Neurologix, Inc., recently announced at the annual meeting of the American Association of Neurological Surgeons (AANS) that their work with the glutamic acid decarboxylase (GAD) gene which synthesizes the inhibitory neurotransmitter, gamma-aminobutyric acid (GABA), in the subthalamic nucleus (STN), is nearing completion of Phase I clinical trials. This is the first such gene therapy that has received FDA approval. The study is an open-label dose-escalation study in which groups of trial participants will receive increasing dosages. The participants were chosen from among people who have had severe Parkinson's Disease for a minimum of five years and for whom all traditional medical therapies have not resulted in any improvement of their condition or symptoms.
The procedure requires stereotactical surgery (similar to procedures used in Deep Brain Stimulation) in which the subthalamic nucleus is localized using MRI and the insertion of a microelectrode to monitor signature STN neuron firings, and a small tubule is then used to deliver 35 microliters containing the AAV (adeno associated virus) viral vector and GAD gene (cDNA) directly into the localized region.
Read more about it in the article, Phase I trial of Gene Therapy to Treat Parkinson's Disease Nears Completion in the RxPG News, the news channel for medical professionals, or visit the Neurologix, Inc. Web site for more info.
