Current Challenges in Cell Therapy for Neurodegenerative Diseases

Cell Transplantation and Gene Therapy in Neurodegenerative Disease, Volume 166 in the International Review of Neurobiology series, highlights new advances in the field with this new volume presenting interesting chapters written by an international board of authors who cover Challenges in translating a cell therapy to GMP, The challenges in developing a cell therapy for Huntington’s disease, Challenges of cell therapies for retinal diseases, Challenges of gene therapy in Huntington’s Disease, Technological advances and barriers to gene therapy, Considerations in the development of cell therapy modulation for spinal cord injury treatment, Challenges of developing glial cell therapy for ALS, and more.

Other chapters in this comprehensive release include Exploring cell and gene therapy in current animal models of Parkinson’s and Huntington’s disease, Considerations for the use of biomaterials to support cell therapy in degenerative disease, Neurosurgical challenges/innovations in cell and gene therapy delivery, Neuroimaging: the challenge of harnessing imaging tools to facilitate cell and gene therapy in neurodegenerative diseases/The contribution and challenges for imaging in advanced therapies of movement disorders, Considerations for clinical trial design for novel advanced therapeutics in neurodegenerative disease, and More than a trial participant: The role of the patient in ATMP development and trials for neurodegenerative disease.

I was promoted to Reader in Neuropharmacology in 2020 having been a Senior Lecturer since 2015. I was Director of Postgraduate Research Studies for 5 years, managing all the PhD students in the school, in particular supporting them as they navigated a PhD during the COVID pandemic.

I established my own group in 2009 as a Lecturer in Pharmacology at the School of Pharmacy and Pharmaceutical Sciences. This group has focused on Parkinson's disease and now has a profile of research in both lab and patient-facing activities.

I first came to Wales in 2006 as a PDRA working with Prof Stephen Dunnett (2006-2009) in the Brain Repair Centre, Department of Neuroscience, Cardiff University. Here I continued my work on L-dopa and post-transplantation dyskinesia started in Lund, Sweden, contributing to two successful EU FP7 grant awards.

Following my PhD (2004-2006) I was a Post-Doctoral Research Associate in the Units of Neuronal Survival and Basal Ganglia Pathophysiology, Lund University, Sweden. I spent 2 years in Sweden with Prof Patrik Brundin and Prof Angela Cenci developing, characterizing and working with a model of post-transplantation dyskinesia.

My PhD was conducted at King's College London, UK (2000-2004). I had a BBSRC CASE award studentship with Prof Peter Jenner and Dr Sharon Cheetham (then Knoll Pharmaceuticals) on the potential of BTS 74398, a monoamine uptake inhibitor, as a possible anti-parkinsonian medication in rodent models of the disease.

My undergraduate degree was a BSc in Pharmacology (1996-2000) achieving a 1st class Honours with industrial placement, University College London, UK. Specialising in neuropharmacology options and spending an industrial year at Knoll Pharmaceuticals working on the pharmacology of the anti-obesity agent sibutramine.

I am a research fellow and senior trials manager for the Mind, Brain Neuroscience study portfolio at the Centre for Trials Research in Cardiff University. I have a background in pharmacological research, with particular reference to neurodegenerative diseases, having completed my PhD in Huntington’s disease.

Since transitioning to clinical research, I have been involved in the development and delivery of trials aimed at evaluating interventions (non-pharmacological and novel advanced therapies) in people with Huntington’s disease and other neurological disorders including Tuberous Sclerosis and epilepsy.

I am member of the European Huntington’s Disease Network advanced therapies working group, aimed at devising the most efficient and robust methods for evaluating novel, non-traditional, treatments for this rare disorder.

Given the complex and experimental nature of advanced therapies, I am particularly interested in how participants are approached, informed and provide consent to involvement in these trials. This includes listening to the participant voice as a central tenet of trial design.

My PhD (2005-2010, Prof M Good) investigated neural network dysfunction using mathematical modelling in a mouse model of Alzheimer's disease, with a particular focus on amyloid-induced dysfunction in the amydala and hippocampal formation.

My post-doctoral research (2010-2015, Prof S Dunnett) aimed to understand basal ganglia dysfunction in Parkinson's and Huntington's disease and to explore the use of human fetal and novel stem cell derived therapies to alleviate the behavioural impairments.

I was awarded a Senior Research Fellowship from Parkinson's UK (2015-2018) to investigate the ability of human stem cell derived cell therapy products to ameliorate cognitive impairments in rodent models of Parkinson's disease.

I have recently commenced a Senior Lectureship in the neuroscience department and my lab is exploring several new avenues. We are seeking to establish novel cell therapies to treat Parkinson's and Huntington's diseases, using novel DREADDs manipulations, undertaking PET/MR imaging and using rabies tracing technologies. We are also studying the impact of novel gene therapies on cellular function and on cognitive dysfunction.