They advance in gene therapy for Parkinson's disease

MADRID, 29th November (EUROPA PRESS) –

Scientists have discovered that an emerging gene therapy for Parkinson's disease creates new circuits in the brain associated with better motor movement. These findings, published Wednesday in "Science Translational Medicine" by Professor Feinstein Medical Research Institute (US), David Eidelberg and his team, explain the therapeutic mechanisms involved in gene therapy of emerging Parkinson's disease, called AAV2-GAD .

These findings help scientific and clinical communities in advancing AAV2-GAD clinical trials, while enabling better understanding of the response and efficacy of Parkinson's disease treatment, the second most common neurodegenerative disease. widespread in the United States. United

Patients with Parkinson's disease often have tremors, slow movement (bradykinesia), stiffness, and problems with balance and coordination, resulting in difficulty walking, speaking, or performing simple tasks. daily. Current therapies and medications for Parkinson's disease help relieve symptoms, but do not reduce the underlying neural degeneration.

IMPROVING MOTOR SYMPTOMS

Gene therapy, which involves injecting genes into cells to correct abnormalities of brain function, is an emerging therapeutic approach for neurodegenerative disorders, such as Parkinson's disease. Recent phase 2 clinical trials have shown that delivery of the glutamic acid decarboxylase (GAD) gene into a part of the brain called the subthalamic nucleus has therapeutic effects in patients. Dr. Eidelberg's analysis of the mechanisms of AAV2-GAD therapy revealed that the mechanism of action of the therapy is unique compared to other treatments for Parkinson's disease.

"Current therapies for Parkinson's disease affect the network of abnormal brain diseases and often stop working as the body gains tolerance." What we observe with AAV2-GAD therapy is exactly the opposite. Dr. Eidelberg, lead author of the article, said "We found that AAV2-GAD led to the formation of new neuronal pathways in the brain, linking the subthalamic nucleus to the brain." other motor areas, thus improving motor symptoms up to 12 months, "he adds.

In this study, Eidelberg and his team analyzed the metabolic PET of 15 patients with Parkinson's disease who had received gene therapy and 20 others who had been randomly assigned to a dummy surgery and then re-examined six and 12 months after # 39; transaction. . What they discovered, is that people undergoing gene therapy began to establish new brain connections, which matured at the end of the 12-month study. The Eidelberg team plans to use the emergence of these new circuits as a biomarker of treatment as part of a future phase 3 clinical trial of this new intervention for Parkinson's disease .

"Dr. Eidelberg's Parkinson's research has opened new and important pathways for mapping brain networks that are critical to understanding debilitating movement disorders," notes Kevin J. Tracey, President and CEO of Parkinson's. Feinstein Institute. The work that traces the therapeutic benefit of AAV2-GAD gene therapy is a big step forward in perfecting the therapies that fight the causes of the disease. "