Gene-Based Approach to Treating ALS

The biology of amyotrophic lateral sclerosis (ALS) is increasingly recognized to be heterogeneous, with an important contribution from genetic susceptibility. Mutations in the superoxide dismutase (SOD1) gene are responsible for about 20% of familial cases of ALS. Aiming to translate information on the genetics of ALS into a clinical treatment, investigators now report the results of a phase I study of antisense oligonucleotides (ASOs) directed against the SOD1 gene. ASOs are synthetic nucleic acids that have been chemically modified to increase their potency and stability. In this partially manufacturer-funded, randomized, masked, placebo-controlled trial, four successive cohorts of patients with familial ALS and mutations in the SOD1 gene…