EXPERTS-ALS completes a bench to bedside ALS drug pipeline, enabling prioritisation of the many candidates arising from University- and industry-based research for use in Phase III studies, such as the UK’s MND-SMART platform and the pan-European TRICALS consortium. The platform is divided into three workstreams. 

WORKSTREAM 1: Investigational medicinal product (IMP) identification

Aim: To deliver a continuous pipeline of drugs for workstream 2 using the best available evidence from pre-clinical models and a wide range of data resources, including genomics, AI drug discovery programs and evidence from other neurodegenerative diseases.

Primary objective: To select the most appropriate compounds for prioritisation in EXPERTS-ALS, based on disease relevance, safety profile and central nervous system penetration.

Secondary Objectives: Data from workstream 3 in response to drugs can be fed back to preclinical modelling in workstream 1 to identify new, or refine existing, pathways of disease relevance, and ultimately those which best predict a positive outcome in Phase III trials.

WORKSTREAM 2: Drug Prioritisation Platform 

Aim: To evaluate the effect of candidate drugs on blood NfL levels in a multi-centre open label study as the basis for prioritising formal testing in a Phase III randomly controlled trial (RCT). 

Primary objectives

To determine:

Secondary objectives:

WORKSTREAM 3: Nested biomarker development studies

Aim: To study the changes of the molecular environment in biofluids that occur with disease progression, in parallel with alterations observed in NfL concentration. To develop additional biomarkers of disease activity and drug response.

Primary objectives:

Secondary objective: Work towards integration into the broader UK MND Research Institute (MNDRI) initiative to create a national clinical data and biological samples repository through NIHR and Telehealth in MND (TiM) platforms.