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 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.

The goal of this clinical study is to learn about an investigational gene therapy product called AVB-101, which is designed to treat a disease called Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GRN is an early-onset form of dementia, a progressive brain disorder that affects behaviour, language and movement. These symptoms result from below normal levels of a protein called progranulin (PGRN) in the brain, which leads to the death of nerve cells (neurons), affecting the brain’s ability to function.

The main questions that the study aims to answer are:

  • Is a one-time treatment with AVB-101 safe for patients with FTD-GRN?
  • Does a one-time treatment with AVB-101 restore PGRN levels to at least normal levels?
  • Could AVB-101 work as a treatment to slow down or stop progression of FTD- GRN?

In this study there is no placebo (a dummy pill or treatment used for comparison purposes), so all participants will receive a one-time treatment of AVB-101 delivered directly to the brain, with follow-up assessments for 5 years.

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.”