Trametinib (GSK1120212, SNR1611) is a selective MEK1/2 inhibitor approved for the clinical treatment of metastatic melanoma with BRAF V600E or V600K mutations.639,640 A phase I/II clinical trial evaluating the safety, tolerability, and efficacy of trametinib in ALS patients was initiated in 2020 (ClinicalTrials.gov identifier: NCT04326283), aiming to assess whether suppressing the MAPK/ERK pathway by trametinib is of benefit for ALS, and the trial is currently recruiting participants. The gene discussed is MAP2K1; the disease is amyotrophic lateral sclerosis.