Based on these pre-clinical findings several smaller studies in ALS patients have been initiated with promising outcomes (Table 4) and no severe adverse effects even following long-term administration (Grassinger et al., 2014; Khomenko et al., 2015) Some studies reported no beneficial effects of G-CSF for patients suffering from ALS (Nefussy et al., 2010; Chiò et al., 2011). This evidence concerns the gene CSF3 and amyotrophic lateral sclerosis.