Clinical Trials Day (20 May) is an annual opportunity to reflect on all that has been accomplished thanks to clinical trials and the people behind them. In this blog, Gareth has kindly shared his experience of taking part in a neurosurgical advanced therapy trial.

Gareth’s story

Hi, I’m Gareth. Last year, I took part in a neurosurgical clinical trial testing a new gene therapy for Huntington’s disease (HD).

It was a big decision; one that surprised many of my family and friends but one that I’m proud I made. To that end, I wanted to share a bit about what the journey was like, why I chose to say yes and why I would hope others will also choose to embrace advanced therapies.

Before the trial

I was diagnosed HD gene positive in September 2021. This wasn’t altogether a surprise as I had witnessed several members of my family suffer at the hands of this dreadful condition.

Quite soon afterwards, I was invited to the Huntington’s clinic in Cardiff for a chat with Professor Anne Rosser. We talked openly about HD and what options, though limited, were available.

I was quite uplifted by this information, not realising the huge amount of research that was going on behind the scenes.

I decided that I wanted to help in any way shape or form and left the clinic far more hopeful than when I arrived.

Deciding to take part

My decision to take part in my trial was quite simple in the end, but I was certainly helped by background information I had gathered through ongoing meetings with Professor Rosser and my involvement with the HD Advisory Board, which I am a proud member of.

Whilst my initial motivation was personal, I soon became aware of the much wider influence that Advanced Therapies could have on so many lives.

The following consultations with Professor Liam Gray and his team only further enhanced my determination to get involved.

I appreciated the honesty and respect shown by everyone and I felt a high level of trust immediately. This connection was vital for me and helped erase any doubts in my mind.

The clinical trial experience

Committing to a trial isn’t just about the surgery – I learned that quite quickly after numerous scans, examinations and blood tests. Each part of the process is vital, and I could sense the collective commitment to this project from every individual team member.

Even the hospital staff, though not directly involved, showed huge interest and enthusiasm towards this ground-breaking treatment. This certainly put me at ease as, quite honestly, I am not a fan of hospitals!

Whilst I can’t recall the surgery, I do remember the moment I woke up with a sense of relief and an element of surprise about how good I felt.

The aftercare was immediate and detailed, and I felt comfortable and well looked after.

Reflections

Taking part in the trial gave me hope, and I’m proud I did it.

Even if it hadn’t helped me personally, knowing I contributed to something that might help others was worth it.

I’d encourage anyone offered the chance to be part of research to at least find out more. If I was asked to do it again, I would.

Advanced Therapies offer a new and exciting route into tackling previously untreatable conditions, and I feel privileged to have experienced it first-hand.

Thank you to Gareth for sharing his story with us.

The BRAIN Unit, which will become the Advanced Neurotherapies Centre from April 2025, is pleased to confirm that it will receive £2,856,309 of sustainability funding in a major announcement by Health and Care Research Wales this week.

Health and Care Research Wales has announced Research Development Infrastructure funding for 17 research centres across Wales, including five new organisations.

The funding has been awarded across two categories – sustainability awards, for currently funded groups to maintain effective models of practice and support a trajectory towards self-sustainability, and catalytic awards, to boost capacity and capability in areas of health and care need and emerging Welsh research strength.

Professor Liam Gray, BRAIN Unit director, said: “We are delighted to build on the achievements of the last 10 years as the BRAIN Unit, consolidating and expanding our position as the lead UK centre for the delivery of advanced therapies directly to the human brain.

“From April, we will become the Advanced Neurotherapies Centre, continuing to develop and deliver ground-breaking treatments for the benefit of patients living with neurological and neurodegenerative disease. Thank you to Health and Care Research Wales for their ongoing support.”

The full list of centres that will receive funding are:

Sustainability funding 

Catalytic funding

Jeremy Miles, Cabinet Secretary for Health and Social Care, said: “Research has a critical role to play in helping us to achieve our aim of A Healthier Wales. This is an important investment in new and exciting areas of research, including women’s health; preventing suicide and self-harm and AI – I hope it provides real evidence over the next five years, which will help shape services and care for people across Wales.”

Michael Bowdery, Joint Interim Director at Health and Care Research Wales and Head of Programmes, Research and Development Division at Welsh Government, said: “This announcement represents a significant investment in our funded infrastructure in Wales over the next five years, and reflects our ambition to advance research capability aligned with unmet health and social need in key policy areas.

“Our approach to providing this funding is based on two criteria – firstly, where there is a clear and compelling research and evidence need in the area for Welsh Government, the NHS and social care system in Wales; and secondly, where there is demonstrably strong or emerging research capacity and capability in the area.

