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Publication : Chemotherapeutic agent 5-fluorouracil increases survival of SOD1 mouse model of ALS.

First Author  Rando A Year  2019
Journal  PLoS One Volume  14
Issue  1 Pages  e0210752
PubMed ID  30640943 Mgi Jnum  J:270311
Mgi Id  MGI:6275959 Doi  10.1371/journal.pone.0210752
Citation  Rando A, et al. (2019) Chemotherapeutic agent 5-fluorouracil increases survival of SOD1 mouse model of ALS. PLoS One 14(1):e0210752
abstractText  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.
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