[Comment] Examining the evidence for IL-2 in amyotrophic lateral sclerosis
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterised by progressive muscle weakness. With the possible exception of a superoxide dismutase (SOD1)-targeting antisense oligonucleotide for patients with ALS caused by a pathogenic variant in the SOD1 gene,1 there are no meaningfully effective therapies for this otherwise invariably fatal disease, despite a long and rich history of clinical trials in the field.2,3 Importantly, phase 2 trials have been poorly predictive of phase 3 outcomes, in part because the design and interpretation of phase 2 trials have often been susceptible to false positive discovery.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterised by progressive muscle weakness. With the possible exception of a superoxide dismutase (SOD1)-targeting antisense oligonucleotide for patients with ALS caused by a pathogenic variant in the SOD1 gene,1 there are no meaningfully effective therapies for this otherwise invariably fatal disease, despite a long and rich history of clinical trials in the field.2,3 Importantly, phase 2 trials have been poorly predictive of phase 3 outcomes, in part because the design and interpretation of phase 2 trials have often been susceptible to false positive discovery.