“These centres embody the principle of research having the power to make a difference to people’s health and wellbeing, and we are pleased to be able to support their activity in this field.”

For more information visit: www.healthandcareresearchwales.org

BRAIN Unit director Professor Liam Gray spoke at a recent event hosted by the British Neuroscience Association (BNA) at the Senedd on Tuesday 17 September 2024.

The event, titled ‘Neuroscience and Mental Health Research: Spotlight on Wales’, aimed to emphasise the importance of neuroscientific research to tackle health challenges in Wales to policymakers.  

 Government ministers, funders and other researchers from across Wales gathered to hear about a variety of neuroscientific research, including that of the BRAIN Unit’s who are developing and delivering advanced therapies for neurodegenerative diseases such as Huntington’s disease, which affects around 7000 people in the UK. Currently, the BRAIN Unit is one only five centres worldwide delivering a potentially game-changing gene therapy  UniQure AMT-130 for people with Huntington’s Disease.   

Professor Gray said, “We are at a pivotal and exciting stage in neuroscience research, with Advanced Cell and Gene Therapies holding significant promise for disease modification in heretofore untreatable neurological conditions. We are one of the few centres in the world performing these early phase clinical trials, providing the evidence needed for their translation into clinical care. ” 

Looking to the future, Professor Gray announced that the BRAIN Unit has plans to rebrand in April 2025. The unit will be known as the Advanced Neurotherapies Centre and will continue to work towards being a Centre of Excellence for the development and delivery of advanced therapeutics with trials planned for dementia and Parkinson’s Disease.  

Learn more about the future of the BRAIN Unit in our annual report.

 

On Thursday 10 October 2024, Health and Care Research Wales hosted its annual conference in Sophia Gardens, Cardiff.

The topic of this year’s event was ‘research matters’, and the drew researchers, clinicians and public involvement figures from across the country.

BRAIN Unit Director, Professor Liam Gray, attended this year’s conference as a panellist, discussing how collaboration across sectors drives investment and bring greater benefit to patients in Wales.

Professor Gray had the opportunity to discuss the significant achievements of the BRAIN Unit, how collaboration between BRAIN and partners, including the NHS has resulted in some world leading advanced neurotherapy clinical trials taking place here in Wales.

Hailing from Ireland, Professor Gray explained that unique collaborative opportunities drew him to Wales. He also announced plans for the BRAIN Unit as it becomes the Advanced Neurotherapies Centre and its ongoing aim to become a Centre of Research Excellence, having been awarded another five years of funding from the Health and Care Research Wales infrastructure.

Attendees also had the chance to find out more about BRAIN on its interactive stand throughout the day, with a new clinical trials snakes and ladders game which allowed participants to better understand the challenges and rewarding results of the clinical trials process.

We look forward to returning to next year’s conference as the Advanced Neurotherapies Centre and we will be sharing more information on our new name soon.

Ataxia is a neurodegenerative condition which affects one in 50,000 people. This is Alan’s story, founder of a charity called Ataxia and Me.

What is ataxia?

Ataxia comes from the Greek word, meaning ‘lack of order’. People with ataxia experience problems with movement, balance and speech. Although ataxia is degenerative (and this very slow in my case), there is currently no cure for this life-limiting condition.

Rare conditions affect less than one in 2,000 people; ataxia affects one in 50,000! This means that ataxia is extremely rare, and it is difficult to connect with others who share lived experience of this condition.

My cerebellar ataxia diagnosis

I have walked with a wobble and spoke with a slur from an early age. Many people thought, “Oh, that’s just Alan!”. I also achieved good qualifications at school and went on to run my own electrical contracting business. However, as my condition progressed I soon found out that lack of coordination does not mix with electrical circuitry. Still, I persevered until I started to notice that menial tasks required more and more concentration – even buttering my sandwiches for work.

For many years, I visited my GP on numerous occasions who told me to take time off work and rest. One day, my usual GP was unavailable, and I saw a locum who had recent knowledge of ataxia symptoms. He referred me for more neurological tests and I received a diagnosis for cerebellar ataxia. The consultant explained the diagnosis, its outcomes and that nothing could be done.

A support network for ataxia

When I got home, I started to research ataxia in more detail and began to find that there wasn’t much helpful information or support out there. Whilst I receive some care from my local hospital, I have to travel to Sheffield to receive specialist ataxia care – some 258 miles away, and five hours by train!

