Centre for Neurodegeneration Research

Lead Research Organisation: King's College London
Department Name: Unlisted

Abstract

We will extend and enhance our current activities which in 2004 included an open day for patients, carers, school students and other interested members of the lay public. We produce a newsletter for participants in one clinical study. Members of the Centre Faculty engage in both print and broadcast media on a regular basis. Further details are described in the main description of the proposed Centre s plans.

Technical Summary

The understanding of the molecular pathogenesis of neurodegenerative diseases has advanced to such a degree that translation of this research into clinically relevant outcomes is a reality. The mission of the Centre will be to further understand mechanisms of neurodegeneration and to translate this into new treatments, new biomarkers and improved understanding of the signs and symptoms of these diseases.

Publications

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Al Khleifat A (2019) Telomere length is greater in ALS than in controls: a whole genome sequencing study. in Amyotrophic lateral sclerosis & frontotemporal degeneration

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Bajc Cesnik A (2019) Nuclear RNA foci from expansion mutation form paraspeckle-like bodies. in Journal of cell science

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Balendra R (2014) Estimating clinical stage of amyotrophic lateral sclerosis from the ALS Functional Rating Scale. in Amyotrophic lateral sclerosis & frontotemporal degeneration

 
Title Paintings about dementia 
Description Paintings depicting aspects of dementia and research to find a cure 
Type Of Art Artwork 
Year Produced 2018 
Impact Inspiring people working in the field 
 
Description Chair of review of DZNE
Geographic Reach Europe 
Policy Influence Type Participation in a advisory committee
Impact Improved diagnostics, research impact, drug trials and improved therapeutics
URL https://www.dzne.de/
 
Description Sumission to Parliamentary Science and Technology Committee
Geographic Reach National 
Policy Influence Type Gave evidence to a government review
Impact Permitted the use of hybrid embryos in research in UK
 
Description Identification of compounds that enhance TDP43 clearance in ALS and FTD
Amount £462,000 (GBP)
Organisation Eli Lilly & Company Ltd 
Sector Private
Country United Kingdom
Start 12/2015 
End 05/2020
 
Description MNDA studentship
Amount £87,502 (GBP)
Organisation Motor Neurone Disease Association (MND) 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2010 
End 09/2013
 
Description Testing chaperone gene therapy in a mouse model of Motor Neuron Disease and Fronto-Temporal Dementia
Amount £120,000 (GBP)
Organisation Peter Samuel Trustees 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2017 
End 09/2021
 
Description Viral vector gene therapy for FTD and ALS: from constructs to clinical trials
Amount £2,012,076 (GBP)
Organisation UK Dementia Research Institute 
Sector Charity/Non Profit
Country United Kingdom
Start 01/2019 
End 08/2022
 
Title Adeno-associated viral vector manufacture 
Description Using AAVVectors to express genes in cultured cells and in mammals (mice, sheep) 
Type Of Material Technology assay or reagent 
Year Produced 2019 
Provided To Others? No  
Impact Able to present data for further funding 
 
Title High-Density Surface Electromyography 
Description Using high density surface electrodes to record fasciculations in patients with motor neuron disease and benign fasciculation syndrom as a potental predictor of disease course 
Type Of Material Model of mechanisms or symptoms - human 
Year Produced 2018 
Provided To Others? Yes  
Impact Multiple publications and sharing of methodology and tool on line 
URL https://spiqe.co.uk
 
Description Exome sequencing in motor neuron disease: bioinformatic analyses and biological validation of novel variants 
Organisation Motor Neurone Disease Association (MND)
Country United Kingdom 
Sector Charity/Non Profit 
PI Contribution ALS research excellence
Collaborator Contribution financial
Impact Publication of new ALS genes
Start Year 2015
 
Description From ALS exomes to Functional assays: turning candidates into confirmed genes 
Organisation Medical Research Council (MRC)
Department Medical Research Foundation
Country United Kingdom 
Sector Charity/Non Profit 
PI Contribution Scientific expertise on ALS pathogenesis
Collaborator Contribution financial support
Impact None yet as started 5 months ago
Start Year 2015
 
Description Generation and characterisation of induced pluripotent stem cells from ALS lymphoblasts to model disease pathogenesis and advance drug discovery 
Organisation Motor Neurone Disease Association (MND)
Country United Kingdom 
Sector Charity/Non Profit 
PI Contribution Research excellence
Collaborator Contribution Financial and access to lymphoblast cell lines
Impact Nil yet as just started
Start Year 2015
 
Description Identification of compounds that enhance TDP43 clearance in ALS and FTD 
Organisation Eli Lilly & Company Ltd
Department Neuroscience Eli Lilly
Country United States 
Sector Private 
PI Contribution Identify therapeutic targets for drug discovery
Collaborator Contribution Expertise in drug discovery
Impact About to start
Start Year 2016
 
Description Investigating the role of autophagy and the UPS in TDP-ALS 
Organisation Motor Neurone Disease Association (MND)
Country United Kingdom 
Sector Charity/Non Profit 
PI Contribution ALS pathobiology
Collaborator Contribution Financial Support
Impact None
Start Year 2015
 
