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Research

New pan-European platform will boost research sharing

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A new European initiative will see the development of a collaborative platform for data and sample sharing for neurodegenerative diseases. 

The project, from the European Platform for Neurodegenerative Diseases (EPND), will enable sample and data sharing, leveraging and linking existing European research infrastructures to accelerate the discovery of biomarkers, new diagnostics and treatments for the benefit of people with neurodegenerative diseases such as Alzheimer’s and Parkinson’s.

There is currently a lack of available treatments that can prevent or modify the progression of neurodegenerative disorders like Alzheimer’s disease (AD) and Parkinson’s disease (PD), which affect millions of people in Europe. 

Despite robust research efforts to accelerate biomarker discovery, at this time there are few secure, accessible ways for clinical samples and data to be discovered and shared within the neurodegenerative disease research community.

“Alzheimer’s and Parkinson’s cost millions of lives and will create an estimated economic burden in Europe of €267 billion by 2030,” said project co-coordinator Pieter Jelle Visser, associate professor at the University of Maastricht. 

“EPND will answer this massive challenge with a solution on the same scale, bringing together teams, samples and data from across the continent.”

The development of effective treatments requires biomarkers for early detection of disease in individuals, for assessing treatment efficacy, and for patient stratification. 

Through funding from the Innovative Medicines Initiative (IMI), EPND will begin a five-year effort to establish a self-sustaining network that will support the discovery, harmonisation, storage and analysis of high-quality clinical samples and data from neurodegenerative disease research.

The programme will build a secure platform via a European node on the AD Workbench of the Alzheimer’s Disease Data Initiative (ADDI), a medical research organisation committed to increasing interoperability of data platforms and empowering scientists to analyse data for new discoveries in dementia research. 

Through this collaboration, EPND aims to integrate and build on existing initiatives while removing barriers to collaboration and discovery.

“The EPND platform will move us towards comprehensive data discovery, harmonisation, storage and analysis, by virtue of a core workbench concept that builds upon national and international data infrastructures.” said project co-coordinator Anthony Brookes, professor of genetics at the University of Leicester. 

“The collaboration will deliver on the promise of big data to rapidly generate new knowledge and share emerging insights, and thereby advance AD and PD treatments whilst also providing an integrative technical and governance template that can be adopted by many more healthcare domains.”

Beyond establishing the network, the project aims to create agreed principles to enable access to samples and data, establish fair and transparent governance and processes, and achieve self-sustainability after five years. 

When complete, it will create a new entity supported by some of the most prestigious medical and research organisations in the EU. 

The public-private partnership will accelerate and simplify innovation in the areas of R&D, regulatory, clinical and healthcare practices.

With a commitment to enabling secure and transparent data sharing and sample access, EPND aims to optimise the use of existing resources, including a large portfolio of longitudinal research cohorts, while leading with high ethical standards and robust protection for the fundamental rights of research participants in compliance with the GDPR.

Research

Neurodegenerative disease research backed by £58.6m

AviadoBio has secured investment to further its pioneering work into frontotemporal dementia and ALS

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Pioneering research which sees the use of a gene therapy platform to explore treatments for neurodegenerative diseases has been backed by new investment of £58.6million. 

AviadoBio has raised the funds to use its patented platform to initially look into therapies for Frontotemporal dementia (FTD) and Amyotrophic Lateral Sclerosis (ALS, or motor neurone disease, MND).

The substantial investment to the venture – developed at the UK DRI at King’s – follows £12million in seed funding, and will allow King’s spinout AviadoBio to take their research into human clinical trials in FTD patients for the first time. 

The funds will also help advance other pre-clinical work into ALS and ultimately other neurogenerative diseases.

Frontotemporal dementia is a progressive neurodegenerative disease affecting the frontal and temporal lobes of the brain and is the second most common form of young-onset dementia (over 65 years). FTD is characterised by changes in personality, behaviour and language, rather than short-term memory deficits seen in Alzheimer’s disease. 

ALS/MND causes progressive muscle paralysis affecting limb movements speech and swallowing, leading to death from respiratory failure within three to five years. 

