It has been announced today that Addenbrooke’s Hospital is one of the sites participating in an innovative, international trial of a novel gene therapy that offers the potential of a cure for patients with a particular inherited form of frontotemporal dementia (FTD).
FTD is the leading cause of dementia in those under the age of 65 years. It is a devastating form of early-onset dementia that varies in each individual. Symptoms can include changes in personality or uncharacteristic behaviours, progressive loss of language, loss of executive function and cognitive abilities, apathy, and reduced mobility.
The ASPIRE-FTD trial is investigating a therapy that could stop FTD progressing. It aims to repair a gene in the brain known as progranulin (GRN), which is altered in up to a quarter of families with inherited FTD. A condition known as FTD-GRN. It is the first time this treatment has been trialled in patients.
As an internationally renowned centre of excellence, providing support and care for people and families affected by FTD, Cambridge University Hospitals NHS Foundation Trust (CUH) is one of three participating sites in the UK.
The potential to halt FTD-GRN with a single procedure could be truly transformative for patients and their families. Combining excellence in clinical care with cutting-edge research is vital to driving progress in this extremely challenging field. Through studies like this we hope to one day be able to provide a cure.
Professor James Rowe, Consultant Neurologist at Addenbrooke’s Hospital and Professor of Cognitive Neurology at the Cambridge Centre for Frontotemporal Dementia
Eligible patients will undergo a one-off procedure to deliver the gene therapy, called AVB-101, into the right parts of the brain. The procedures will be performed at the Advanced Neurotherapies Centre at Cardiff and Vale University Health Board in Wales.
We are delighted to be able to deliver the ASPIRE-FTD trial in Cardiff, offering hope to patients living with FTD in the UK. This trial represents a major step forward in the search for a treatment in FTD, potentially bringing a new therapy to reality for patients.
Professor William Gray, Local PI and Neurosurgeon at Cardiff University and Cardiff and Vale University Health Board
The trial is being sponsored by AviadoBio a London-based gene therapy company. Other locations currently involved in the trial include the US, Poland, Netherlands, Spain and Sweden.
Find out about how you can take part in research at CUH with Love Research.