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Drug development and clinical trials for Neurodegenerative Disorders

drug development and clinical trials

Our research is closing a critical gap in translating basic research and new discoveries into treatments that can make a real difference to health outcomes. We focus on the two key areas of translating discoveries into effective treatment and repurposing existing drugs, bringing them into clinical practice.

We are leading an international programme to accelerate promising new therapies to bring them forward to large-scale cohorts and robust clinical trials. As well as the development of some exciting new drugs, we research existing drugs that can be repurposed for other uses for which they are effective. As these drugs are known to be safe, it reduces time and cost, meaning they can make a meaningful difference to people’s lives as swiftly as possible. 

Many people with dementia are over-medicated for distressing symptoms, such as anxiety, agitation and psychosis. We have shown that widely-used medications, such as antipsychotic medications and sleeping tablets can have devastating side-effects, including increased mortality. Our work has played a key role in achieving a substantial reduction in the use of these drugs and the serious related consequences. We also want to develop new and safer treatment approaches.

We have developed and evaluated our nursing home training programme (WHELD) in large clinical trials and demonstrated significant improvement in quality of life and mental health for residents with dementia. Visit the WHELD website.

We have shown in clinical trials that better treatment of pain significantly reduces agitation and we have played a pivotal role in re-focussing of the non-dopaminergic antipsychotic pimvanserin and conducting the key clinical trials to demonstrate the benefits as a treatment for psychosis in people with Parkinson’s disease and people with dementia, with a much better tolerability profile than currently used antipsychotics. We also currently have ongoing trials of cannabidiols and ondansetron for the treatment of psychosis in people with Parkinson’s disease.

We have evaluated the currently available evidence with systematic reviews and international consensus to identify the best treatments. We have already completed several trials, including a very encouraging study of the anti-diabetes drug liraglutide; and we are about to commence trials of two of the best emerging candidate treatments – phenserine (two trials: Alzheimer’s disease and dementia with Lewy bodies) and fasudil (Alzheimer’s disease). We also have an exciting clinical trial programme focussing on disease modifying therapies for Parkinson’s disease, including an ongoing trial of a repurposed experimental compound (AZD0328) and 2 trials about to commence with a novel intervention targeting Parkinson’s pathology in the gut.

Neurodegeneration is a long process that pre-dates clinical symptomatology by decades and the attention of researchers is shifting from the description of the symptoms towards the characterization of what biological changes take place from the pre-clinical stage to advanced disease using sophisticated techniques of neuroimaging such as Positron Emission Tomography (PET) and Magnetic Resonance Imaging (MRI) . One specific programme is evaluating the glymphatic system, a newly-discovered system of waste disposal of the brain, as an early treatment target. Our study programme aims to provide a deep characterization of how the glymphatic system is altered in Parkinson’s disease and Alzheimer’s disease as opposed to normal ageing, how its function can be measured by the identification of new biomarkers, and how it can be modulated by pharmacological intervention with promising, existing drugs such as Carvedilol and Prazosin.

The Parkinson’s genetic programme comprises of a series of studies funded by the Michael J. Fox Foundation for Parkinson’s research for the identification of biological signatures of the disease before the clinical onset of parkinsonism and the understanding of evolution of the pathology in vivo. We aim to identify potential targets for antinflammatory therapies that could improve this condition and prevent the progression of the disease.

We are part of a large collaboration with academic and industrial partners, called MIND-MAPS, which aim is to find new PET and MRI biomarkers that can be used to develop new drugs. In this collaboration, we have previously demonstrated alterations in the synapses of patients with Parkinson’s disease and are currently expanding this study on patients with Multiple System Atrophy and Amyotrophic Lateral Sclerosis, two progressive disorders for which no therapy is currently available.

We are developing approaches for precision medicine, to develop targeted treatments and clinical trials for people with risk genes for Parkinson’s disease, such as a GBA polymorphism.

Using the PROTECT platform, with more than 25,000 healthy people over 50 signed up online in the UK, and more than 50,000 worldwide with participants in the US, Norway, Spain and Canada, we run a series of digitally enabled trials simultaneously on the factors that help people stay mentally healthy in later life, already demonstrating the benefits of brain training in large clinical trials (available free as part of our PROTECT programme) and we are currently evaluating a range of other interventions including self-directed exercise and vitamin D.

Visit the PROTECT website

Drug development and clinical trials - primary investigators

Professor Clive Ballard                    Professor of Age-Related Diseases Dementia, antipsychotic medications, non-pharmacological interventions, cognitive health, neuropsychiatric symptoms, dementia care, clinical trials.
Professor Marios Politis Professor of Neurology Neurology, neuroimaging, Parkinson’s disease, dementia, clinical trials
Professor Roger Mills Professorial Advisor Drug Development, Clinical Trial Design
Professor Jeffery Cummings Professorial Advisor Neurology, dementia, clinical trials, Parkinson’s disease
Professor Zahinoor Ismail Professor of Old Age Psychiatry Mild Behavioural Impairment, dementia, clinical trials
Dr Anne Corbett Senior Lecturer in Dementia Research Dementia, dementia reduction, drug discovery, clinical trials, care home research, clinical trial delivery, online research, translational research, patient and public involvement
Helen Brooker Senior Research Fellow Ageing, cognitive function, automated testing, clinical trials
Professor Joe Butchart Honorary Associate Professor Geriatric medicine, dementia, clinical trials
Professor Ray Sheridan Honorary Associate Professor Geriatric medicine, Parkinson’s disease, dementia, clinical trials
Professor Chris Fox Professor of Clinical Psychiatry Dementia, dementia prevention, antipsychotic medications, non-pharmacological interventions, cognitive health, neuropsychiatric symptoms, dementia care, clinical trials, artificial intelligence, care home research