of-the-art molecular and cellular biology, imaging, electrophysiology and behavioural techniques, supported by computational modelling, to investigate how brain circuits process information to create neural representations and guide behaviour. A key strength of the Centre is the fact that it includes the Gatsby Computational Neuroscience Unit, which combines research in theoretical neuroscience and research in machine learning.

Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø was chosen as the founding academic partner of the Francis Crick Institute (formerly the UK Centre for Medical Research and Innovation), which opened in 2015 under the Directorship of Nobel laureate Prof Sir Paul Nurse. With funding of £750M, its mission as an interdisciplinary medical research institute is to understand why disease develops and to find new ways to prevent and treat major medical disorders.

The Ïã¸ÛÁùºÏ²ÊÖÐÌØÍø Drug Discovery Institute was launched in 2015 with funding from Alzheimer’s Research UK of £10M for the first five years, recently renewed until 2025 with a further £12M funding. It has also been awarded additional funding of over £1.3M from the Dementia Discovery Fund, and has established a new company (AstronauTx) to develop therapies for Alzheimer’s disease. The Institute works alongside Institutes at the Universities of Oxford and Cambridge to create one of the largest coordinated efforts to translate academic research into new therapeutics for dementia and neurodegenerative diseases.