Drug repositioning programs

Etravirine

The screening of 853 FDA-approved drugs revealed that the antiviral etravirine is able to elevate frataxin in cells derived from Friedreich ataxia patients. The mechanism of action involves the enhancement of frataxin mRNA translation. With its excellent safety profile in children, etravirine may represent a promising drug candidate for Friedreich ataxia (US patent 10,426,775).

Alfedi G, Luffarelli R, Condò I, Pedini G, Mannucci L, Massaro DS, Benini M, Toschi N, Alaimo G, Panarello L, Pacini L, Fortuni S, Serio D, Malisan F, Testi R, Rufini A. 2019. Drug repositioning screening identifies etravirine as a potential therapeutic for Friedreich’s ataxia. Mov Disord. Epub 2019, Jan 9.

Interferon gamma (IFNγ)

IFNγ elevates frataxin in multiple cell types, including cells derived from Friedreich ataxia patients. Moreover, treatment with IFNγ elevates frataxin in dorsal root ganglia neurons and ameliorates sensorimotor performances of Friedreich ataxia mice. Interferon gamma is approved for different indications. Clinical trials are currently underway to test the safety and efficacy of interferon gamma in Friedreich ataxia (US patent 8,815,230 and EU Patent 2 611 457).

Tomassini B, Arcuri G, Fortuni S, Sandi C, Ezzatizadeh V, Casali C, Condò I, Malisan F, Al-Mahdawi S, Pook M, Testi R. 2012. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum Mol Genet. Jul 1;21(13):2855-61. Epub 2012, Mar 23.