MeiraGTx announced publication of research assessing the mechanism underlying the motor improvement observed in patients with Parkinson’s disease who were treated with adeno-associated virus, or AAV, encoding glutamic acid decarboxylase, or AAV-GAD, an investigational gene therapy product candidate, in a completed Phase 2 study. In the new research, which was published in the November 28 issue of Science Translational Medicine, patients treated with AAV2-GAD gene therapy expressed a distinct treatment-related metabolic brain network, providing a clinical benefit by inducing the formation of new polysynaptic pathways connecting the STN to cortical motor regions. Rather than act through conventional motor pathways involving certain regions of the brain, AAV-GAD gene therapy was observed to co-opt adjacent “nonmotor” regions in the treated patients. The research published in Science Translational Medicine showed that patients treated with AAV-GAD gene therapy developed a unique treatment-dependent metabolic brain network, which reflected the formation of new functional pathways linking the STN to motor cortical regions. GADRP was characterized by increased metabolism in certain brain regions along with relatively reduced metabolic activity in other brain regions. The research also showed that the GADRP correlated with clinical improvement in the gene therapy-treated subjects as measured by changes in the Unified Parkinson’s Disease Rating Scale motor ratings. In contrast, the clinical correlation with sham surgery-related pattern was not significant.
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