Neurological Conditions - Q1 2023

Neurological Conditions

“An integrated response to neurology through Intersectoral Global Action Plan.”

Tadeusz Hawrot, Senior Policy Advisor, European Federation of Neurological Associations

Page 05

“In the UK, there are an estimated 700,000 unpaid carers of people with dementia.”

Dr Karen Harrison Dening, Head of Research and

Publications at Dementia

Page 06

UK

Dame Pamela Shaw Head of Neurology and Director of the Neuroscience Institute

WRITTEN BY Mark Nicholls

New drug potential game-changer for MND treatment

A leading neuroscientist highlights how scientists are making significant steps forward in discovering treatments for neurological disorders — but more can be done.

Professor Dame Pam Shaw discusses the impact of devastating neurological conditions such as Motor Neurone Disease (MND), Parkinson’s disease, Alzheimer’s and Multiple Sclerosis, on patients and their families and carers.

“I want to see the same momentum and urgency for the treatment of neurological disorders as there is for cancer,” says Shaw, Professor of Neurology at the University of Sheffield and honorary consultant neurologist at Sheffield Teaching Hospitals.

Flagship research

However, she believes scientists are on the brink of developing effective treatments for neurological disorders that are becoming more common as people live longer.

Shaw is Director of the Neuroscience Institute, one of the University of Sheffield’s flagship research centres, which combines expertise in medicine, science and engineering to improve prevention, diagnosis and treatment of neurological, sensory and development disorders.

Improving outcomes

Researchers at the Neuroscience Institute recently completed a Phase 3 clinical trial, investigating a rare type of MND (responsible for 2% of MND cases) caused by a faulty SOD1 gene. Following the trial, patients reported symptom progression had slowed down 12 months after taking

Paid for by The University of Sheffield

People in charge of dementia care face many grave difficulties

an investigational drug. Shaw describes the trial results as “a game-changer for MND.”

“Never before have I seen patients say, “I’m getting better; I’m doing things today that I couldn’t do a few months ago.”

“Patients with SOD1 mutations are relatively rare, but this trial will change the future of MND trials. We can now investigate other genes that cause MND and measure the effects of treatment much quicker,” she adds.

Promising future

The Neuroscience Institute is constantly enhancing its capacity to develop better devices for neurological disability and continues to form industry partnerships to develop treatments.

The University of Sheffield is a leader in gene therapies; a promising approach to treating neurological disorders. A new gene therapy centre (GTIMC) dramatically broadens the scope of gene therapy research in the UK and will translate scientific discoveries into new treatments.

“We want to strengthen the power of neurosciences to find better solutions for people with neurological disorders,” Shaw concludes.

Read more at sheffield.ac.uk/neuroscience-institute

Project Manager: Matthew Stead

Dr Emma Wolverson Research Lead at Dementia UK and Senior Lecturer in Ageing and Dementia, The University of Hull, UK

Dementia is the leading cause of death in the UK, and the number of people dying from dementia will double by 2040.

Arecent survey found that only 42% of the British public is aware that dementia is a terminal illness. Concerns have been raised that, in an effort to reduce stigma and encourage people to seek an early diagnosis, messages about ‘living well’ with dementia may have inadvertently silenced conversations about the advanced stages of the condition and its life-limiting and progressive nature.

Conversations around dying

Failure to recognise dementia as a terminal illness impacts the end-of-life care provided to people with dementia. Currently, people with dementia and their families face inequalities in access to palliative and end-of-life care services.

As a result, people experience frequent transfers to and from hospitals, burdensome and invasive treatments and poor pain management.

Dying with dementia is different Dementia is often wrongly regarded as being no different from other conditions that people die from. But providing end-of-life care for people with dementia requires a high level of skill, as they tend to live with multiple chronic health conditions and frailty. This makes their care complex.

The course of dementia is difficult

to predict. It does not fit in well with existing palliative or hospice models of care, and there is no one specialist with a clearly defined role to care for the person with dementia during this time.

