Juanita Lopez, Julia Lai-Kwon, Rhoda Molife, Liam Welsh, Nina Tunariu, Desamparados Roda, Paula Fernández-García, Victoria Lladó, Adrian G McNicholl, Catalina A Rosselló, Richard J Taylor, Analía Azaro, Jordi Rodón, Julieann Sludden, Gareth J Veal, Ruth Plummer, Ander Urruticoechea, Ainhara Lahuerta, Karmele Mujika, Pablo V Escribá
BACKGROUND: The first-in-class brain-penetrating synthetic hydroxylated lipid idroxioleic acid (2-OHOA; sodium 2-hydroxyoleate), activates sphingomyelin synthase expression and regulates membrane-lipid composition and mitochondrial energy production, inducing cancer cell autophagy. We report the findings of a multicentric first-in-human Phase 1/2A trial (NCT01792310) of 2-OHOA, identifying the maximum tolerated dose (MTD) and assessing safety and preliminary efficacy. METHODS: We performed an open-label, non-randomised trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and anti-tumour activity of daily oral treatment with 2-OHOA monotherapy (BID/TID) in 54 patients with glioma and other advanced solid tumours...
July 24, 2023: British Journal of Cancer