First patient treated in clinical study designed to create new brain circuits, improve symptoms
Posted on 12/7/2022
MORGANTOWN, W.Va. – The West Virginia University Rockefeller Neuroscience Institute today announced the initiation of a clinical trial evaluating the safety and efficacy of a new innovative gene therapy in the treatment of Parkinson’s disease. While surgical implants used in deep brain stimulation can treat Parkinson’s disease, gene therapy can be of particular benefit without the need for implants in the brain.
“This new study is the next step from a previous sham-controlled study that showed promise in Parkinson’s disease,” Ali Rezai, M.D., executive chair of the WVU Rockefeller Neuroscience Institute, said. “In this study, AAV-GAD gene therapy is delivered to the subthalamic nucleus, a key area with dysfunction in a brain with Parkinson’s disease. Over time, the gene therapy is intended to help Parkinson’s disease by making new brain connections and improving symptoms.”
The randomized, double-blind, sham-controlled clinical trial is sponsored by MeiraGTx, a gene therapy innovation leader.
“AAV-GAD is an experimental gene therapy to evaluate safety and treatment regimen using material manufactured with our proprietary production process that could support future commercialization,” Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx, said. “We are excited to be working with the WVU Rockefeller Neuroscience Institute to move forward this innovative treatment for Parkinson’s disease, and we look forward to reporting on the progress of the trial.”
The first participant is a grandparent of 14 from West Virginia who was forced to retire because of the progression and severity of Parkinson’s disease. Medications had become less effective with worsening side effects. They hope their participation in this study not only helps them find relief, but also leads to advances in treatment for others with Parkinson’s.
“I hope this gene therapy will help my Parkinson’s and also help other people,” the participant said.
For more information on the study, refer to ClinicalTrials.gov identifier NCT05603312.
About the Rockefeller Neuroscience Institute
The WVU Rockefeller Neuroscience Institute improves lives by pioneering advances in brain health and therapeutics. With the latest technologies, innovative research, an ecosystem of partners, and a multidisciplinary integrated approach to patient care, the RNI is making tangible progress in its goal to combat public health challenges ranging from addiction to Alzheimer’s. For more information about the WVU Rockefeller Neuroscience Institute, visit WVUMedicine.org/RNI.
