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Gene Therapy for Parkinson's Disease
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. 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.
The study 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 gene transfer can be done safely in the human brain, with no evidence of substantial toxic effects or adverse events in the preoperative period and for at least one year after treatment.”
REHACARE.de; Source: The Lancet
- The Lancet: www.lancet.com