RT001 in Progressive Supranuclear Palsy—Clinical and In-Vitro Observations

Progressive supranuclear palsy (PSP) is a progressive movement disorder associated with lipid peroxidation and intracerebral accumulation of tau. RT001 deuterium reinforced isotopologue linoleic acid that prevents (LPO) through the kinetic isotope effect. The effects pre-treatment on various oxidative bioenergetic parameters were evaluated in mesenchymal stem cells (MSC) derived from patients PSP compared to controls. In parallel, 3 treated followed clinically. MSCs had significantly higher rate LPO (161.8 ± 8.2% control; p < 0.001). A 72-h incubation restored normal levels. Mitochondrial reactive oxygen species (ROS) overproduction PSP-MSCs decreased level GSH control (to 56% 47% 0.05). Incubation increased MSCs. mitochondrial DNA was lower (67.5%), Changes membrane potential, size, shape also observed. Three subjects possible or probable for mean duration 26 months. slope PSPRS changed historical decline 0.91 points/month 0.16 (+/?0.23 SEM). UPDRS an expected increase 0.95 average score 0.28 (+/?0.41 have elevated basal levels LPO, ROS, dysfunction. These findings are reversed after RT001. patients, progression disease may be reduced by treatment