Resumen: Amyotrophic lateral sclerosis (ALS) is a lethal motor neuron disease with no cure. Currently there are only two ALS drugs approved by the FDA, both with a limited therapeutic effect. In the search for drug candidates for ALS, we studied the effect of known stem cell mobilizing agents (treatment) and antimetabolite 5-fluorouracil (5-FU) (anti-treatment) in SOD1G93A model of ALS. Surprisingly, we found that anti-cancer drug 5-FU increases lifespan, delays the disease onset and improves motor performance in ALS mice. Although we were not able to demonstrate the mechanistic basis of the beneficial 5-FU action in ALS mice, our findings suggest that 5-FU or similar drugs are possible drug candidates for the treatment of motor neuron diseases through drug repurposing. Idioma: Inglés DOI: 10.1371/journal.pone.0210752 Año: 2019 Publicado en: PLoS ONE 14, 1 (2019), e0210752 [16pp] ISSN: 1932-6203 Factor impacto JCR: 2.74 (2019) Categ. JCR: MULTIDISCIPLINARY SCIENCES rank: 27 / 71 = 0.38 (2019) - Q2 - T2 Factor impacto SCIMAGO: 1.023 - Multidisciplinary (Q1)