Healthcare staff often report lacking the knowledge, skills and confidence to support people dying with dementia who are unable to communicate their needs and preferences in the advanced stages of the condition.

Involving families

Most people living with dementia are cared for by a family member. As a person approaches the end of their life, exhausted family members who are experiencing anticipatory grief, witnessing distress and disorientation are often asked to become proxy decision-makers. Life and death decisions are quite literally demanded of them. Many have never had important conversations with their relatives about their wishes in such a situation. Clinicians may witness disagreement from family members as a result of a lack of understanding and preparedness.

Dementia UK researchers are leading the way in pioneering research to explore what a good death looks like for people with dementia. The charity is also collaborating with the European Association of Palliative Care to review the definition of optimal palliative care in dementia.

Editor: Angelica Hackett O’Toole | Head of Digital Operations: Harvey O’Donnell Paid Media Strategist: Jonni Asfaha Social & Web Editor: Henry Phillips

Development: Ellie McGregor | Head of Print & Design: Thomas Kent Designer: Aimee Rayment

Assistant: Carolina Galbraith Duarte | All images supplied by Gettyimages, unless otherwise specified | Contact information: uk.info@mediaplanet.com or +44 (0) 203 642 0737

Providing end-of-life care for people with dementia requires a high level of skill, as they tend to live with multiple chronic health conditions and frailty.

I want to see the same momentum and urgency for the treatment of neurological disorders as there is for cancer.

New treatments are urgently needed to support patients with dementia as cases, globally, are set to soar beyond the 150 million mark by 2050.

As people live longer, dementia cases are rising and could soon hit 150-plus million worldwide.

Yet, there has been no new dementia treatment in the UK for two decades.

The most common cause of the neurodegenerative disease is Alzheimer’s, accounting for 60–70% of all cases, but other causes include vascular dementia, frontotemporal dementia and chronic traumatic encephalopathy.

Complexity for patients with dementia

Professor Bjoern Schelter explains there can be lengthy diagnostic pathways for the condition, starting with early symptoms of people forgetting things more often.

“From there,” he continues, “it is a consultation with a primary care physician, specialist appointments and further assessment. These can be questionnaire-based, involve neuroimaging and blood tests, and it often takes 6–12 months for a diagnosis.”

Dr Miller, Head of Medical Affairs at Aberdeen-based TauRx, a company focused on neurodegeneration therapies targeting tau pathology, points to the extraordinarily long journey toward new dementia drugs. However, she also believes much of the research and complex trials conducted since the early 2000s are coming to fruition.

Ongoing clinical trials

One driver of Alzheimer’s is an accumulation of aggregated tau protein inside nerve cells. TauRx is developing a potential treatment targeting this pathology directly, which is in the latter stages of clinical trials.

The company’s CEO and co-founder, Professor Claude Wischik, says that if the results continue to be positive, it will accelerate diagnosis and treatment. The drug, and smarter diagnostic tools, will “empower primary care physicians to become active much earlier in the whole patient journey.”

While his organisation has concentrated on the development of a drug to target the process of tau aggregation directly, he also acknowledges other parallel research looking at amyloid pathology.

Modernising the diagnostic process

Meanwhile, Professor Schelter, CEO of TauRx sister company GT Diagnostics — which is working to develop digital and artificial intelligence (AI) tools to enhance the diagnostic process, reduce the timeline to diagnosis and support early treatment — says dementia diagnostics need to be brought into the 21st century.

“We need robust diagnostics that can detect the disease at very early stages and very efficiently, in a primary care setting.”

While GPs are under increasing pressure, his colleague Dr Sonya Miller believes there’s an opportunity for family doctors to take a more proactive role; but to do this, they need support and better diagnostic tools.

“GPs are a logical group to be empowered to take this on. Dementia is a complex disease and an increasing problem that cannot remain a speciality disease.”

