Exenatide as a Treatment for Patients with Parkinson’s Disease

Objective/Rationale:             
Laboratory data from several teams have indicated potential beneficial effects of the drug Exenatide on the survival of dopamine cells. In a small group of patients with Parkinson’s disease (PD) who injected Exenatide twice daily for a year, the drug was tolerated well and improvements were seen in physical and cognitive functions compared to patients not given the drug. Further exploration is needed to identify whether Exenatide can indeed slow the progression of PD.

Project Description:             
This randomized, double-blind, placebo-controlled, proof-of-concept trial aims to confirm whether the preliminary findings of beneficial effects seen in both motor and non-motor PD symptoms can be replicated. Sixty patients with PD will be randomly assigned to self-administer a slow-release version of Exenatide or matched placebo once weekly for 12 months. Levels of the drug will be measured in blood and spinal fluid, and biological effects on Parkinson’s will be assessed using clinical scales, diaries and scans of dopamine transporter availability (DaTscan). The study will include a washout period at the end to help distinguish whether any changes seen are purely related to symptom relief or are related to effects on the neurodegenerative process. 

Relevance to Diagnosis/Treatment of Parkinson’s Disease:
Currently there are no treatments confirmed to have beneficial effects on slowing or reversing the progression of PD. Identifying agents that can usefully modify the progression of Parkinson’s arguably represents the major priority for research in this field.

Anticipated Outcome:          
The results from this project will provide high-quality data necessary to decide whether Exenatide has useful biological effects in patients with PD. Positive results will form the platform upon which major Phase III trials can be planned and conducted to ultimately enable the regulatory authorities to judge whether the safety and tolerability and biological effects of Exenatide support its widespread use in patients with PD.

Additional Support:
The Michael J. Fox Foundation would like to acknowledge the generous contribution of the Demoucelle Parkinson Charity as a lead supporter providing funding for this project.