SOD1 and amyotrophic lateral sclerosis: These encouraging preclinical data prompted a phase 1, double-blind, placebo-controlled clinical trial (NCT01041222) to evaluate the safety and tolerability of intrathecally delivered ASO 333611 in SOD1 ALS patients [68], making ASO 333611 the first experimental antisense drug to be administered intrathecally to patients for the treatment of a neurodegenerative disease.