In a presentation entitled "Subthalamic GAD gene transfer improves brain metabolism associated with clinical recovery in Parkinson's Disease," Matthew J. During, MD, D.Sc. presented findings of the open label, dose escalating, unilateral trial, which confirmed the safety and tolerability in all 12 patients studied out to one year. Though efficacy was only designated as a secondary outcome, the trial also yielded statistically significant clinical efficacy and neuro-imaging results.
At one year, all 12 patients as a group demonstrated a clinical improvement of 25% in the Unified Parkinson's Disease Rating Scale (UPDRS) compared to baseline (p < 0.005). Nine of the 12 patients showed an average improvement of 37%, and 5 of these patients had substantial improvement of between 40% and 65%. "This gene therapy trial is particularly unique and the clinical data unusually promising because the treatment was confined to just one side of the brain," stated Dr. During. In its next trial, the Company plans to infuse its treatment into both sides of the brain.
Clinical improvement also correlated well to metabolic brain changes as measured by Positron Emission Tomography (PET) scan. PET is an imaging method that measures brain metabolism following the injection of a radioactive analog of glucose (fluorodeoxyglucose). The PET scan data revealed a significant improvement (p < 0.001) in brain metabolism on the treated side of the brain as compared to the untreated side.
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