title: Olesoxime (TRO19622): A Novel Mitochondrial-Targeted Neuroprotective Compound.
abstract: Olesoxime (TRO19622) is a novel mitochondrial-targeted neuroprotective compound undergoing a pivotal clinical efficacy study in Amyotrophic Lateral Sclerosis (ALS) and also in development for Spinal Muscular Atrophy (SMA). It belongs to a new family of cholesterol-oximes identified for its survival-promoting activity on purified motor neurons deprived of neurotrophic factors. Olesoxime targets proteins of the outer mitochondrial membrane, concentrates at the mitochondria and prevents permeability transition pore opening mediated by, among other things, oxidative stress. Olesoxime has been shown to exert a potent neuroprotective effect in various in vitro and in vivo models. In particular olesoxime provided significant protection in experimental animal models of motor neuron disorders and more particularly ALS. Olesoxime is orally active, crosses the blood brain barrier, and is well tolerated. Collectively, its pharmacological properties designate olesoxime as a promising drug candidate for motor neuron diseases.
authors.affiliation: Trophos, Parc Scientifique de Luminy, Case 931, 13288 Marseille cedex 9, France. tbordet@trophos.com.
authors.forename: Thierry
authors.initials: T
authors.lastname: Bordet
authors.affiliation: Trophos, Parc Scientifique de Luminy, Case 931, 13288 Marseille cedex 9, France. pberna@trophos.com.
authors.forename: Patrick
authors.initials: P
authors.lastname: Berna
authors.affiliation: Trophos, Parc Scientifique de Luminy, Case 931, 13288 Marseille cedex 9, France. jlabitbol@trophos.com.
authors.forename: Jean-Louis
authors.initials: JL
authors.lastname: Abitbol
authors.affiliation: Trophos, Parc Scientifique de Luminy, Case 931, 13288 Marseille cedex 9, France. rpruss@trophos.com.
authors.forename: Rebecca M
authors.initials: RM
authors.lastname: Pruss
keywords: TRO19622; amyotrophic lateral sclerosis; mitochondrial permeability transition pore; motor neuron disease; neuroprotection; olesoxime; spinal muscular atrophies