Growing demand for treatments

Dr Miller says patient advocacy groups do an amazing job of raising awareness and working to destigmatise the disease and offer support to patients and families.

“Next, we need support of primary care providers to recognise, diagnose and refer as required,” she says.

A concern has been the lack of new treatments, at a time when more people are needing diagnosis and treatment.

With 152 million people expected to be suffering from dementia by 2050, the fear is that healthcare systems could be overwhelmed without new treatments. “A safe and efficient treatment is needed to stop the disease process very early on, then these diagnostic tools become monitoring tools for the effectiveness of treatments,” he says.

Rethinking brain health

Within the diagnostic and monitoring process, smartphones/tablets can use intuitive AI to interpret psychometric questionnaires, blood tests and brain imaging.

Patients also need to be proactive in keeping their brains healthy through lifestyle choices, says Dr Miller: “We require a whole societal paradigm shift in thinking about brain health.”

Underpinning that, she adds, are new diagnostics and an oral medication for dementia. By treating dementia with more urgency, we can be better prepared to manage it — today and in the future.

Dr Sonya Miller Head of Medical Affairs, TauRx

Professor Bjoern Schelter CEO, GT Diagnostics and Chief Analytics Officer, TauRx

Professor Claude Wischik CEO and Co-founder, TauRx

gtdiag.com

Dementia cases are soaring high: new treatments are needed to help patients

Paid for by TauRx Therapeutics

With 152 million people expected to be suffering from dementia by 2050, the fear is that healthcare systems could be overwhelmed without new treatments.

WRITTEN BY Mark Nicholls

Dr Farrar liaises with physicians responsible for referring and scanning patients, keeping them abreast of new trends and image analysis methods and supporting their research efforts.

How has your role evolved in recent years?

The trend is towards using software tools to enhance image interpretation as well as collaborating with consortiums such as AMYPAD In Alzheimer’s disease and PPMI (sponsored by MJ Fox Foundation) in Parkinson’s disease.

How do neurology imaging agents work?

Molecular imaging tracers bind to brain pathology (proteins that are abnormally present or contain structural deficits not present in a healthy person) and are labelled with a short-lived radioisotope. Once administered, the radioisotope decays and emits energy or particles detected by a camera, and the image is then reconstructed, showing the absence or presence of pathology.

The tracer Vizamyl targets amyloid, a seminal feature of Alzheimer’s disease (AD), and is seen at an early stage of the disease. In Parkinson’s disease (PD), the tracer DaTSCAN detects the loss of dopaminergic neurones. DaTSCAN differentiates both PD from essential tremor; and dementia with Lewy bodies from AD.

Both tracers are used in combination with clinical work-up and provide adjunctive information about the pathological status of the patient.

What value does the scan bring to the patient?

In routine clinical work-up, the signs and symptoms of these neurological diseases are quite difficult to tease apart.

We are directly imaging in the brain the neuropathology associated with that disease. The scan adds a huge amount of weight to the final clinical diagnosis. Once a diagnosis has been established, the patient and their families — with the physician — can plan for the future.

How might these tracers be used in the future?

For the Vizamyl agent, the advent of new anti-amyloid therapies could potentially designate a patient as amyloid-positive and hence receive and possibly monitor therapy impact. For DaTSCAN, the current focus is on early disease detection and, in combination with software tools, could aid a more timely diagnosis.

Dr Gill Farrar Global Medical Leader Neurology, Medical Affairs, GE Healthcare

Paid for by GE HealthCare

Image provided by GE HealthCare | Vizamyl Negative and Positive

Molecular brain imaging can facilitate an accurate diagnosis of neurological disorders

Around 145,000 people in the UK are living with Parkinson’s, and it is estimated that 1 in 37 people alive today will be diagnosed with the condition in their lifetime.