That was when I established Ataxia and Me, a patient-led charity based in West Wales with a global following. Together, we share our lived experiences of this extremely rare condition.

If you are an ataxia patient or relative, carer, friend or healthcare professional, we invite you to support us in promoting awareness and sharing helpful resources. Just go to ataxia-and-me.org.

 

Alan Thomas

Founder of Ataxia and Me

Follow us on X and Instagram: @ataxia_and_me /  @Atacsia_a_fi

 

 

Dr Chloe Ormonde is a lab technician at the BRAIN Unit. In this piece, Chloe shares her career journey so far and why she enjoys working in the laboratory.

About me

I’m Chloe, and I’ve worked for the university for over 16 years. I studied at Cardiff University myself, completing a BSc degree in Pharmacology and a PhD in cardiovascular sciences. I joined the laboratory group of Professor William Gray and the BRAIN Unit at the end of 2016. When I’m not in the lab, I’m a busy mum to two children, a son who is five and a daughter who has just turned 16 months. My time outside of work is normally spent exploring the various playgrounds around Cardiff, building LEGO and practicing ‘first words’ with my daughter. I’m a keen cook, and I enjoy experimenting and trying out new recipes with my family.

My role at the BRAIN Unit

A laboratory technician has the responsibility of overseeing all activities that take place within the research team. On a typical day, we assist in preparing for collection, and the processing of primary human tissue, which is obtained from consented patients undergoing elective neurosurgery at the University Hospital of Wales (UHW), Cardiff. Under the Human Tissue Act, we are responsible for keeping track of all human tissue activities taking place within our research team, and keeping inventories regularly updated.

We also provide technical support for our research group and are responsible for tasks such as ordering and stock checking, protocol generation, formulation of risk assessments and standard operating procedures, as well as providing laboratory support by training new members of staff and students to work safely within the lab and ensuring the correct usage of laboratory equipment.

Why are lab technicians so important?

The technician role is integral to the research team. It is important because we carry out practical and technical support to all staff and students working within the laboratory. We deliver an array of essential, routine laboratory techniques to support any ongoing scientific research projects and are involved in receiving, labelling, and analysing samples. We are responsible for making sure the laboratory remains a user-friendly space, by keeping the lab benches clean and clutter free, disposing of rubbish and keeping track of laboratory consumables such as reagents and plasticware.

What I love about being a lab technician

I’ve always enjoyed tissue culture studies and the fact the BRAIN Unit works directly with human tissue collected straight from surgery really piqued my interest in this role. I was intrigued to see how a 3D representation of normal and diseased brain tissue could be generated in the laboratory. I was also interested in the exciting prospect of using this tissue not only to closer investigate the complex processes that underpin neurological disease, but also as a model to predict the clinical efficacy of drug treatments. These processes help us to identify resistance, toxicity, and aid therapeutic strategies.

With thanks to Chloe for her contributions.

The BRAIN Unit were delighted to support the launch of the new Huntington’s Disease Centre in Wales which took place in Cardiff University’s Hadyn Ellis Building on Wednesday 8 March, 2024.

Huntington’s Disease (HD) is an inherited neurodegenerative disease which causes brain cells to be lost, impacting thinking, movement, behaviour and mental health.

During a warm welcome to the launch event, Professor Anne Rosser explained that the launch of the Huntington’s Disease Centre in Wales marks a new era for HD research. The aim is to bring together researchers across multiple disciplines, institutions and sectors across Wales for collaboration and to promote world leading research into HD.

The local breadth and depth of expertise in HD research from fundamental understanding of disease biology to the clinical testing of new therapies provides a great opportunity to achieve the ultimate goal of finding treatments that can slow down or stop HD progression. The centre also recongises the importance of continuing to provide support to people and families living with HD.  Therefore, research into reducing the impact of HD on families, working alongside key patient-focussed organisations also features as a key theme within the centre.

The centre will continue to work alongside its collaborators within Cardiff University, which include the BRAIN Unit, National Neuroscience and Mental Health Innovation Institute (NMHII), the Centre for Neuropsychiatric Genetics and Genomics (CNGG) and the Cardiff University Brain Research Imaging centre (CUBRIC).

The BRAIN Unit looks forward to building on the existing foundation of excellence in HD through collaborative activity.

 

On Saturday 20 April, BRAIN co-hosted an event with the National Centre for Mental Health (NCMH) to celebrate the importance of patient and public involvement (PPI) in research.

Whilst BRAIN is developing and trialling advanced therapies for neurodegenerative disease and NCMH is looking into the causes of mental health disorders, both are united in one cause: ‘Working together for better brain health’.

