US scientists reveal new gene therapy for Parkinson’s disease

Scientists from Feinstein Institute for Medical Research in the US have revealed that a gene therapy called AAV2-GAD for Parkinson’s disease will create new brain circuits for motor function.

The findings published by Feinstein Institute for Medical Research professor Dr David Eidelberg and his team explain the therapeutic mechanisms that are involved in the emerging therapy.

Scientific and clinical communities can use these findings to advance the AAV2-GAD therapy into clinical trials while providing a better understanding of the disease treatment response.

Eidelberg said: "Current Parkinson’s disease therapies act on the abnormal disease network in the brain and often stop working over time as the body builds a tolerance. What we observed with AAV2-GAD therapy is quite the opposite.

"We found that AAV2-GAD leads to the formation of new neural pathways in the brain, connecting the subthalamic nucleus to other motor regions, thereby improving motor symptoms for as long as 12 months."

"The appearance of the circuits is planned to be used as a treatment biomarker in an upcoming phase III clinical trial for the new disease intervention."

As part of the study, metabolic PET scans from 15 patients with Parkinson’s disease were analysed by the team.

They received the gene therapy and 20 of them were randomised for placebo surgery and then rescanned six and 12 months after surgery.

It was found that patients who received the gene therapy started to form new brain connections, which matured by the end of the study for 12 months.

The appearance of the circuits is planned to be used as a treatment biomarker in an upcoming phase III clinical trial for the new disease intervention.

Feinstein Institute for Medical Research president and CEO Kevin Tracey said: "Dr Eidelberg’s research in Parkinson’s disease has opened important new avenues for mapping brain networks that are fundamental to understanding debilitating movement disorders.

"This latest work mapping the therapeutic benefit of AAV2-GAD gene therapy is a major next step to further refining therapies that combat the root causes of the condition."

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