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.
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.
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.
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.
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.
A new clinical trial, delivered in Cardiff, will investigate the use of a one-time gene therapy to stop the disease progression in patients with frontotemporal dementia.
The ASPIRE-FTD Phase 1/2 clinical trial is recruiting patients in the UK to investigate the use of AVB-101, a gene therapy, in people with frontotemporal dementia with progranulin (FTD-GRN) gene mutations.
The trial, run by AviadoBio, will recruit patients from across Europe. The Advanced Neurotherapies Centre at Cardiff University and Cardiff and Vale University Health Board will be the UK surgical site for the trial, as the centre is the only place in the UK that can deliver drugs straight to the human brain in an MRI scanner for precise treatment and real-time imaging.
This new gene therapy, AVB-101, is a one-time therapy that has the potential to halt the progression of frontotemporal dementia. In order to fully understand its impacts on the brain, we need to overcome some of the barriers that can prevent drugs from reaching the brain and we also need precise measurement of how it’s working. At the Advanced Neurotherapies Centre, we are able to deliver drugs directly into the brain, targeting specific regions. Not only this, but we are able to do so in an MRI scanner, to get real-time imaging of the process and its impact.
AVB-101 is delivered as a one-time-only treatment using a minimally invasive stereotactic neurosurgical procedure directly to the thalamus – a key brain area affected in frontotemporal dementia.
David Cooper, Chief Medical Officer of AviadoBio, said: “Launching ASPIRE-FTD and treating our first patients with AVB-101 have been significant milestones in FTD-GRN research and gene therapy development. AviadoBio was built on pioneering research from King’s College London and the UK Dementia Research Institute. The opening of our UK clinical trial sites reflects this strong research heritage, and we’re proud to bring this clinical trial to the UK to make it more accessible to people living with familial frontotemporal in the region.”
We are delighted to be able to surgically deliver the ASPIRE-FTD trial in Cardiff, offering hope to patients living with frontotemporal dementia in the UK. The Advanced NeuroTherapies Centre is the only UK and one of two European centres able to undertake this type of work. This trial represents a major step forward in the search for a treatment in frontotemporal dementia, potentially bringing a new therapy to reality for patients.
Suzanne Rankin, Chief Executive Officer at Cardiff and Vale University Health Board, and Senior Responsible Officer for the Advanced Therapies Wales Programme, said: “It’s great to see these advanced therapies positively impacting patients across Wales. I’m proud that Cardiff and Vale University Health Board continue to contribute to this exciting research. It’s brilliant to be able to support such pioneering work, and I’m grateful to our researchers and wider team involved for their hard work and dedication.”
The Advanced Neurotherapies Centre was formerly the BRAIN Unit, hosted by Cardiff University, and is funded by Health and Care Research Wales.
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