Current Challenges in Cell Therapy for Neurodegenerative Diseases
- 1st Edition, Volume 166 - November 16, 2022
- Imprint: Academic Press
- Editors: Emma L Lane, Cheney J.G. Drew, Mariah J Lelos
- Language: English
- Hardback ISBN:9 7 8 - 0 - 3 2 3 - 9 8 9 3 7 - 4
- eBook ISBN:9 7 8 - 0 - 3 2 3 - 9 8 9 3 8 - 1
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… Read more
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Request a sales quoteCell 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.
- Provides the authority and expertise of leading contributors from an international board of authors
- Presents the latest release in International Review on Neurobiology serials
- Updated release includes the latest information on Cell Transplantation and Gene Therapy in Neurodegenerative Disease
Undergraduates, graduates, academics, and researchers in the field of gene therapy of neurodegenerative diseases
- Cover image
- Title page
- Table of Contents
- Copyright
- Contributors
- Preface
- Chapter One: Challenges in progressing cell therapies to the clinic for Huntington's disease: A review of the progress made with pluripotent stem cell derived medium spiny neurons
- Abstract
- 1: Introduction
- 2: Derivation of MSN-like cells from hPSCs: What has been shown in vitro and in vivo?
- 3: Evidence for functional recovery following CRT and underlying mechanisms
- 4: Challenges
- 5: Conclusions and authors perspectives
- References
- Chapter Two: Challenges of cell therapies for retinal diseases
- Abstract
- 1: Introduction
- 2: Retinal disorders: Focus on AMD and RP
- 3: Human pluripotent stem cells for retinal cell therapy
- 4: Transposing bench-scale practices to industrialization
- 5: Conclusion
- Funding Statement
- References
- Chapter Three: Cell transplantation to repair the injured spinal cord
- 1: Introduction
- 2: Transplantation for spinal cord repair
- 3: Pro-regenerative transplants: Bridging for spinal cord repair
- 4: Building novel neuronal pathways: Neuronal relays for repair
- 5: Cell transplantation for neuroimmune modulation and neuroprotection
- 6: Considerations for contraindications and adverse effects
- 7: Closing remarks
- References
- Further reading
- Chapter Four: Investigating cell therapies in animal models of Parkinson's and Huntington's disease: Current challenges and considerations
- Abstract
- 1: Introduction
- 2: Challenge 1: Identifying a good model for cell transplantation studies
- 3: Challenge 2: Survival of xenografts in rodent models
- 4: Challenge 3: Assessing the functional efficacy of the graft
- 5: Challenge 4: Creating clinically relevant models
- 6: Discussion
- References
- Chapter Five: Considerations for the use of biomaterials to support cell therapy in neurodegenerative disease
- Abstract
- 1: Using biomaterials to support cell therapies
- 2: Considerations for the use of biomaterials
- 3: Promising biomaterial and cell based therapies for neurodegenerative disease
- 4: Clinical translation
- 5: Conclusion
- References
- Chapter Six: Challenges of translating a cell therapy to GMP
- Abstract
- 1: The history of cell and gene therapy manufacturing
- 2: Characterization of the NSCs
- 3: History and track record
- 4: QC/QA at the UC Davis GMP facility
- References
- Chapter Seven: Considerations for clinical trial design and conduct in the evaluation of novel advanced therapeutics in neurodegenerative disease
- Abstract
- 1: Introduction
- 2: Principles of clinical trial design
- 3: Outcome measures in cell and gene therapy trials in neurodegenerative disease
- 4: Technical and practical factors for consideration in operationalizing the intracranial delivery of cell and gene therapies
- 5: Considerations for the inclusion of participants in trials of cell and gene therapies
- 6: Summary
- References
- Chapter Eight: More than a participant in trials of cell and gene therapy: Hearing the voices of people living with neurodegenerative diseases
- Abstract
- 1: Introduction
- 2: Public and patient perceptions of cell and gene therapy
- 3: The experiences of clinical trial participants with neurodegenerative diseases
- 4: How is the voice of those living with degenerative disease engaged with?
- 5: Factors that contribute to a meaningful patient voice
- 6: The patient voice in clinical delivery
- 7: Conclusion
- Acknowledgments
- The authors
- References
- Edition: 1
- Volume: 166
- Published: November 16, 2022
- No. of pages (Hardback): 324
- No. of pages (eBook): 324
- Imprint: Academic Press
- Language: English
- Hardback ISBN: 9780323989374
- eBook ISBN: 9780323989381
EL
Emma L Lane
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.
Affiliations and expertise
School of Pharmacy and Pharmaceutical Sciences, Cardiff University, Redwood Building, UKCD
Cheney J.G. Drew
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.
Affiliations and expertise
Research Fellow - Senior Trial Manager for Mind, Brain Neuroscience, Centre for Trials Research, Cardiff University, UKML
Mariah J Lelos
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.
Affiliations and expertise
Co-director, Brain Repair Group, School of Biosciences, Cardiff University, UKRead Current Challenges in Cell Therapy for Neurodegenerative Diseases on ScienceDirect