NIHRDataInsights/HRCSData huggingface dataset:
Title: Drug product production, clinical support studies, and phase 1 trial of a new MS drug
Funder: Innovate UK

Abstract: We have invented a new, first in class, drug for the treatment of multiple sclerosis (MS). This drug has the potential to become the standard of care for the treatment of muscle spasticity in MS with fewer and less severe side effects for patients than current drugs. In particular earlier treatment should be achievable, countering the onset of disability and allowing a normal life to be sustained for longer. TSB funding will enable the first clinical studies for this drug. Part funding has already been secured from the Wellcome Trust, and UCL business. The first volunteer is expected to be dosed in Q3/Q4 2013.


Research activity codes: 6.1 PHARMACEUTICALS
Health categories: NEUROLOGICAL
Title: Pre-clinical proof of concept for a new agent in the treatment of ALS
Funder: Innovate UK

Abstract: Chronos Therapeutics Ltd, a company spun out of The University of Oxford, has received a grant to study its lead compound RDC5 in the motor neurone disease: Amyotrophic Lateral Sclerosis or ALS. RDC5 has shown very promising results in the company's ChronoscreenTM and in their models of other diseases involving accelerated cell death and neurodegeneration. The grant will enable Chronos to develop models of ALS and test RDC5 in these models. If, as expected RDC5 has positive results in the grant-funded experiments, Chronos expects to raise new finance to move rapidly to clinical trials in ALS with the hope to bring another treatment option to patients with this devastating disease.


Research activity codes: 5.1 PHARMACEUTICALS
Health categories: NEUROLOGICAL
Title: Novel Engineered Living Neural Tissue, for Peripheral Nerve Repair
Funder: Innovate UK

Abstract: We plan to develop a novel ‘living nerve growth guide’ as an off the shelf therapy to treat peripheral nerve injury, which can cause lifelong pain and disability and is a significant unmet medical need. Our therapy would substitute for an autograft, the current ‘gold standard’ for peripheral nerve repair. Partners TAP Biosystems and The Open University have already shown that combining their novel tissue engineering technologies enables robust, self-aligning cellular tissues to be made from collagen. These mimic the structure of peripheral nerve and support neuronal regeneration in vivo. In this project we will build on initial work using partner ReNeuron’s clinical grade neural stem cells. We will develop production tools, optimise the process, characterise the properties of our living nerve growth guide and then carry out efficacy tests of nerve regeneration. Success in the project will provide initial proof of concept data, so that this novel therapy can progress towards the clinic.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: NEUROLOGICAL
Title: Developing a novel non-invasive aid for early diagnosis of Parkinson's Disease
Funder: Innovate UK

Abstract: The project will develop and make market-ready an innovative user-friendly, low cost tool, the Manus system, to aid in the differential diagnosis of Parkinson’s disease (PD), based around a sensor-pen that uses drawing and handwriting motion analyses to provide objective information to clinicians. The device may be used by non-specialists with minimal training to enable decentralized healthcare and screening to improve the capacity of effective and early diagnosis and intervention by neurology clinics. Improvements will be made to the existing prototype hardware and mathematical algorithms with diagnostic capability by Manus. This will take place in conjunction with Prof. Walker and colleagues at North Tyneside General Hospital, who will perform a clinical assessment of the utility of the platform and comparison with standards (DaTSCAN and diagnosing by neurologist). Prior to commencing the trial, ethical approval and MHRA approval for use in trial will be obtained.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES;4.2 EVALUATION OF MARKERS AND TECHNOLOGIES
Health categories: NEUROLOGICAL
Title: Developing a Continuous Non-invasive Intracranial Pressure Monitoring Device
Funder: Innovate UK

Abstract: The human skull forms a rigid box. The pressure inside this box is normally fairly low. In many brain disorders this pressure can increase leading to headaches and, in more severe cases, confusion coma or death. Examples of disorders which cause raised pressure inside the head include head injury, a brain haemorrhage, or a build up of fluid on the brain (hydrocephalus) as well as many others. It is therefore essential to be able to recognise increased pressure inside the head in order to provide early effective treatment. Currently the only effective way to measure pressure is to perform an operation to drill a hole through the skull and to insert a probe or a tube into the brain. Aside from the obvious discomfort of the procedure it carries significant risks such as infection or bleeding in the brain and it is therefore only performed in the most severe cases. We have been working to develop a way to measure the pressure inside the head without the need to open the skull and insert something into the brain. Any specialist in Neurosugery, Emergency Medicine or even General Practice will tell you that this would be an extremely useful device. That is because it will save those currently needing an operation, to measure the pressure, the discomfort and risks of this. Perhaps, even more importantly, it will provide a way, in people with much less severe symptoms, of differentiating between a common headache that is likely to pass or something more serious that requires treatment. Our aim is to make it possible to measure the pressure inside the head just as easily as we can measure blood pressure. We have already developed a prototype method. Now we are making it easier to use and testing exactly how well it works.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES
Health categories: NEUROLOGICAL
Title: Dementia Diagnosis: A tool for healthcare and pharma
Funder: Innovate UK