Description RDC5 screen in cellular and animal models of TDP-43 proteinopathy 
Organisation Chronos Therapeutics
Country United Kingdom 
Sector Private 
PI Contribution ALS pathobiology
Collaborator Contribution Compounds to be tested in cellular and animal lmodels
Impact None yet
Start Year 2015
 
Description Remote home assessment of patients with amyotrophic lateral sclerosis: a multimodal integrative approach 
Organisation Imperial College London
Country United Kingdom 
Sector Academic/University 
PI Contribution We have developed a robust means of quantifying fasciculations n people with motor neuron disease and we linked this up with the Remote Disease monitoriting system developed by David Sharp at Imperial
Collaborator Contribution Integrated multimodal home monitoring system
Impact Just starting
Start Year 2020
 
Description Vertex Pharmaceutical 
Organisation Vertex Pharmaceuticals
Country United States 
Sector Private 
PI Contribution Developing a cellular screening platform to identify small molecule correctors of the TDP-43 pathology
Collaborator Contribution Funding to develop Cellular assay for high throughout screening
Impact None so far
Start Year 2013
 
Title Clinical Trial of antisense oligonucleotide drug targeting the C9orf72 mRNA given intrathecally 
Description Clinical Trial of antisense oligonucleotide drug targeting the C9orf72 mRNA given intrathecally 
Type Therapeutic Intervention - Drug
Current Stage Of Development Early clinical assessment
Year Development Stage Completed 2019
Development Status Under active development/distribution
Clinical Trial? Yes
Impact First in man trial of a drug targeting this genetic population 
URL https://clinicaltrials.gov/ct2/show/NCT03626012
 
Title SURFACE POTENTIAL QUANTIFICATION ENGINE 
Description Open to anyone 
Type Of Technology Software 
Year Produced 2018 
Open Source License? Yes  
Impact Software available to other researchers and industry 
URL https://spiqe.co.uk/#
 
Company Name NEUROGENEUS LTD 
Description Designi and manufacture of gene therapies for neurodegenerative disorders 
Year Established 2019 
Impact None yet as just formed
 
Description MRC Centre Open Day 
Form Of Engagement Activity Participation in an open day or visit at my research institution
Part Of Official Scheme? No
Primary Audience Participants in your research and patient groups
Results and Impact Our Open Day is for patients and carers with approximately 150 attendees. We organise 8 laboratory demonstrations presented by PhD students and postdoctoral researchers around which small groups (6-8 persons) circulate. We also have a group of about 12 sixth form students who perform a laboratory experiment. At the end of the day the sixth formers present their results to the who audience and this is followed by an "Any Questions" session with clinical and non-clinical experts answering questions from the visitors.

The Open Day has now been run on three occasions and each time has received universal acclaim from visitors. The report is also made available on our web site.
Year(s) Of Engagement Activity 2008
 
Description Many media interviews TV, Radio, Newspapers, Radio 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Primary Audience Public/other audiences
Results and Impact Many media interviews TV, Radio, Newspapers, Radio

Legislation modified
Year(s) Of Engagement Activity 2008
 
Description Multi-site Phosphorylation Assays for Tau Protein and their relevance to Alzheimer's disease and other neurological disorders 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Primary Audience Participants in your research and patient groups
Results and Impact Poster presentation at American Society of Mass Spectrometry conference

None
Year(s) Of Engagement Activity 2008
 
Description Patient and Carers meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? Yes
Primary Audience Participants in your research and patient groups
Results and Impact Each meeting has 40-60 attendees and lasts 1.5 hours with talk and questions

Patients have come to our laboratory open day and are actively fundraising to create a studentship
Year(s) Of Engagement Activity 2006,2007,2008,2009,2010
 
Description Patient and Carers meeting 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Primary Audience Participants in your research and patient groups
Results and Impact 40-60 Patients and Carers lasting 1.5 hours of talk and discussion

Members of the audience are fundraising for a studentship for my laboratory
Year(s) Of Engagement Activity 2006,2007,2008,2009,2010
 
Description Presented to Members of Parliament 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Primary Audience Policymakers/politicians
Results and Impact Presented to parliamentary parties

Legeislation changed
Year(s) Of Engagement Activity 2008
 
Description School visit Peterborough 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Primary Audience Schools
Results and Impact Lectured to school students and informal chat

Enthusiastic discussion
Year(s) Of Engagement Activity 2006
 
Description Sleep and circadian rhythm disorder in Parkinson's Disease: association with hallucinations 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Primary Audience Participants in your research and patient groups
Results and Impact A world conference on sleep.

Received a citation award.
Year(s) Of Engagement Activity 2007
 
Description Talk at Dementia Discovery Fund Conference 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Presentation on opportunities for Gene Therapy in motor neuron disease and fronto-temporal dementia
Year(s) Of Engagement Activity 2019
URL https://theddfund.com/
 
Description Talk to MND Association of New Zealand annual general meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Patients, carers and/or patient groups
Results and Impact Public Presentation
Year(s) Of Engagement Activity 2019
URL https://mnd.org.nz/inaugural-mnd-new-zealand-research-conference/
 
Description The Use of Mass Spectrometry for Multi-site Phosphorylation Assays for Tau Protein 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Primary Audience Participants in your research and patient groups
Results and Impact Poster presentation at International Conference on Alzheimer's Disease, Chicago

None
Year(s) Of Engagement Activity 2008