Europe-wide gene therapy trial being delivered in Cardiff

A groundbreaking Europe-wide gene therapy trial, delivered in Wales by the Advanced Neurotherapies Centre, funded by Health and Care Research Wales, could potentially stop the progression of frontotemporal dementia (FTD) with a single procedure.

Patients across the continent are being recruited to the ASPIRE-FTD trial, which will be delivered at Cardiff and Vale University Health Board. The Advanced Neurotherapies Centre is currently the only centre in the UK, and one of just two across Europe, able to perform MRI-guided infusions of gene therapies, providing the capability to dose clinical trial participants from across the UK and beyond.

This first in-human trial, sponsored by AviadoBio, aims to evaluate an investigational gene therapy, AVB-101, in people with frontotemporal dementia with GRN gene mutations (FTD-GRN). 

FTD is a form of early-onset dementia, the most common form in people under the age of 60. It accounts for around one in 30 cases of dementia in the UK. Symptoms include reduced mobility, changes in personality or uncharacteristic behaviour, progressive loss of language and loss of executive function and cognitive abilities. 

People with FTD-GRN produce a reduced amount of a protein called progranulin. The investigational therapy uses a minimally-invasive procedure to deliver a functional copy of the GRN gene directly to the brain. This could potentially restore progranulin levels and, crucially, stop progression of the disease.

Michael Bowdery, Head of Programmes, Research and Development Division at Welsh Government, said: “This is a really exciting study which has the potential to be absolutely transformative in the treatment of this devastating form of dementia. 

“Thanks to funding from Health and Care Research Wales, the Advanced Neurotherapies Centre – and its predecessor, the BRAIN unit – over nearly a decade, Wales has the capacity and capability to deliver this cutting-edge clinical trial which has the potential to change lives for patients across Europe and the UK.

“We are very proud that the Advanced Neurotherapies Centre is spearheading the delivery of this trial in the UK.”

Professor William Gray, PI, Neurosurgeon at Cardiff University and Cardiff and Vale University Health Board and Health and Care Research Wale Senior Research Leader, said, “We are delighted to be able to surgically deliver AVB-101 in the ASPIRE-FTD trial in Cardiff, offering hope to patients living with FTD in the UK and beyond. This trial represents a major step forward in the search for a treatment in FTD, potentially bringing a new therapy to reality for patients.

“In order to fully understand the impact of AVB101 on the brain, we need to overcome some of the barriers that can prevent drugs from reaching the brain. 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.”

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."