Abstract: There are currently no drugs for Alzheimer’s Disease, AD, available that stop or cure the disease. Results from recent clinical trials, where the drug has been shown not to work, suggest that many of those enrolled in the trial did not have AD at the start. While patients with AD are expected to deteriorate over the course of a trial in terms of their memory recall and ability to look after themselves, many enrolled subjects did not show this deterioration. The inclusion of a large portion of patients who were not declining confounds the results and possibly hides any real affect of the drug. The difficulty in diagnosing patients correctly is not just a problem for drug trials. Patients visiting their GP are even less likely to get an accurate diagnosis as the patient visit in trials is more thorough than in primary care, or even than in most memory clinics. A correct diagnosis can have significant positive impact on patients even in the absence of drugs but will be critical to ensure patients get early access to drugs as they become available. In this project, we will develop a dementia prognosis tool that uses data collected in clinics (MRI scans, cognitive tests, demographic data), to help diagnose patients by predicting their outcome and expected rate of decline. We have demonstrated proof-of-principle on a relatively small amount of data and in this project we will work closely with some large pharmaceutical companies and Imperial College to develop better methods and test it on many 1000s of patients from a global population including 100s of patients from each of N America, Europe, China, Australia, Japan.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES;4.2 EVALUATION OF MARKERS AND TECHNOLOGIES
Health categories: NEUROLOGICAL
Title: Imaging Informatics for Early Diagnosis of Dementia in Chinese Elderly Population
Funder: Innovate UK

Abstract: Timely and accurate Dementia diagnosis is a huge unmet need globally. As the population ages, the health and social care costs will rapidly become unsustainable. China may become the world's largest market for dementia diagnosis: China is estimated to have 25% of the world's dementia cases but ~80% of the dementia population remain undiagnosed, and the one child policy means individuals may have 4 grand parents to care for. While there are no cures for dementia, there are effective drug and non-drug interventions which can extend independent life and save society costs. There is a pressing need for technology to support diagnosis in a community setting if China is to make these treatments available to the patients that need it. This feasibilty award enables IXICO to explore the opportunity to create a focused product which is adapted to the Chinese population and healthcare system and identify the partners needed to enter this market.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES
Health categories: NEUROLOGICAL
Title: Optimising nutrition to improve the growth and reduce neuro-disability in young children with cerebral palsy
Funder: SPARKS

Abstract: Known as the Dolphin Study, this is a two year double-blind randomised controlled trial. It is investigating whether dietetic and nutritional intervention with neurotrophic supplementation improves brain growth and neurodevelopmental outcomes in young children with a suspected or confirmed clinical diagnosis of Cerebral Palsy.


Research activity codes: 3.3 NUTRITION AND CHEMOPREVENTION
Health categories: NEUROLOGICAL
Title: The introduction of Petra running-bikes to encourage and facilitate weight-bearing exercise for children with cerebral palsy who are unable to walk independently: A pilot study
Funder: SPARKS

Abstract: Children with CP, unable to walk independently, have limited opportunities to participate in weight-bearing exercise. This study will introduce supported running-bikes to twelve children to ascertain if it is a feasible/enjoyable mode of exercise facilitating their participation in physical activity. Children aged 4-12 will trial the running-bikes, individually adapted for their requirements, for six weeks. At the end of the trial the physiotherapists and children will be interviewed to find out their views and experiences of using the running-bike. Any changes in bone status, gross motor function, quality of life and ability to use the running-bike will be investigated.


Research activity codes: 7.1 INDIVIDUAL CARE NEEDS
Health categories: NEUROLOGICAL
Title: Combination therapies for juvenile Batten Disease
Funder: SPARKS

Abstract: Batten disease attacks the brain, but also activates the immune system, causing more harm. We have recently shown that suppressing this response has positive effects in mice, but they still remain ill. We shall now test whether commonly used anti-inflammatory drugs or those that protect brain cells will be any better. Different combinations of these drugs will be screened, alone or together with immunosuppression, to find the best means to treat diseased mice. By evaluating drugs that are already used in children we will be able to move more quickly to clinical testing.


