Ellen P. Neff. Dis. Model Mech.11, dmm033811 (2018) doi:10.1038/s41684-018-0143-3
Friedreich’s ataxia (FA) is a degenerative disorder caused by mutations to the mitochondrial protein frataxin. Muscles, including those of the heart, waste over time, and cardiomyopathy is the leading cause of death. Researchers at Universite Paris Diderot previously described a frataxin-deficient Drosophila line that models FA cardiomyopathies. In their latest paper, they use their fly model to screen 1280 drugs that have been approved for use in humans.
Some drugs made things worse—five were toxic—but eleven improved cardiac function in the flies; most effective was the chemotherapy drug paclitaxel. Paclitaxel stabilizes microtubules; in the heart, these cytoskeletal fibers contribute to proper cardiac function. The drug can be toxic so the authors don’t recommend it for therapeutic use, but they suggest that its efficacy indicates a novel mechanism to investigate further.
Wednesday, February 13, 2019
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