We have synthesized various compounds with high affinities for the sigma-1 receptor. From investigating the features of these sigma-1 ligands, we selected one agonist, MC116 as a backup for cutamesine. While cutamesine is a highly selective sigma-1 agonist, MC116 shows affinity for other neurotransmitter receptors as well.
MC116 could potentially be used as a therapeutic drug for numerous diseases; however, we selected Parkinson’s disease as the first target disease. This disease is a CNS disorder in which dopaminergic neurons are mainly damaged. Recent papers have reported that mitochondrial dysfunction is involved in the etiology of this disease. Since one of the basic physiological functions of the sigma-1 receptor is to stimulate mitochondrial function, there is strong rationale for the use of a sigma-1 receptor agonist for the treatment of Parkinson’s disease. In addition, we have already confirmed the efficacy of MC116 in a Parkinson’s disease animal model.
Currently available drugs for Parkinson’s diseases, such as levodopa and dopamine agonists, ameliorate symptoms, but do not affect the cause of the disease. As MC116 is expected to stop or impede the progression of Parkinson’s disease, this is a novel drug concept for Parkinson’s disease therapy.