Research activity codes: 5.1 PHARMACEUTICALS
Health categories: NEUROLOGICAL
Title: UK Multicentre Study of Children with Opsoclonus Myoclonus Syndrome (UMSCOM) (UK arm of Multinational European Trial for Children with Opsoclonus Myoclonus Syndrome
Funder: SPARKS

Abstract: Opsoclonus myoclonus syndrome (OMS) or Dancing eye syndrome (DES) is a rare childhood disease of the nervous system. It is often associated with neuroblastoma, a common tumour of childhood. This will be the first large scale study of the disease in the UK and EU. Recruited children will follow a systematic programme of drug therapy that will be modified according to response and will be carefully followed to up to school entry to identify their strengths and difficulties. The results of the study will provide better treatment and management of children with OMS and will launch further treatment trials.


Research activity codes: 6.1 PHARMACEUTICALS
Health categories: NEUROLOGICAL
Title: Neural stem cells as therapeutic targets in germinal matrix haemorrhage
Funder: SPARKS

Abstract: As a consequence of improved survival, the prevalence of neurological disability associated with premature birth continues to increase. Haemorrhage within the germinal matrix (GMH) has been shown to affect stem cell function and this mechanism is thought to contribute to the developmental abnormalities seen. In this project, we will test the hypothesis that fine tuning of cellular properties of neural stem/progenitor cells (NSPC) is a viable approach to improve neurogenesis and/or oligodendrogenesis in a mouse model of GMH. Autologous blood is injected into the sub ventricular zone (SVZ) to mimic the effect of GMH on the developing neocortex. Stereological quantitative assessment of layer thickness and cellular composition is then used to determine the pathological effect of GMH, in combination with daily behavioural analysis. To analyse the contribution of the SVZ NSPC to recovery following GMH, genetically engineered mice in which the self renewal properties of NSC are either suppressed or enhanced have been generated and extensively characterised in the host laboratory. Further we will test whether enhancing SVZ NSPC activity through injection of exogenous NSC into the injured SVZ has a similar effect. Correlation with findings on human autoptic material of GMH/IVH neonates will be carried out in collaboration with dr D. Rowitch, Department of Neurological Surgery, UCSF.


Research activity codes: 5.2 CELLULAR AND GENE THERAPIES
Health categories: NEUROLOGICAL
Title: Is Batten disease primarily caused by a deficiency in free amino acids or energy?
Funder: SPARKS

Abstract: The health of the central nervous system is particularly affected by a reduction in available energy and metabolites. Defects in mitochondria and lysosomes, important for ATP generation and amino acid recycling, have been implicated in a variety of neurodegenerative diseases, including Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL), a paediatric progressive neurodegenerative disease that is the focus of this research. We hypothesise that low levels of ATP and amino acids contribute substantially to the pathology seen in LINCL. We will use the validated zebrafish model of this disease to test this hypothesis and determine if either amino acid supplementation or upregulation of mitochondrial ATPsynthase Inhibitory Factor 1 (IF1) function via a compound we have designed ameliorates the neurodegenerative phenotype of the zebrafish model of LINCL and therefore has potential for treating affected children. Therefore, this project not only has the potential to identify pathological disease mechanisms but also to identify potentially therapeutic strategies. If successful, we will apply for funding to perform trials in the mouse and canine models of LINCL with collaborators.


Research activity codes: 2.1 BIOLOGICAL AND ENDOGENOUS FACTORS
Health categories: NEUROLOGICAL
Title: Improvements in neuromodulation for late-stage Parkinson's disease
Funder: Innovate UK

Abstract: This project is a study of a new type of "brain pacemaker" used for treatment of Parkinson’s disease, the second most common neurodegenerative disorder after Alzheimer's disease. Advanced-stage patients suffer from the highest unmet medical need, with only 36% being adequately controlled by drugs and many suffering severe side-effects. The most effective and popular surgical treatment for advanced-stage Parkinson’s disease is deep brain stimulation, but at present under 5% of patients receive this treatment. This new device enables stimulation to be activated in response to feedback from the brain thereby improving the targeting of therapy delivery. The device and procedure have a number of innovations that potentially cut surgical time in half, reducing costs and improving access to the procedure. The objective is to help improve the quality of life of many more Parkinson’s sufferers and their carers.


Research activity codes: 6.3 MEDICAL DEVICES
Health categories: NEUROLOGICAL
Title: Clinical Development of OXB-102
Funder: Innovate UK

Abstract: Parkinson's disease is caused by the degeneration of nerve cells in part of the brain; leading to the loss of dopamine, a chemical messenger which plays a vital role in coordinating body movement. In early stages of PD, oral levadopa (L-DOPA) medication is effective in managing the symptoms that include tremor, muscle stiffness and slow physical movement. However, the body progressively loses its ability to convert L-DOPA to dopamine thereby reducing its effectiveness and leading to the development of uncontrolled motor function. Oxford BioMedica has developed a 'once-only' gene therapy approach to treat individuals with PD called OXB-102 that is administered once to the target region in the brain where it converts cells into a replacement dopamine factory. In essence, OXB-102 replaces a patient’s own lost source of the neurotransmitter analogous to the natural dopamine supply in the absence of PD.


