Parkinson's disease (PD) is a degenerative neurological disorder marked by a progressive loss of motor control. Despite intensive research, there are currently no approved therapies that have been demonstrated to alter the progression of the disease.
In this issue of the Journal of Clinical Investigation, Dr. Thomas Foltynie and colleagues at the National Hospital for Neurology and Neurosurgery in London investigated the use of a drug approved for diabetes care, Exenatide, in PD patients. PD patients were divided into two groups: 20 patients received Exenatide injections for 12 months, while the other group of 24 patients served as controls.
Due to high manufacturing costs, the control group did not receive placebo injections and the patients were aware of their group assignment. Foltynie and colleagues observed that Exenatide was well tolerated. After one year of treatment patients receiving Exenatide displayed improved cognitive ability and motor skills, while control patients declined.
Though this trial cannot rule out a placebo effect, the study suggests that Exenatide may improved motor function in PD patients and provides a strong rationale for conducting a larger, blinded study to determine the effectiveness of Exenatide in PD.
In the accompanying commentary, Roger Barker (University of Cambridge), Mark Stacy (Duke University) and Patrik Brundin (Van Andel Institute) discuss the novel, cost-saving clinical trial design used in this study.
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Exenatide and the treatment of patients with Parkinson's disease, J Clin Invest. doi:10.1172/JCI68295
A new approach to disease-modifying drug trials in Parkinson's disease, J Clin Invest. doi:10.1172/JCI69690