UK research partners team up to accelerate MND drug discovery

Demyelination of neuron

UK charity LifeArc has pledged £1.5 million to fund a new two-year collaborative partnership with King’s College London (KCL) to validate a portfolio of human genetic driven therapeutic targets for motor neuron disease (MND).

The new partnership, which will involve scientists from both organisations working closely together, is 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.

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.

Amyotrophic lateral sclerosis (ALS) is the most common form of MND.

Identifying genes linked with MND

A key part of the MND Translational Challenge therapeutic strategy is to validate and de-risk MND targets using human genetic evidence. The proposed programme uses bioinformatics approaches and experimental models to validate and de-risk a portfolio of potential MND therapeutic targets. Historical pipeline data suggests that drug targets with human genetic disease association are twice as likely to lead to approved drugs.

Zhi Yao, Principal Scientist at LifeArc, explained: “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. 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.”

“Drug development is inherently risky and expensive. The MND STaR programme at King’s College London seeks to mitigate these risks by providing robust validation of therapeutic targets. Our use of big data, genomics, and extensive laboratory experiments is designed to enhance the success rate of future clinical trials and bring effective treatments for motor neuron disease closer to reality,” added Dr Ahmad Al Khleifat, the lead scientist at the King’s MND Care and Research and Centre.

Diana Spencer, Senior Digital Content Editor, DDW

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