~ Arthur Roach Director of Research, Parkinson’s UK

Supporting the implementation of the neurological global action plan

After years of neglect, neurological disorders and brain health are coming out of the shadows. Neurological conditions are the leading cause of disability-adjusted life years and the second leading cause of death worldwide.

The UN High-Level Political Declaration added neurological and mental disorders as the fifth priority in the global noncommunicable diseases agenda. In 2020, WHO established a Brain Health Unit that played a central role in developing an integrated response to neurology through Intersectoral Global Action Plan on Epilepsy and Other Neurological Disorders 2022–2031. The IGAP was adopted by all WHO Member States at the World Health Assembly in 2022.

Towards a united voice for neurology

Those exceptional developments called for a global united voice of the neurological community, capable of exerting political influence and engaging with policymakers at the highest levels. In 2019, the European Federation of Neurological Associations (EFNA) started engaging at a global level to capitalise on the promising trends and started advocating for a comprehensive global response to neurological disorders and brain health.

Soon, it became clear that to effect a real change, there is a need to build a global coalition that would have the patient voice strongly built in. Consequently, in 2021, EFNA created the OneNeurology Initiative which evolved into the OneNeurology Partnership co-founded by EFNA and the European Academy of Neurology.

A vehicle to support the IGAP implementation

The Partnership is a patient-focused and multi-stakeholder alliance bringing together international neurological organisations and regional umbrellas. As such, it is the only global partnership where patients, clinicians and researchers come together to advocate for neurological conditions as one.

The partnership is united by a vision of a world where neurological health is valued and protected as our most vital asset — to the benefit of all society.

It is calling for neurological conditions to be prioritised globally within the context of brain health. The Partnership speaks in one voice to raise awareness of the impact of neurological conditions, the challenges to overcome and the opportunities to improve brain health for everyone. The iGAP is central to the OneNeurology work that is strategically placed to assist WHO and Member States in successfully implementing the plan. Leveraging the plan, the Partnership encourages patient advocates worldwide to drive change.

Tadeusz Hawrot Senior Policy Advisor, European Federation of Neurological Associations

The Partnership speaks in one voice to raise awareness of the impact of neurological conditions, the challenges to overcome and the opportunities to improve brain health for everyone.

A pioneering approach to Parkinson’s research

WRITTEN BY Arthur Roach Director of Research at Parkinson’s UK

Parkinson’s is a progressive neurological condition with a variety of genetic and environmental contributors. Loss of dopamine neurons in the brain produce its three hallmark symptoms of tremor, slowness of movement and muscle stiffness. However, there are more than 40 symptoms affecting the gut, thinking, voice and mental health. Around 145,000 people in the UK are living with Parkinson’s, and it is estimated that 1 in 37 people alive today will be diagnosed with the condition in their lifetime.

Drug discovery and development

The Parkinson’s Virtual Biotech is the international drug discovery and development programme founded in 2017 by Parkinson’s UK, the largest European charitable funder of Parkinson’s research. It operates as an evergreen seed venture fund but is driven by the priorities of people with Parkinson’s, not investor profit. Collaborative and agile, it adapts successful methods from the business world to deliver new treatments in years — not decades.

Uniting global experts

With international supporters and investments in the UK, US and Australia, the Parkinson’s Virtual Biotech has grown into a global movement uniting scientists, supporters, investors and innovators worldwide. In 2022, a long-term multimillion-dollar strategic partnership with the Parkinson’s Foundation in the US was initiated, which was a key development for the programme.

Currently, the programme plans to provide £50 million in funding over 10 years to help the companies it is working with move their projects forward, but there are hopes that even more will be possible with new funding partnerships.

The primary criterion for project selection is: having a realistic chance of creating a new treatment that addresses one of the top priorities of people living with Parkinson’s. Thus, the programme may take a chance on slightly earlier projects, with more technical risk, than other funds. However, investments are made on the terms that the same kinds of financial returns any other early investor would expect are brought to the patient community.

