Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

Gene Therapy for Parkinson's Disease

22.06.2007
Injection of genetic information directly into the brain cells of patients (gene therapy) with neurodegenerative conditions such as Parkinson’s disease could safely alleviate symptoms of these conditions, conclude authors of a study published in this week’s edition of The Lancet.

However an accompanying comment questions the advantages of gene therapy over deep-brain stimulation, a current method for treating Parkinson’s disease.

Professor Matthew During, Weill Medical College of Cornell University, New York, USA, and colleagues did a safety and tolerability trial on 11 men and one woman, average age 58 years. The patients were divided into groups of four and given low, medium, or high dose, injections of the glutamic acid decarboxylase (GAD) gene, with associated adeno-associated virus (AAV). The injections were given into the subthalamic nucleus of the brain, and all took place at the Weill Medical College of Cornell University/New York Presbyterian Hospital, USA.

All patients survived the surgery and had no side effects related to gene therapy. Substantial improvements in movement were seen within three months of surgery in the side of the body opposite to that of the brain where injections were given, and continued until 12 months after surgery – the endpoint of the trial. Improvements in motor scores were also seen in both the “on” state (when subjects were on standard medication, and typically not improved with other surgical approaches), as well as the “off” state despite the gene therapy being given in only one hemisphere.

... more about:
»Parkinson »advantage »surgery »therapy

Brain scans also showed reductions in metabolism in the ipsilateral thalamus (IT), similar to reductions which occur after other successful surgery for Parkinson’s disease. The study also showed a correlation between improved clinical motor scores and brain metabolism in the supplementary motor area, again similar to findings reported after surgery of patients with Parkinson’s disease.

The authors also point out that the gene therapy approach has advantages over the currently used deep-brain stimulation method. They say: “The absence of indwelling hardware [for gene therapy] reduces the risk of infection, and some patients with Parkinson’s disease simply prefer not to have the implanted device.” They add that frequent hospital visits would not be necessary for the gene therapy approach.

The authors conclude: “Our results show that AAV-mediated gene transfer can be done safely in the human brain, with no evidence of substantial toxic effects or adverse events in the perioperative period and for at least one year after treatment.”

In the accompanying Comment, Dr Jon Stoessl, Pacific Parkinson’s Research Centre, University of British Columbia, Canada, asks: “Apart from the avoidance of stimulator adjustments and potential hardware problems, what is the real advantage of this approach compared with deep-brain stimulation of the subthalmic nucleus?”

He concludes: “During and colleagues have taken a proactive approach to the treatment of neurodegenerative disease, and should be congratulated for their circumspection in the description of the potential implications of their findings. They made an important step in showing proof of principle, but much work should be done before neurologists and neuroscientists will regard this therapy as an effective approach.”

Tony Kirby | alfa
Further information:
http://www.thelancet.com/webfiles/images/clusters/thelancet/press_office/Parkinson's.pdf

Further reports about: Parkinson advantage surgery therapy

More articles from Life Sciences:

nachricht Embryonic development: How do limbs develop from cells?
18.05.2018 | Humboldt-Universität zu Berlin

nachricht Reading histone modifications, an oncoprotein is modified in return
18.05.2018 | American Society for Biochemistry and Molecular Biology

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Explanation for puzzling quantum oscillations has been found

So-called quantum many-body scars allow quantum systems to stay out of equilibrium much longer, explaining experiment | Study published in Nature Physics

Recently, researchers from Harvard and MIT succeeded in trapping a record 53 atoms and individually controlling their quantum state, realizing what is called a...

Im Focus: Dozens of binaries from Milky Way's globular clusters could be detectable by LISA

Next-generation gravitational wave detector in space will complement LIGO on Earth

The historic first detection of gravitational waves from colliding black holes far outside our galaxy opened a new window to understanding the universe. A...

Im Focus: Entangled atoms shine in unison

A team led by Austrian experimental physicist Rainer Blatt has succeeded in characterizing the quantum entanglement of two spatially separated atoms by observing their light emission. This fundamental demonstration could lead to the development of highly sensitive optical gradiometers for the precise measurement of the gravitational field or the earth's magnetic field.

The age of quantum technology has long been heralded. Decades of research into the quantum world have led to the development of methods that make it possible...

Im Focus: Computer-Designed Customized Regenerative Heart Valves

Cardiovascular tissue engineering aims to treat heart disease with prostheses that grow and regenerate. Now, researchers from the University of Zurich, the Technical University Eindhoven and the Charité Berlin have successfully implanted regenerative heart valves, designed with the aid of computer simulations, into sheep for the first time.

Producing living tissue or organs based on human cells is one of the main research fields in regenerative medicine. Tissue engineering, which involves growing...

Im Focus: Light-induced superconductivity under high pressure

A team of scientists of the Max Planck Institute for the Structure and Dynamics of Matter (MPSD) at the Center for Free-Electron Laser Science in Hamburg investigated optically-induced superconductivity in the alkali-doped fulleride K3C60under high external pressures. This study allowed, on one hand, to uniquely assess the nature of the transient state as a superconducting phase. In addition, it unveiled the possibility to induce superconductivity in K3C60 at temperatures far above the -170 degrees Celsius hypothesized previously, and rather all the way to room temperature. The paper by Cantaluppi et al has been published in Nature Physics.

Unlike ordinary metals, superconductors have the unique capability of transporting electrical currents without any loss. Nowadays, their technological...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

VideoLinks
Industry & Economy
Event News

Save the date: Forum European Neuroscience – 07-11 July 2018 in Berlin, Germany

02.05.2018 | Event News

Invitation to the upcoming "Current Topics in Bioinformatics: Big Data in Genomics and Medicine"

13.04.2018 | Event News

Unique scope of UV LED technologies and applications presented in Berlin: ICULTA-2018

12.04.2018 | Event News

 
Latest News

Supersonic waves may help electronics beat the heat

18.05.2018 | Power and Electrical Engineering

Keeping a Close Eye on Ice Loss

18.05.2018 | Information Technology

CrowdWater: An App for Flood Research

18.05.2018 | Information Technology

VideoLinks
Science & Research
Overview of more VideoLinks >>>