«Repurposing ciclopirox as a pharmacological chaperone for the treatment of congenital erythropoietic porphyria»Oscar Millet (CIC-BIOGUNE, Bilbao)

Congenital erythropoietic porphyria is a serious, rare autosomal recessive disease produced by a deficient activity in the uroporphyrinogen III synthase. The disease impacts in many organs, as toxic uroporphyrin by-products from the frustrated biosynthesis accumulate. In here, we demonstrate that the antimicrobial ciclopirox associates to the enzyme and stabilizes it. The drug alleviates most of the clinical signs in a mouse model of the disease, establishing a novel therapeutic intervention line. 

After obtaining his Ph.D. in Organic Chemistry (University of Barcelona, 1999), Oscar Millet joined the group of Lewis Kay in Toronto as a post-doct. He later became a Ramon y Cajal researcher at the Parc Cientific de Barcelona (2004-2006). Currently he is group leader at the CIC bioGUNE, where he focuses on the use of NMR to study biologically relevant proteins and enzymes, paying special attention to the delicate balance existing between protein stability and dynamics.