Crucially, there are no treatments that can slow or halt progression of either disease.

Gene therapy is a promising approach for the treatment of neurodegenerative diseases, based on the principle of delivering DNA into cells to supplement or knock down mutated genes. 

AviadoBio – founded by Professor Christopher Shaw, Dr Youn Bok Lee and Dr Do Young Lee – is using the Adeno-associated virus (AAV), which can be engineered to carry theses gene therapies to target specific cells such as neurons in the brain and spinal cord. This technology proved life changing for babies with the genetic motor neuron disease, spinal muscular atrophy (SMA), who rarely lived beyond two years without being ventilated. 

A single injection in infancy protected their motor neurons such that they could walk, talk and did not need ventilation.

The therapy will target the gene encoding the protein progranulin, which is mutated in about 20 per cent of inherited and five per cent of non-inherited or sporadic cases. 

Progranulin is important for biological processes such as inflammation and the growth of neurons, and mutations of the gene in FTD cause a significant reduction in the levels of the protein. Delivering unmutated copies of the gene will help restore this imbalance in progranulin and improve the symptoms experienced in the disease.

“AviadoBio’s unique platform combines next-generation gene therapy design with deep neuroscience expertise and a novel neuroanatomy-led approach to drug delivery,” says Professor Christopher Shaw, professor of neurology and neurogenetics at the Institute of Psychiatry, Psychology & Neuroscience (IoPPN) at King’s College London, and co-founder, chief scientific and clinical advisor of AviadoBio.

“Precision micro dosing achieves extensive gene expression throughout the nervous system, maximising the therapeutic potential for patients living with devastating neurological diseases.

“While many neurodegenerative conditions start in a specific region gene, the pathology eventually spreads throughout the nervous system. 

“We have seen that modifying gene expression can be curative, but achieving widespread distribution is the greatest challenge. We have shown that precision micro dosing to neural networks will deliver broad central nervous system (CNS) expression, providing safe and effective treatments.”

The investment marks a huge milestone for the new company, as well as for Professor Shaw personally, whose decades of discovery research into FTD and ALS have set the groundwork for these promising new disease therapies.

Professor Shaw continues “After spending my entire career to date researching the causes of these diseases and designing therapies that bring hope of a cure for patients, I believe that AviadoBio has the potential to achieve this.”

Professor Shitij Kapur, president and principal of King’s College London, said: “Chris has worked with King’s for over 25 years. 

“I’m honoured that he chose our university to develop this ground-breaking research, and that staff and students have benefited from his world leading science along the way. I am pleased King’s is helping to take that work forward that has the potential to change the world and make a huge impact on many people’s lives.”

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Dementia

De-cluttering ‘may not help’ with dementia

People with moderate dementia performed better when surrounded by their usual clutter, a new study has revealed

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A clutter-free environment may not help people with dementia carry out daily tasks in the way that has traditionally been thought, a new study has revealed. 

Researchers at the University of East Anglia (UEA) studied whether people with dementia were better able to carry out tasks, such as making a cup of tea, at home – surrounded by their usual clutter – or in a clutter-free environment.

And they admit to being “surprised” to find that participants with moderate dementia performed better when surrounded by their usual clutter.

But the different environments made no difference to people with mild and severe dementia, who were able to perform at the same level in both settings.

Professor Eneida Mioshi, from UEA’s School of Health Sciences, said: “The majority of people with dementia live in their own home and usually want to remain living at home for as long as possible.

“So it’s really important to know how people with dementia can be best supported at home – one possible route would be by adapting the physical environment to best suit their needs.

“As dementia progresses, people gradually lose their ability to carry out daily tasks due to changes in their cognitive, perceptual and physical abilities. Participation in daily tasks could then be improved by adapting the person’s environment.

“To this end, we wanted to investigate the role of clutter in activity participation, given the potential to use de-cluttering to support people with dementia to continue to be independent.

“Environmental clutter has been defined as the presence of an excessive number of objects on a surface or the presence of items that are not required for a task.

