Therapy for a genetic type of FTD hailed as potentially ‘transformative’
A gene therapy has been hailed as potentially ‘transformative’ in stopping the progression of a genetic type of frontotemporal dementia (FTD) known as FTD-GRN. This type of FTD is caused by mutations of a gene that leads to a deficiency of progranulin (GRN), a protein that is essential for maintaining healthy brain cells.
The company working on the therapy, AviadoBio, was born out of research done at King’s College London by Professor Christopher Shaw, a neurologist who has focused on FTD and amyotrophic lateral sclerosis (ALS) for more than three decades, and Dr Youn Bok Lee and Dr Do Young Lee from the UK Dementia Research Institute’s (UK DRI) centre based at King’s.
Clinical trials in Cambridge and Cardiff, and in other cities across the world, are now recruiting participants. Professor James Rowe, consultant neurologist at Cambridge’s Addenbrooke’s hospital who is involved in the UK trial, says:
“It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives.”
Watch a film from Professor Christopher Shaw explaining how this research began: https://youtu.be/9eG6M5UPWz4?si=ER8DynJfGST7lX8v
Read more from AviadoBio here: https://aviadobio.com/aviadobio-announces-aspire-ftd-phase-1-2-clinical-trial-in-the-uk/ and from UK DRI here: https://www.ukdri.ac.uk/projects/developing-gene-therapy-treatments-neurodegenerative-diseases
Find out more about the clinical trial here: https://clinicaltrials.gov/study/NCT06064890