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.
