Vinblastine significantly counteracts locomotor impairment and neurodegeneration in laboratory tests on fruit flies.
Dominant mutations in the human SPG4 gene, encoding spastin, are responsible for the most frequent form of HSP. Here we report the generation of a spastin-linked HSP animal model and show that in Drosophila [fruit fly], some aspects of the human disease including adult onset, locomotor impairment, and neurodegeneration can be replicated.
We provide evidence that administration of the drug vinblastine significantly counteracts these effects in the living flies. Our findings raise the possibility that therapy with Vinca alkaloids may be effective in spastin-associated HSP and other disorders related to microtubule dysfunction.
J Clin Invest. 2005 Nov;115(11):3026-34.
Disease-related phenotypes in a Drosophila model of hereditary spastic paraplegia are ameliorated by treatment with vinblastine.
Orso G, Martinuzzi A, Rossetto MG, Sartori E, Feany M, Daga A.
Dulbecco Telethon Institute and Department of Pharmacology, University of Padova, Padova, Italy.