New guiding principles to overcome barriers to MND drug discovery

Demyelination of neuron

New guidance for the motor neuron disease (MND) drug discovery community has been published to ensure that future drug discovery and development efforts have the best chance of successfully translating from the lab to the clinic.

The whitepaper ‘Guiding principles for drug discovery and development in amyotrophic lateral sclerosis’ outlines a consensus-based approach to de-risk clinical trials of novel therapeutics by an enhanced evidenced-based approach to pre-clinical studies.

Despite recent leaps forward in our understanding of MND biology, treatment options remain limited.

A cross-sector working group of MND experts was convened by MND Association and My Name’5 Doddie Foundation and coordinated by Medicines Discovery Catapult throughout 2022. The group examined barriers to translating basic science into effective therapies for MND and suggested guiding principles for future research.

Prof Kevin Talbot, Head of Department and Professor of Motor Neuron Biology at the University of Oxford, said: “We have generated recommendations for the optimisation and conduct of pre-clinical studies to improve confidence that a drug will show efficacy in human trials. We hope this guidance will catalyse high quality pre-clinical drug discovery efforts and create a shared vision for all stakeholders involved in MND drug discovery research.”

The Guiding Principles underpin a new £5 ($6.2) million funding call from LifeArc, which aims to identify medicines used in other diseases and repurpose them for MND.

MND drug repurposing fund

The fund is part of LifeArc’s multi-million pound Motor Neuron Disease Translational Challenge to make MND treatable by 2030 and ultimately find a cure.

International research teams in academia and small and medium-sized enterprises (SMEs) are eligible to take part in the programme, with funding of up to £750,000 ($944,176) per project available. To be considered eligible for funding, applicants must have identified a new repurposed drug with experimental evidence that shows it has potential for treating MND.

The chosen projects will benefit from LifeArc’s deep expertise, advice and mentorship in translational research, its drug-repurposing know-how and access to its collaborative network of project partners.

Dr Paul Wright, Head of LifeArc’s MND Translational Challenge, said: “We are fortunate to have exceptional research in the MND community both in the UK and further afield but despite this, most clinical trials using repurposed drugs fail due to lack of effectiveness. Improving confidence in drug repurposing requires more robust preclinical research, meaning that repurposed drug candidates are progressed into trials with sufficient evidence to predict they could benefit someone living with MND. To achieve our ambition of making MND treatable by 2030, we need to start with better decision making at the pre-clinical stage to demonstrate as early as possible that a repurposed drug candidate has the potential to deliver improvements for patients.”

A parallel funding scheme to support data science and AI/machine learning approaches to repurposed drug candidate identification in MND will also be made available through the programme. LifeArc will fund up to five bioinformatic projects that aim to exploit MND data sets with up to £100,000 ($125,890) available per project over 12 months.

Diana Spencer, Senior Digital Content Editor, DDW

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