Potential compounds for the treatment of mitochondrial disease

Abstract

Introduction: Mitochondrial diseases are a group of heterogeneous disorders for which no curative therapy is currently available. Several drugs are currently being pursued as candidates to correct the underlying biochemistry that causes mitochondrial dysfunction.

Sources of data: A systematic review of pharmacological therapeutics tested using in vitro, in vivo models and clinical trials. Results presented from database searches undertaken to ascertain compounds currently being pioneered to treat mitochondrial disease.

Areas of agreement: Previous clinical research has been hindered by poorly designed trials that have shown some evidence in enhancing mitochondrial function but without significant results.

Areas of controversy: Several compounds under investigation display poor pharmacokinetic profiles or numerous off target effects.

Growing points: Drug development teams should continue to screen existing and novel compound libraries for therapeutics that can enhance mitochondrial function. Therapies for mitochondrial disorders could hold potential cures for a myriad of other ailments associated with mitochondrial dysfunction such as neurodegenerative diseases.

Keywords: clinical trials; mitochondrial disease; oxidative phosphorylation; therapeutic intervention; treatment.