“It is generally assumed that a person with dementia will be better able to carry out daily tasks when their home space is tidy and clutter free.

“However, there has been very little research to really test this hypothesis.

“We wanted to see whether clutter was negatively affecting people with dementia. So we studied how people at different stages of dementia coped with carrying out daily tasks at home, surrounded by their usual clutter, compared to in a clutter-free setting – a specially designed home research lab.”

Occupational therapist and PhD student Julieta Camino carried out the study with 65 participants who were grouped into those with mild, moderate and severe dementia.

They were asked to carry out daily tasks including making a cup of tea and making a simple meal, both at their own home and at UEA’s specially-designed NEAT research bungalow – a fully furnished research facility that feels just like a domestic bungalow.

The researchers evaluated performance of activities in both settings, and also measured the amount of clutter in the participants’ homes. Meanwhile, the NEAT home setting was completely clutter free.

Julieta, also from UEA’s School of Health Sciences, said: “We thought that the complete absence of clutter in our research bungalow would play a beneficial role in helping people with dementia with daily living activities. But we were wrong.

“We were surprised to find that overall, people with moderate dementia, in particular, performed daily tasks better at home – even though their homes were significantly more cluttered than our research bungalow.

“And it didn’t seem to make any difference how cluttered the participant’s home was. The only factor that contributed to how well they could carry out tasks at home was their level of cognition – with those with severe dementia encountering the same difficulties to perform the tasks at home and in the research bungalow.”

This research received funding from the Alzheimer’s Society and National Institute of Health Research (NIHR) Applied Research Collaboration East of England (ARC EoE) programme.

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Research

1 in 100 COVID patients develop brain conditions

A new study reveals the risk of stroke, haemorrhage or encephalitis as a result of the virus

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Around one in 100 patients hospitalised with COVID-19 will develop brain conditions including stroke and haemorrhage as a result of the virus, the largest multi-site study to date has found. 

The multi-institutional research investigated brain complications of COVID and found that complications of the central nervous system can occur in one in 100 people. 

“Much has been written about the overall pulmonary problems related to COVID-19, but we do not often talk about the other organs that can be affected,” said study lead author Dr Scott H. Faro, professor of radiology and neurology and director of the division of neuroradiology/head and neck imaging at Thomas Jefferson University in Philadelphia. 

“Our study shows that central nervous system complications represent a significant cause of morbidity and mortality in this devastating pandemic.”

The most common complication was ischemic stroke, with an incidence of 6.2 per cent, followed by intracranial haemorrhage (3.72 per cent) and encephalitis (0.47 per cent), an inflammation of the brain.

Dr Faro initiated the study after discovering that existing literature on central nervous system complications in hospitalised COVID-19 infected patients was based on a relatively small number of cases.

To derive a more complete picture, he and his colleagues analysed nearly 40,000 cases of hospitalised COVID-19 positive patients from seven US and four western European university hospitals. 

The patients had been admitted between September 2019 and June 2020. Their average age was 66 years old, and there were twice as many men as women.

The most common cause of admission was confusion and altered mental status, followed by fever. Many of the patients had co-morbidities like hypertension, cardiac disease and diabetes.

There were 442 acute neuroimaging findings that were most likely associated with the viral infection. The overall incidence of central nervous system complications in this large patient group was 1.2 per cent.

“Of all the inpatients who had imaging such as MRI or a CT scan of brain, the exam was positive approximately ten per cent of the time,” Dr Faro said. 

“The incidence of 1.2 per cent means that a little more than one in 100 patients admitted to the hospital with COVID-19 are going to have a brain problem of some sort.”

The researchers also discovered a small percentage of unusual findings, such as acute disseminating encephalomyelitis, an inflammation of the brain and spinal cord, and posterior reversible encephalopathy syndrome, a syndrome that mimics many of the symptoms of a stroke.

“It is important to know an accurate incidence of all the major central nervous system complications,” Dr Faro said. 

“There should probably be a low threshold to order brain imaging for patients with COVID-19.”

The study is being presented at the annual meeting of the Radiological Society of North America (RSNA).

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