LifeArc MND Translational Challenge: Preclinical Discovery and Development of Repurposed Drug Candidates for MND

Funding call issued through the Drug Repurposing Programme of the LifeArc MND Translational Challenge to support the preclinical translation of repurposed drug candidates and drug candidate combinations for motor neuron disease, with a focus on a robust, evidence-based approach to optimise pre-clinical decision making to ensure that only the most promising repurposed drug candidates are progressed into clinical trials.

The LifeArc Translational Challenge in MND is a multi-million pound programme that aims to accelerate scientific innovation for people living with motor neuron disease (MND). The challenge aims to address various strategic gaps in the development of therapies for MND and will include multiple calls, funded by LifeArc and other partners, to support research which aims to reduce the diagnostic delay experienced by people with MND, enable those receiving a positive diagnosis to access treatment sooner, and facilitate earlier recruiting to clinical trials to increase the chances of trial success and developing new disease modifying treatments.

‘Preclinical Discovery and Development of Repurposed Drug Candidates for MND’ is a £5 million call to support the preclinical translation of repurposed drug candidates and drug candidate combinations for MND. The main objective of the call, in addition to funding preclinical translational research, is to develop a robust, evidence-based approach to optimise pre-clinical decision making to ensure that only the most promising repurposed drug candidates are progressed into clinical trials.

Accordingly, proposals will focus on the generation of high-quality preclinical data packages and independent replication of data – two significant factors that are critical for drug discovery and successful clinical trials, but are historically under-funded in academia.

Projects within scope of this call include:

• Preclinical stage projects for MND using a repurposed drug or drug combination.
• Applications that can demonstrate evidence for a repurposed drug or drug combination and a relevant MND drug target.
• Earlier stage applications with target validation plans in the first milestone to evaluate repurposed drugs or drug combinations against a relevant MND drug target.
• Projects that also include biomarker guided approaches for repurposed drug-target engagement, mechanism and efficacy (applicants are encouraged to consider the utilisation of biomarkers that may be suitable for future clinical research studies).

Projects should aim to generate repurposed drug candidate preclinical data packages for target validation, efficacy, safety and PK/PD studies in vivo to demonstrate appropriate target tissue exposure and target engagement, and also include the validation of available biomarkers of repurposed-drug target engagement for MND.

Research outputs will contribute to building a strong body of evidence to enable evidence-based decision making on which repurposed drugs should enter into biomarker guided clinical studies and clinical trial platforms for MND – including the existing UK platforms EXPErimental medicine Route to Success in Amyotrophic Lateral Sclerosis (EXPERTS-ALS), and MND systematic multi arm adaptive randomised trial (MND-SMART).