Delivering results

In just five years, the Parkinson’s Virtual Biotech has supported 13 projects including six clinical trials that are hoping to stop or slow progression or target specific symptoms of more advanced Parkinson’s, including hallucinations, psychosis and dyskinesia (uncontrolled movements).

In a major milestone in 2022, NRG Therapeutics in the UK secured £14 million from other investors, alongside £2 million from the Parkinson’s Virtual Biotech to fund its promising research into treatments to slow the progression of Parkinson’s, after the programme provided it with seed funding over two years.

To find out more about Parkinson’s Virtual Biotech, visit parkinsons.org.uk/ research/parkinsonsvirtual-biotech

are the benefits of specialist nursing in dementia care?

An estimated 944,000 people are living with dementia in the UK,1 and their needs affect all aspects of their life.

In the UK, there are an estimated 700,000 unpaid carers of people with dementia.3 Dementia is a progressive condition and can have a significant impact on the health and wellbeing of family members.

The importance of interventions after diagnosis

In a Cochrane Review, additional benefits were found to adopting a case management approach in dementia. These include reducing inappropriate admission to hospital, delaying a transition to care homes, reducing the length of stay in hospital and reduction in behaviour disturbance.4

Dr Karen Harrison Dening Head of Research and Publications, Dementia UK

Families affected by dementia often require support from a complex matrix of health and social care services.2 For many families, concerns arise very early on when they notice that ‘something is not right’ before a diagnosis of dementia is given.

Once a diagnosis has been made, family-centred approaches can help to clarify issues and provide a structure to unite families, while still recognising the individuality of the person with dementia.

Case management in dementia care

Such relationship-centred approaches are best delivered through case management.² Case management is a collaborative process led by a case manager to both deliver care and coordinate care from others, such as a district nurse or social worker. It involves triage, assessment, planning and review of care.

A case management approach may increase the use of community services initially; however, it has demonstrated cost savings within the first year.5

Specialist nurse case management model

The Admiral Nursing case management model was established in the UK to uniquely join the different parts of the health and social care system and enable the needs of family carers and people with dementia to be addressed in a coordinated way.6

Admiral Nurses (specialist dementia nurses) work across a variety of NHS organisations (primary, secondary, acute and community care) as well as in social care, care homes and hospice settings. Case management is central to their role.

Impact of specialist dementia nurses

To find out more about Admiral Nurses, visit dementiauk. org/getsupport/ what-is-anadmiral-nurse/

Case management has a long and successful history of supporting people with long-term conditions, such as severe mental illness and brain injury. In dementia care, case management is a relatively new concept by comparison and can provide consistency of specialist services and support to a family affected by dementia to reduce the fragmentation of services often experienced to enable receipt of the right care at the right time.²

Admiral Nursing aims to work with more complex cases and with high-level needs. They are required to engage with all local services and agencies to ensure families with less complex needs receive an appropriate level of care.

Admiral Nurse case management ensures families affected have access to support across the life course of the condition. Evaluations of Admiral Nursing services show clear benefits and outcomes to both families affected by dementia and to health and social care organisations.

References avaliable online at healthawareness. co.uk/campaign/ neurology/

Parkinson’s is the fastest-growing neurological condition in the world, and there is currently no cure.

What

At UCB, we have been focused on the research and development of treatments for epileptic seizures for more than 20 years, and our commitment to providing solutions for those in need continues. We are working towards a future where we hope to impact the underlying causes of the epilepsies and potentially change the course of disease.

To date, the research field has mainly focused on treating epilepsy symptoms (ie. seizures). However, a growing understanding of how and why seizures occur is taking research in exciting new directions.

An important shift in the field has been to recognise that there are multiple types of epilepsy. In fact, we now more commonly refer to epilepsy as the ‘epilepsies,’ indicating that it’s not really a single disease. In addition, for many people — particularly those living with certain rare epileptic syndromes — seizures are not the primary or most impactful symptom.

