SOD1 and amyotrophic lateral sclerosis: Another pharmacological strategy targeting SOD1 involved the use of oral pyrimethamine, an FDA-approved medication used for the treatment of malaria and toxoplasmosis; in the clinical study, NCT01083667 demonstrated that pyrimethamine significantly lowered cerebrospinal fluid (CSF) SOD1 levels in ALS patients with SOD1 mutations, with a mean reduction of 13.5% at 18 weeks and 10.5% at 36 weeks.