Gene therapy to treat Parkinson’s disease

Have you met somebody, who is having difficulty in taking care of himself and he cannot walk or eat properly? Well, now he can be treated.


A major worldwide breakthrough in gene therapy was signaled last night after injections into the brain were used for the first time to successfully treat a degenerative brain disease.

Parkinson's disease robs sufferers of the ability to walk and even eat, causes long motionless periods known as "freezing" as well as head and limb tremors.

Parkinson's occurs when the brain cells - neurons - that release the messenger chemical known as dopamine die. Protein deposits also form in the brain, and levels of another messenger chemical called GABA - which calms overexcited brain cells - drop.

As the disease progresses, higher doses of drugs are required, leading to side-effects that include involuntary movements.


The 12 patients involved in the study - a world-first human gene therapy trial for a brain disease - all reported a substantial reduction in their symptoms after having a human gene injected.

Within months, their ability to move had improved on average by 30 per cent. Some reported a 65 per cent improvement in their mobility


The study was carried out by a team in the United States. The lead researcher, Dr Michael Kaplitt, said: "We believe this is a milestone - not only for the treatment of Parkinson's disease, but for the use of gene-based therapies against neurological conditions generally."
Dr Kieran Breen, the director of research and development for the UK Parkinson's Disease Society, said: "There are many potential ways to treat or cure Parkinson's, and gene therapy is one potential route holding a lot of promise.
"The results of the study are encouraging in terms of safety and efficacy and we look forward to seeing the results of the larger trial planned for later this year."
The study, begun in 2003, was carried out on 11 men and one woman with an average age of 58, who had all had severe Parkinson's for at least five years and for whom current therapies were no longer effective.

They were given injections of billions of copies of a genetically altered virus into part of the brain called the subthalamic nucleus.

The altered virus carried the human gene for an enzyme, called GAD, which helps to make GABA. Once implanted, brain cells of the patients started to make the GABA chemical, said Prof During.

"This ground-breaking study represents not only an encouraging first step in the development of a promising new approach to Parkinson's disease therapy, but also provides a platform to translate a variety of new gene therapy agents into human clinical trials for many devastating brain disorders," said Paul Greengard, the chairman of the Neurologix Scientific Advisory Board.

Prof Alan Kingsman of Oxford Biomedica, who will soon test his own gene therapy treatment for Parkinson', also lauded the results. "This is very good news for the field of gene therapy for neurological disorders," he said.

Source:http://www.telegraph.co.uk/
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