Research activity codes: 6.2 CELLULAR AND GENE THERAPIES
Health categories: NEUROLOGICAL
Title: A blood test for Alzheimer's disease - diagnosis and monitoring.
Funder: Innovate UK

Abstract: Dementia is a growing problem with 800,000 people affected in the UK alone. Alzheimer's disease is the most common accounting for 62% of cases. Parkinson' disease is the next most common neurological disorder and subsets of this disease also cause dementia as well. At present there is no simple blood test that can accurately diagnose these different diseases and that causes complications in prescribing the correct treatment and also changing treatments quickly as they stop working. Proteins have been measured in cerebro-spinal fluid but collection is painful, can cause problems and cannot be used as a repeat monitoring diagnostic. Our consortium brings together 3 technologies that allows measurement of these proteins in blood and particularly the aggregated forms which are through to be toxic and cause the nerve damage in the brain. This feasibility study will measure samples from Alzheimer's disease patients and change our understanding of diagnosis of this disease.


Research activity codes: 4.1 DISCOVERY AND PRECLINICAL TESTING OF MARKERS AND TECHNOLOGIES
Health categories: NEUROLOGICAL
Title: Development of a translational model system of the spinal pain synapse
Funder: Innovate UK

Abstract: The discovery of new drugs has become an extremely costly and time-consuming activity, and is associated with many late-stage failures. After 30 years of research there still remains a large unmet need for safe and effective treatments for chronic pain. One of the reasons for so many clinical failures, especially in the area of pain drug discovery, is the difficulty in predicting whether a potential drug will work in man. In order to address this problem we intend to develop a new technique that will allow the detailed evaluation of new drugs in a model pain system and so bridge the gap between traditional and well characterised but simple primary testing assays and more complex whole animal behavioural models. Success in this project will allow us to prioritise new pain drugs for testing in animal models, reducing the unnecessary use of animals as well as accelerating the drug discovery process and improving the chances of achieving success in man.


Research activity codes: 5.9 RESOURCES AND INFRASTRUCTURE (TREATMENT DEVELOPMENT);2.5 RESEARCH DESIGN AND METHODOLOGIES (AETIOLOGY)
Health categories: NEUROLOGICAL
Title: Screening for cognitive and behavioural change in ALS
Funder: Motor Neurone Disease Association

Abstract: In up to 50% of people with ALS, it is not only the motor system which is affected, but other areas of the brain, in particular the frontal lobes become involved. These people with ALS have corresponding changes in their thinking, language and behaviour, from subtle impairments to dementia. Unfortunately, available screening tests developed for other diseases are not well suited to assess cognitive function in ALS because of the range of physical disability in people with the disease, which can affect speaking and writing. Due to the lack of appropriate screening tools, clinicians often find it difficult to detect cognitive and behavioural changes in ALS and to determine how people are affected. Aims: This study will validate a newly developed screening measure, the Edinburgh Cognitive ALS Screen (ECAS), designed to detect changes in thinking, language and behaviour in ALS. In contrast with other screening procedures, the ECAS has been specifically developed for ALS and will distinguish between those types of impairments which are typically found in ALS from those associated with other factors common in older adults (such as Alzheimer’s Disease). Moreover, it has been developed to minimize the effects of physical disability (e.g. slurred speech, hand weakness) on performance in order to obtain results which accurately reflect the patient’s cognitive ability. It consists of a 15-20 minute assessment which is suitable for use within the ALS clinic by health professionals such as nurse specialists. Procedures: In Phase 1, 60 patients with a diagnosis of ALS, recruited through the new Scottish MND register, and 30 Healthy controls will undergo the screen and in a separate interview a full neuropsychological interview with standard tests covering all major domains of thinking and language. In Phase 2 we will compare the data from ALS patients with 20 patients with Alzheimer’s Disease and 20 with Frontotemporal Dementia, recruited through the Dementia Register for Scotland. The results will be analysed in terms of the screen’s sensitivity at detecting changes in thinking, language and behaviour in ALS as compared to the full neuropsychological interview, and its specificity at differentiating changes resulting from ALS from other disorders. A short within clinic assessment will have immediate significance in assisting MND health professionals in the planning and implementation of care and providing tailored intervention. The screen will also provide essential information for future evaluation of treatment efficacy.


Research activity codes: 4.4 POPULATION SCREENING
Health categories: NEUROLOGICAL