 

“PPI is the most important part of the health research puzzle…”

Public and patient involvement is an integral part of BRAIN and NCMH’s research, as it brings both researchers and members of the public together to help shape and inform the direction of research. This makes research outcomes more reliable, more relevant, and more likely to be used to improve health and social care services.

On the day of the event, BRAIN and NCMH PPI groups had a chance to meet in person and attend a guided lab tour to learn more about how human samples are used in research.

Later on, we welcomed members of the wider public to network with researchers and take part in interactive ‘brain games’. Attendees took part in a variety of discussions from what getting involved in brain health research looks like to an interview with Principal Investigator and BRAIN director, Professor William Gray.

NCMH PPI member, Jacqueline Campbell interviewed Professor Gray on his experiences as a neurosurgeon and working in partnership with PPI groups.

Professor Gray said, “Getting the chance to go into someone’s brain and make a positive difference is such a privilege.”

“It’s important that people who have these conditions have a voice on how these trials are conducted. They are advocates for continuing research in these areas.”

All fun and brain games

Attendees had the opportunity to test and broaden their knowledge of brain health research through an array of interactive stands and brain-related games run by centres from across the Division of Psychological Medicine and Clinical Neurosciences in Cardiff University.

Researchers from NMHII ran two interactive games for guests, including the opportunity to practise their hand at pipetting and guessing different animal brain sizes in a game called ‘Whose brain is it anyway?‘.

Attendees try their hand at pipetting.

Staff based in the Centre for Neuropsychiatric Genetics and Genomics demonstrated how the power of poetry can be used to express experiences of different mental health and brain disorder diagnoses through writing Cinquain poems.

There were also Virtual Reality headset experiences, Pin the ball on the brain, and the ever-popular Stroop Mat.

We also asked guests to take part in our Paint a brain activity which demonstrated how different parts of the brain are responsible for different functions and processes, such as movement and memory.

 

Soapbox scientists

As well as hearing personal PPI stories, attendees were invited to discover more about specific research areas in the division and pose questions to our ‘soapbox scientists’.

These short talks ranged from the genetics of treatment resistant schizophrenia, the use of virtual reality (VR) in healthcare, and how memory can be impacted by mental illness.

BRAIN Involve lead, Dr Cheney Drew said, “Both BRAIN and NMCH are focused on ensuring that the public and patient voice remains at the heart of the research we carry out. Today’s event enabled us to continue those important conversations between researchers and public contributors. It is important for us to feedback to our public contributors the value they have within the team and the impact they have on research. The event today was a really enjoyable way of being able to do that.”

We’d like to thank all of our attendees for their great insight and contributions on the day.

 

 

 

 

 

 

 

The BRAIN Unit and National Centre for Mental Health (NCMH) will co-host an event on Saturday 20 April to celebrate the role patient and public involvement groups play in research.

The event will bring together public and patient involvement groups, researchers and the general public who share one common cause: working together for better brain health.

Patient and public involvement (PPI) groups are a vital partnership between members of the public and researchers to make research more relevant, reliable and more likely to be implemented into our health and social care services.

This event will allow attendees to have their say on what public involvement means to them, hear personal stories from BRAIN and NCMH PPI group members, and take part in interactive brain-related activities and panel discussions. We are also offering research lab tours (booking essential),.

This is a free event and we welcome anyone with an interest in brain research and PPI to join us.

Find out more and register your place today.

On Thursday 7 December, our BRAIN Involve lead Dr Cheney Drew delivered a public lecture on the use of advanced therapies in Huntington’s and Parkinson’s Disease.

The lecture was part of the Science in Health public lecture series, hosted by the School of Medicine at Cardiff University. The free public lecture series welcomes a diverse audience, from the public and secondary school pupils to professionals.

In the lecture, Dr Drew spoke about how advanced therapies (ATMPs) can be used as an alternative to traditional drug therapies to treat neurodegenerative disease.

ATMPs fall into three main categories:

Clinical trials in ATMPs such as gene therapy and cell replacement therapy are currently underway to see if they are safe and effective to use in people with neurodegenerative diseases such as Huntington’s and Parkinson’s.

Dr Drew explained exactly how these therapies are used and what clinical trials research currently tells us about their effectiveness, safety and patient experience.

“Clinical trials can be really difficult for people to take part in. It’s really important that we not only monitor for outcome assessment and safety but also understand participant experiences and support their ability to advocate for future therapies.”

Catch up on the lecture and register for the next Science in Health public lecture.

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