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Gene Therapy for Parkinson's Improves Motor Symptoms in Early Study — Physician’s First Watch
Gene Therapy for Parkinson's Improves Motor Symptoms in Early Study
By Joe Elia
A preliminary study of gene therapy for Parkinson disease showed improved motor symptoms, according to a Lancet report.
Under the direction of the manufacturer, researchers injected various doses of a viral vector (ProSavin) bilaterally into the putamen of 15 patients with Parkinson's. The vector carried three genes enabling the continuous production of dopamine.
During a 12-month follow-up, improvement in motor function occurred in all patients, without detectable antibody response against any of the vector products. The most common treatment-related effects were increased on-medication dyskinesias (addressed by reduction of oral dopaminergic drugs) and "on-off" phenomena. But the authors caution that the magnitude of efficacy falls within the range observed in placebo treatments during other trials.
Asked to comment, NEJM Journal Watch neurologist Michael Okun wrote: "It will be intriguing to observe how (in the planned follow-up and larger study) this approach will compare to deep brain stimulation, and also whether patients will [experience] any delayed adverse events as the result of the gene therapy."