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.
Chemotherapeutic agent 5-fluorouracil increases survival of SOD1 mouse model of ALS / Rando, A.; De La Torre, M.; Martinez-Muriana, A.; Zaragoza, P.; Musaro', A.; Hernandez, S.; Navarro, X.; Toivonen, J. M.; Osta, R.. - In: PLOS ONE. - ISSN 1932-6203. - 14:1(2019), pp. 1-16. [10.1371/journal.pone.0210752]
Chemotherapeutic agent 5-fluorouracil increases survival of SOD1 mouse model of ALS
Musaro' A.;
2019
Abstract
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.File | Dimensione | Formato | |
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Rando_Chemotherapeutic_2019.pdf
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