A more in-depth understanding of epilepsy is shaping a different research approach

Looking into underlying causes of epilepsy

Our approach to epilepsy research at UCB is grounded in developing a better understanding of the molecular signatures and pathways of underlying disease — getting to the root cause of specific conditions. With this, it is our ambition to define and regroup epilepsy populations based on mechanisms with a view of developing more targeted approaches.

cause disease directly.

But for complex epilepsies, where mechanisms may be less well-understood, the situation is even more challenging. Here, we are building on genetics to help understand universal disease mechanisms across the epilepsies. In addition, we have also developed an artificial intelligence computational framework in collaboration with various academic partners to investigate disease mechanisms and processes, which may help us to identify novel areas for future investigation.

Innovation is at our core

For example, when we understand the root cause of certain epilepsies, it opens opportunities to potentially target the underlying mechanisms that

Our commitment to epilepsy research has never been stronger, and as science advances, so does our curiosity. We are hopeful for the future, and we will learn and refine our thinking as we progress. Innovation is at our core, meaning we are constantly evolving and changing as new technologies, trends, research findings and ideas emerge. Not everything will work, and this is part of the learning — but research is in a good place.

Our focus is on

Paid for by UCB Pharma

Millions of people around the world are affected by epilepsy. The science behind research is moving in exciting new directions.

Stefanie

Dedeurwaerdere

Head of Epilepsy Discovery Research, UCB

Our approach to epilepsy research at UCB is grounded in developing a better understanding of the molecular signatures and pathways of underlying disease.

the moments that matter. On seeking out scientific innovations with the greatest impact on the lives of people with severe diseases. On value that can’t be expressed in numbers alone.

Because nothing else matters

Almost two-thirds of people needing mental health support were not in contact with NHS services in 2021, according to a recent report from the National Audit Office.

An estimated 8 million people did not access the mental health support they needed, highlighting a significant treatment gap in England. We also know at least 1.4 million people are currently waiting to receive care from NHS mental health services.

At first glance, these statistics may give the impression that services are falling short. However, the number of people in contact with NHS mental health services grew at an alarming rate from 3.6 million to 4.5 million in the space of five years.

The problem is that demand for mental health services is rising at an unprecedented rate, and faster than investments are being made into expanding the workforce. This sits alongside other barriers to people accessing support, such as stigma and long waiting times.

Cost of living and mental health crises

The need for a properly funded mental health workforce has never been greater, as millions of people

across the country endure the cost-of-living crisis. The Office for National Statistics (ONS) has found that food prices remain at record highs while the cost of electricity and gas continues to rise. It is only natural that people who are struggling to eat or stay warm may also experience difficulties with a mental illness, such as anxiety or depression.

adults, people living in deprived areas and other vulnerable groups.

Protecting patients and practitioners

The Government needs to honour its commitment to publishing a ‘comprehensive’ NHS Workforce Plan this year. This should provide ways to improve retention and recruitment so that we can alleviate the pressure impacting patients and practitioners.

We are calling for NHS trusts to be supported to meet an annual 4% improvement target in retention of mental health staff and increase medical school places to 15,000 by 2028/29.

The cost-of-living crisis is also a mental health crisis. Around one in four adults who struggled to afford their rent or mortgage payments had moderate to severe depressive symptoms, according to the ONS. We also know mental illnesses disproportionately affect disabled

The College’s latest analysis found that at least £500 million of the NHS Long Term Plan’s original settlement for services, could be lost due to inflationary pressures. The Government needs to address this gap in mental health funding if it is to ensure its commitment of £2.3 billion in real terms by 2023/24. A larger and better-trained mental health workforce will save more lives, ensure everyone has access to care and reduce waiting times.

We can’t talk our way out of the chronic mental health workforce shortage

Dr Adrian James President of the Royal College of Psychiatrists

The cost-ofliving crisis is also a mental health crisis.

The need for a properly funded mental health workforce has never been greater, as millions of people across the country endure the costof-living crisis.