The Feinstein Institute for Medical Research and Weill Cornell Medical Center in Manhattan have conducted a gene therapy experiment that has shown to normalize brain function in patients with Parkinson's disease. This study involved the use of brain scan and the effects are present even after a year.
During the study, the researchers delivered genes for glutamic acid decarboxylase (GAD) into the subthalamic nucleus of the brain in patients with Parkinson's.
The first phase of the study was carried out as a safety study, and the genes were delivered to only one side of the brain to reduce risk and to better assess the treatment.
The subjects received the viral vector-carrying genes to the side of the brain that controls movement on the side of their body most affected by the disease. Upon performing Position emission tomography (PET) scans on the patients, the researchers found that the motor network on the untreated side of the body got worse, while that on the treated side got better.
"This is good news. You want to be sure that the treatment doesn't make things worse," Dr. David Eidelberg of The Feinstein Institute for Medical Research. "Having this information from a PET scan allows us to know that what we are seeing is real," Dr. Eidelberg said.
The scans also detected differences in responses between dose groups, with the highest gene therapy dose demonstrating a longer-lasting effect. "This study demonstrates that PET scanning can be a valuable marker in testing novel therapies for Parkinson's disease," Dr. Eidelberg said.
The scans further confirmed that the therapy did not cause any see changes in cognition.
The study has been published in the Proceedings of the National Academy of Sciences.