The Advanced Neurotherapies Centre hosted the inaugural neuro multi-disciplinary research group (MDRG) meeting this month in Cardiff.

The collaborative meeting took place on 11 July and was chaired by ANTC director Professor Liam Gray.

The session brought together 37 researchers from across the neuro research community for an afternoon of discussion and networking.

The session featured 11 rapid-fire flash talks covering everything from advanced therapy medicinal product (ATMP) delivery and development to clinical trial design.

Professor Liam Gray said: “It was great to welcome colleagues to the centre here in Cardiff. The aim of the meeting was to spark fresh ideas and foster new partnerships—and it’s already working, with several exciting collaborations beginning to take shape.

“This kind of cross-disciplinary collaboration is exactly what we hoped to create. It was fantastic to see so many researchers coming together to share, connect, and innovate.”

The neuro MDRG is part of a wider series of themed meetings, organised by Advanced Therapies Wales, designed to build bridges across research areas and accelerate progress in advanced therapies.

The next neuro-focused MDRG session is set for will take place in on Friday 14 November 2025 and anyone with an interest in neurological research is warmly invited to join.

For more information or to get involved, please contact antc@cardiff.ac.uk

The event brought together researchers, clinicians, industry leaders, and individuals with lived experience of neurological conditions.

It marked a significant milestone in the evolution of the former Brain and Intracranial Neurotherapeutics (BRAIN) Unit, now transformed into ANTC with the support of a £2.9 million investment from Health and Care Research Wales.

Building on the legacy of the BRAIN Unit, the Advanced Neurotherapies Centre is dedicated to improving the delivery of life-changing medicines directly into the human brain.

As one of the few centres globally with the expertise and research excellence to tackle this challenge, ANTC is focused on the safe and effective administration of Advanced Therapy Medicinal Products (ATMPs) for treating neurological conditions.

The launch event showcased the full spectrum of the centre’s translational research, featuring keynote presentations from international research leaders, NHS partners, public involvement collaborators, and industry experts in gene and cell therapy. Attendees explored a poster exhibition highlighting the depth and breadth of ongoing work, spanning every stage of the research journey.

Professor Liam Gray, director of the Advanced Neurotherapies Centre, said: “This launch marks more than a name change—it’s a bold step forward in our mission to bring cutting-edge therapies to those living with neurological conditions.

“By uniting world-class research, clinical expertise, and the voices of those with lived experience, we are creating a centre that is truly built for impact.”

Speakers included Dr David Fluck from Cardiff and Vale University Health Board and Gareth Cross from Health and Care Research Wales, who emphasised the importance of collaborative partnerships and the unique opportunity Wales presents for developing advanced therapies for neurodegenerative diseases.

Professor Wendy Larner, Vice-Chancellor of Cardiff University, set the scene for the research environment, while ANTC director Professor Liam Gray outlined the centre’s mission and the urgent need for its work.

A trial participant, Gareth, also shared his personal perspective of taking part in a clinical trial. Read more from Gareth in his Clinical Trials Day blog post.

Professor Anne Rosser presented on the transformative potential of ATMPs in treating previously untreatable brain diseases. International guest speaker Professor Leslie Thompson from the University of California, Irvine, provided expert insight into the journey of ATMP translation.

The afternoon session featured Professor Liam Gray and Dr Dmitri Sastin discussing the critical role of Centres of Excellence and the challenges of delivering ATMPs into the brain. Dr Stephen Mittermeyer from AskBio concluded the day with an industry perspective on convention-enhanced delivery (CED), highlighting ANTC’s unique position in bridging gaps in the development pipeline.

This inspiring event set the tone for the next five years of the centre’s work and reaffirmed the power of collaboration in transforming the future of brain health.

Jo-Ann Baker, ANTC’s centre manager, concluded: “Our event was a celebration of the incredible teamwork and dedication that has brought us to this point.

“It’s also a reminder of the responsibility we carry—to push boundaries, to collaborate meaningfully, and to ensure that our research translates into real-world benefits for patients and families affected by neurological conditions.

“Thanks to everyone who joined us and contributed to a special day.”

Photography by Matthew Horwood.

The ASPIRE-FTD clinical trial will investigate the use of a gene therapy in people with frontotemporal dementia.

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.

Professor William Gray, Professor of Neurosurgery

 

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.

Professor William Gray, Professor of Neurosurgery

 

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.

Participants, funders and principal investigators came together to discuss the key findings of a recent Cardiff University study, and to present resources that people with Parkinson’s disease and their support partners can use when considering research participation.

The LEARN study stands for ‘listening to the experiences of participants taking part in neurosurgical trials’. The study sought to understand both the experiences and any barriers to participating in trials involving neurosurgery, to administer both pharmacological and advanced therapies, as well as the the understanding of clinical trials within the general Parkinson’s population.  

Researchers obtained this data through interviews with trial participants and an additional survey generated in collaboration with Cure Parkinson’s UK. The interview content was informed by a participant consultation group, ethics advisory group and an independent advisory group made up of neurosurgeons, researchers, and individuals with lived experience of Parkinson’s disease (PD).  

From analysis of the interview transcripts, researchers were able to identify key issues affecting the experience and participant’s experience which was then used create informative resources that may help improve the experience of participation in future neurosurgical trials and reduce some of the problems that might cause people to drop out of research.  

“The study has highlighted many issues that need resolution before further neurosurgical trials are designed,” said Lesley Gosden, a LEARN study participant.  

Key issues identified were challenges associated with brain imaging, taking part in clinical trials when ‘off’ medication and support for support partners, plus the need to understand and retain a lot of information about the trial.  

Chief Investigators, Dr Emma Lane and Dr Cheney Drew were pleased to introduce several information videos and written guides on each of these areas so that participants and their carers may be better supported and prepared in future trials. 

Support partner, Jayne Calder said, I believe that the LEARN study has created an opportunity to improve the journey of a participant from start to finish.”

Watch the webinar

 

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