title: Potential compounds for the treatment of mitochondrial disease.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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.
  
  
  RESULTS
  Previous clinical research has been hindered by poorly designed trials that have shown some evidence in enhancing mitochondrial function but without significant results.
  
  
  RESULTS
  Several compounds under investigation display poor pharmacokinetic profiles or numerous off target effects.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research, Institutes of Neuroscience and Cellular and Molecular Bioscience, Newcastle University Medical School, Newcastle University, Newcastle upon Tyne, Tyne and Wear, UK.
authors.forename: P K
authors.initials: PK
authors.lastname: Rai
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research, Institutes of Neuroscience and Cellular and Molecular Bioscience, Newcastle University Medical School, Newcastle University, Newcastle upon Tyne, Tyne and Wear, UK.
authors.forename: O M
authors.initials: OM
authors.lastname: Russell
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research, Institutes of Neuroscience and Cellular and Molecular Bioscience, Newcastle University Medical School, Newcastle University, Newcastle upon Tyne, Tyne and Wear, UK.
authors.forename: R N
authors.initials: RN
authors.lastname: Lightowlers
authors.affiliation: Wellcome Trust Centre for Mitochondrial Research, Institutes of Neuroscience and Cellular and Molecular Bioscience, Newcastle University Medical School, Newcastle University, Newcastle upon Tyne, Tyne and Wear, UK doug.turnbull@newcastle.ac.uk.
authors.forename: D M
authors.initials: DM
authors.lastname: Turnbull
mesh_terms: D000818:Animals; D004579:Electron Transport / Q000187:drug effects; D006706:Homeostasis / Q000187:drug effects; D006801:Humans; D008928:Mitochondria / Q000187:drug effects; D028361:Mitochondrial Diseases / Q000188:drug therapy*; D058990:Molecular Targeted Therapy / Q000379:methods*; D019636:Neurodegenerative Diseases / Q000188:drug therapy; D001678:Organelle Biogenesis; D015388:Organelles / Q000187:drug effects; D056811:Unfolded Protein Response / Q000187:drug effects
keywords: clinical trials; mitochondrial disease; oxidative phosphorylation; therapeutic intervention; treatment