Panel 4.
Priority areas for amyotrophic lateral sclerosis (ALS) research
| • Understanding of the development of cortical and spinal motor neuron populations, their interaction with each other, and how it changes with normal ageing and in ALS |
| • Factors determining rate of disease progression |
| • Development of in-vitro and in-vivo models that accurately mimic ALS-related protein misfolding, aggregation, and accumulation |
| • Identification of the complete ALS-related genome |
| • Identification of the earliest, presymptomatic characteristics of ALS through the collaborative identification and study of relatives of patients with familial ALS |
| • Identification and standardisation of a biomarker for presymptomatic or early ALS and incorporation (with a view to validation) of at least one biomarker candidate in future therapeutic trials |
| • High-throughput compound screens in cellular and animal models of ALS (including zebrafish and worm models) |
| • Identification of possible environmental factors through large-scale international collaboration |