New gene therapy could help address motor symptoms in patients with Parkinson’s disease, says GlobalData

One of the unmet needs in patients with Parkinson’s disease (PD) is the treatment of motor complications; the ability of new gene therapy to form new neural pathways connecting the subthalamic nucleus (STN) of the brain to the motor cortical regions may potentially help address this gap in the market, says GlobalData, a leading data and analytics company.

Results from an ongoing Phase II clinical study have revealed the ability of glutamic acid decarboxylase (GAD) gene therapy to create new brain circuits in patients with PD. The study was led by the Feinstein Institute of Medical Research and involved 35 patients, 15 of whom were given the AAV2-GAD gene therapy and remaining were assigned a sham surgery.

Vinie Varkey, MSc, Senior Neurology & Ophthalmology Analyst at GlobalData, comments, “While the newly revealed mechanism of action isn’t expected to cure PD, it will prove useful as an additional tool for combating the motor symptoms associated with PD.”

Additionally, the therapy could also be potentially used in combination with another treatment that is aimed at, for example, increasing the production of dopamine or providing a neurotrophic support for neurons that produce dopamine.

Varkey concludes, “A major theme adopted by drugs for PD has been the optimization of the motor symptoms. If late-stage clinical trials for AAV2-GAD can help establish its safety and efficacy, it will be transformative in the sense that it will be the first gene therapy for treating motor symptoms in patients with PD.”

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