Guiding Principles for ALS Drug Discovery and Development
SUMMARY
Since 2020, three UK-based organisations, Medicines Discovery Catapult, MND Association and My Name’5 Doddie Foundation, have been working together to galvanise the cross-sector ALS research community to identify key opportunities and challenges to accelerating drug discovery. A key challenge identified was the need for more consensus on what constitutes a robust preclinical pathway and data package in ALS.
To address this, an Expert Steering Group was convened over a period of 9 months. The Group discussed the factors governing the barriers to translating advances in basic science into effective therapies and suggested Guiding Principles for de-risking clinical trials of novel therapeutics by an enhanced evidenced-based approach to preclinical studies.
To enable community consensus on these Guiding Principles, a workshop of leading global experts in ALS drug discovery and development was convened in June 2022. Before the workshop, a White Paper outlining the Guiding Principles proposed by the Expert Steering Group was circulated to all delegates. Delegates were invited to complete a survey to provide their input on whether they agreed with the proposed Principles.
