King's College London

King’s College London is proud to announce a new collaborative partnership with LifeArc to accelerate the development of treatments for motor neuron disease (MND). LifeArc has pledged £1.5 million to fund this two-year initiative, which aims to validate a portfolio of human genetic-driven therapeutic targets for MND within the new UK MND Research Institute.

The programme, named MND STaR (Solution for Motor Neuron Disease Target Validation and Research) focuses on early-stage preclinical validation, addressing a critical area in MND research that has been identified as an area of ‘translational gap’ in the MND field. Therefore, a key part of the MND Translational Challenge therapeutic strategy is to validate and de-risk MND targets using with human genetic evidence. The proposed programme uses cutting-edge bioinformatics approaches and a wide range of experimental models to validate and de-risk a portfolio of potential MND therapeutic targets, ultimately aiming to integrate well validated targets into downstream drug discovery portfolios.

De-risking novel targets to a stage where they are ready for drug discovery efforts has significant potential for paving the way for new, potentially disease-modifying, MND treatments. Historical pipeline data suggests that drug targets with human genetic disease association are twice as likely to lead to approved drugs.

Dr Ahmad Al Khleifat, Senior Research Fellow at King's IoPPN and lead scientist at the King’s MND Care and Research and Centre said, "The program combines King’s College London and LifeArc expertise to find treatments for Motor Neuron Disease using human genetics. This approach aims to pinpoint precise treatment targets, to cut costs and to improve outcomes, pushing forward effective solutions for MND."

Ammar Al-Chalabi, Professor of Neurology and Complex Disease Genetics, co-Director of the UK MND Research Institute said, "This collaborative project will reduce risks in drug development, allowing us to find and test the most promising treatments more quickly and reliably."

The new partnership, which will involve scientists from both organisations working closely together, is an integral part of LifeArc’s MND Translational Challenge, aimed at discovering innovative and potentially disease -modifying treatments for MND, a key component of the new UK MND Research Institute.

Zhi Yao, Principal Scientist at LifeArc, said "If we can identify the human genes linked to the condition it is more likely that treatments will succeed. Our plan is to use cutting-edge bioinformatics approaches and a wide range of experimental models to validate and de-risk a portfolio of potential MND therapeutic targets."

Dr Alfredo Iacoangeli, Reader in Bioinformatics said, “In this LifeArc partnership, King’s College London brings its extensive experience in genetics and genomics to the forefront. We use state-of-the-art computational methods and advanced bioinformatic tools developed right here at King’s to analyse omics data and to dissect the complexity of our targets’ underlying biology.”

Dr Bradley Smith, Lecturer in Dementia and Neurodegeneration said, “Our work focuses on analysing brain samples and cell lines to identify the most promising genes for MND treatment. We aim to find specific genetic markers in brain tissues and confirm their presence in more accessible cell lines. This helps us ensure that we are targeting the right genes for effective treatments.”

Dr. Jackie Mitchell, Lecturer in in vivo Modelling of Neurodegeneration said, “Our work in the lab focuses on replicating the complexities of MND in experimental models. This allows us to test and refine potential treatments in conditions that closely mimic the human disease, enhancing the likelihood of successful outcomes in clinical trials.”

 

For more information, please contact Annora Thoeng (Communications Manager - School of Neuroscience)