Two autophagy modulators, colchicine, which upregulates autophagy-related proteins in motor neurons in combination with riluzole (Mandrioli et al., 2019), and the mTOR inhibitor rapamycin (sirolimus) (Mandrioli et al., 2018) are being evaluated for their therapeutic efficacy against ALS in two separate double-blind, randomized, placebo-controlled phase-II trials. The gene discussed is MTOR; the disease is amyotrophic lateral sclerosis.