LifeArc and King’s College London (KCL) have announced a collaborative partnership to accelerate treatment for motor neurone disease (MND) by validating a portfolio of human genetic-driven therapeutic targets for the condition.
Supported by £1.5m in funding from LifeArc, the two-year programme will work in alignment with LifeArc’s MND Translational Challenge, launched in December 2023, to discover innovative and potential disease-modifying treatments for the rare neurological condition.
Affecting one in every 300 people in the UK, MND is a neurodegenerative disease that affects the nerve cells in the brain and spinal cord.
Within a year of diagnosis, around one-third of patients with MND die and more than half within two years.
The Solution for MND Target Validation and Research (MND STaR) programme, focusing on early-stage preclinical validation, aims to address a key area in MND research known as the ‘translational gap’.
Within the new UK MND Research Institute, the programme will validate and de-risk MND targets using human genetic evidence, cutting-edge bioinformatic approaches and a variety of experimental models to integrate well-validated targets into downstream drug discovery portfolios.
First launched in November 2023, the MND Research Institute in London aims to discover a cure and new treatments for the condition, bringing together MND labs, clinical centres and researchers.
Previous pipeline data has suggested that drug targets with human genetic disease associations are twice as likely to lead to approved drugs.
By de-risking novel targets to be ready for discovery efforts, findings could pave the way for new, potentially disease-modifying MND treatments.
Zhi Yao, principal scientist, LifeArc, said: “We want to ensure that before we embark on drug discovery and development, we are confident the targets we are aiming for are truly promising.
“If we can identify the human genes linked to the condition, it is more likely that treatments will succeed.”
Dr Ahmad Al Khleifat, lead scientist, KCL MND Care and Research Centre, commented: “The MND STaR programme… seeks to mitigate these risks by providing robust validation of therapeutic targets… to enhance the success rate of future clinical trials and bring effective treatments for MND closer to reality.”