Andrew Feigin, MD, and David Eidelberg, MD, of The Feinstein Institute for Medical Research collaborated with Michael Kaplitt, MD, of Weill Cornell Medical Center in Manhattan and others to deliver genes for glutamic acid decarboxylase (or GAD) into the subthalamic nucleus of the brain in Parkinson’s patients. The study was designed as a phase I safety study, and the genes were delivered to only one side of the brain to reduce risk and to better assess the treatment.
A recently published study included the clinical results of the novel gene therapy trial, but this new report from the same study focuses on the power of modern brain scans to show that the gene therapy altered brain activity in a favorable way. This latest study is published this week in the Proceedings of the National Academy of Sciences.
The patients only 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. It was a so-called open-label study -- everybody received the gene therapy so the scientists knew that there could be a placebo effect. That is why brain scans were so critical to the experiment. Dr. Eidelberg and his colleagues pioneered the technology and used it to identify brain networks in Parkinson’s disease and a number of other neurological disorders.
In Parkinson’s, they identified two discrete brain networks -- one that regulates movement and another that affects cognition. The results from the brain scan study on the gene therapy patients show that only the motor networks were altered by the therapy. “This is good news,” said Dr. Eidelberg, the senior investigator of the study. “You want to be sure that the treatment doesn’t make things worse.” The gene makes an inhibitory chemical called GABA that turns down the activity in a key node of the Parkinson’s motor network. The investigators were not expecting to see changes in cognition, and the scans confirmed that this did not occur.
Position emission tomography (PET) scans were performed before the surgery and repeated six months later and then again one year after the surgery. The motor network on the untreated side of the body got worse, and the treated side got better. The level of improvements in the motor network correlated with increased clinical ratings of patient disability, added Dr. Feigin.
“Having this information from a PET scan allows us to know that what we are seeing is real,” Dr. Eidelberg added. 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,” he said.
The gene therapy technique was developed by Neurologix Inc., a New Jersey-based company. Scientists are now working on a design for a phase 2 blinded study that would include a larger number of patients to test the effectiveness of the treatment.
Jamie Talan | EurekAlert!
Novel PET tracer identifies most bacterial infections
06.10.2017 | Society of Nuclear Medicine and Molecular Imaging
Teleoperating robots with virtual reality
05.10.2017 | Massachusetts Institute of Technology, CSAIL
University of Maryland researchers contribute to historic detection of gravitational waves and light created by event
On August 17, 2017, at 12:41:04 UTC, scientists made the first direct observation of a merger between two neutron stars--the dense, collapsed cores that remain...
Seven new papers describe the first-ever detection of light from a gravitational wave source. The event, caused by two neutron stars colliding and merging together, was dubbed GW170817 because it sent ripples through space-time that reached Earth on 2017 August 17. Around the world, hundreds of excited astronomers mobilized quickly and were able to observe the event using numerous telescopes, providing a wealth of new data.
Previous detections of gravitational waves have all involved the merger of two black holes, a feat that won the 2017 Nobel Prize in Physics earlier this month....
Material defects in end products can quickly result in failures in many areas of industry, and have a massive impact on the safe use of their products. This is why, in the field of quality assurance, intelligent, nondestructive sensor systems play a key role. They allow testing components and parts in a rapid and cost-efficient manner without destroying the actual product or changing its surface. Experts from the Fraunhofer IZFP in Saarbrücken will be presenting two exhibits at the Blechexpo in Stuttgart from 7–10 November 2017 that allow fast, reliable, and automated characterization of materials and detection of defects (Hall 5, Booth 5306).
When quality testing uses time-consuming destructive test methods, it can result in enormous costs due to damaging or destroying the products. And given that...
Using a new cooling technique MPQ scientists succeed at observing collisions in a dense beam of cold and slow dipolar molecules.
How do chemical reactions proceed at extremely low temperatures? The answer requires the investigation of molecular samples that are cold, dense, and slow at...
Scientists from the Max Planck Institute of Quantum Optics, using high precision laser spectroscopy of atomic hydrogen, confirm the surprisingly small value of the proton radius determined from muonic hydrogen.
It was one of the breakthroughs of the year 2010: Laser spectroscopy of muonic hydrogen resulted in a value for the proton charge radius that was significantly...
17.10.2017 | Event News
10.10.2017 | Event News
10.10.2017 | Event News
20.10.2017 | Information Technology
20.10.2017 | Materials Sciences
20.10.2017 | Interdisciplinary Research