title: Exenatide and the treatment of patients with Parkinson's disease.
abstract: BACKGROUND. There is increasing interest in methods to more rapidly and cost-efficiently investigate drugs that are approved for clinical use in the treatment of another condition. Exenatide is a type 2 diabetes treatment that has been shown to have neuroprotective/neurorestorative properties in preclinical models of neurodegeneration. METHODS. As a proof of concept, using a single-blind trial design, we evaluated the progress of 45 patients with moderate Parkinson's disease (PD), randomly assigned to receive subcutaneous exenatide injection for 12 months or to act as controls. Their PD was compared after overnight withdrawal of conventional PD medication using blinded video assessment of the Movement Disorders Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS), together with several nonmotor tests, at baseline, 6 months, and 12 months and after a further 2-month washout period (14 months). RESULTS. Exenatide was well tolerated, although weight loss was common and l-dopa dose failures occurred in a single patient. Single-blinded rating of the exenatide group suggested clinically relevant improvements in PD across motor and cognitive measures compared with the control group. Exenatide-treated patients had a mean improvement at 12 months on the MDS-UPDRS of 2.7 points, compared with mean decline of 2.2 points in control patients (P = 0.037). CONCLUSION. These results demonstrate a potential cost-efficient approach through which preliminary clinical data of possible biological effects are obtainable, prior to undertaking the major investment required for double-blind trials of a potential disease-modifying drug in PD.
  
  
  BACKGROUND
  Clinicaltrials.gov NCT01174810.
  
  
  BACKGROUND
  Cure Parkinson's Trust.
authors.affiliation: Sobell Department of Motor Neuroscience, UCL Institute of Neurology, London, United Kingdom.
authors.forename: Iciar
authors.initials: I
authors.lastname: Aviles-Olmos
authors.forename: John
authors.initials: J
authors.lastname: Dickson
authors.forename: Zinovia
authors.initials: Z
authors.lastname: Kefalopoulou
authors.forename: Atbin
authors.initials: A
authors.lastname: Djamshidian
authors.forename: Peter
authors.initials: P
authors.lastname: Ell
authors.forename: Therese
authors.initials: T
authors.lastname: Soderlund
authors.forename: Peter
authors.initials: P
authors.lastname: Whitton
authors.forename: Richard
authors.initials: R
authors.lastname: Wyse
authors.forename: Tom
authors.initials: T
authors.lastname: Isaacs
authors.forename: Andrew
authors.initials: A
authors.lastname: Lees
authors.forename: Patricia
authors.initials: P
authors.lastname: Limousin
authors.forename: Thomas
authors.initials: T
authors.lastname: Foltynie
mesh_terms: D000368:Aged; D000978:Antiparkinson Agents / Q000008:administration & dosage* / Q000009:adverse effects; D003248:Constipation / Q000139:chemically induced; D018450:Disease Progression; D058492:Drug Repositioning; D000077270:Exenatide; D005260:Female; D006801:Humans; D007004:Hypoglycemic Agents / Q000008:administration & dosage / Q000009:adverse effects; D007279:Injections, Subcutaneous; D008297:Male; D008875:Middle Aged; D009043:Motor Activity / Q000187:drug effects; D056687:Off-Label Use; D010300:Parkinson Disease / Q000188:drug therapy* / Q000503:physiopathology; D010455:Peptides / Q000008:administration & dosage* / Q000009:adverse effects; D014688:Venoms / Q000008:administration & dosage* / Q000009:adverse effects; D015431:Weight Loss / Q000187:drug effects
chemical_list: D000978:Antiparkinson Agents; D007004:Hypoglycemic Agents; D010455:Peptides; D014688:Venoms; D000077270:Exenatide