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 Gene Therapy Breakthrough (12 Patients with PD) 
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Post Gene Therapy Breakthrough (12 Patients with PD)
The Telegraph, a UK-based newspaper, had an article on Saturday 6/23 about a breakthrough in gene therapy. Twelve patients with PD were involved in this first human gene therapy trial. The research findings are reported in today's issue of Lancet. (I read about this on an MSA-related online discussion group.)

Here's the full text of the article and a link to the web page: (Note that on the web page, there's an audio clip on "The highs and lows of 10 years of gene therapy.") ... ain122.xml

Gene breakthrough against brain diseases
By Roger Highfield, Science Editor
Telegraph (UK-based newspaper)
Last Updated: 2:52am BST 23/06/2007

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

In a pioneering study, researchers used the treatment to bring about significant improvements in the mobility of Parkinson's sufferers. They said it could also herald a breakthrough in the treatment of other neurological disorders, such as Alzheimer's or epilepsy.

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.

Nathan Klein, 59, the first to undergo the pioneering treatment told The Daily Telegraph last night that before the gene therapy, he had been "in a state that nobody could survive". He said: "The treatment saved my life."

Prof Matthew During, one of the lead researchers, said: "Nathan Klein has further improved, I saw him just a week ago and he looked great, further improved since I had last seen him 18 months ago."

Parkinson's affects about 120,000 people in Britain, with 10,000 new cases diagnosed every year. It robs sufferers of the ability to walk and even eat, causes long motionless periods known as "freezing" as well as head and limb tremors.

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

Sufferers include the former world boxing champion Muhammad Ali and the actor Michael J Fox.

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 research team, from the New York-Presbyterian Hospital/Weill Cornell Medical Centre and the New Jersey-based company Neurologix, report their findings in today's edition of The Lancet.

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.

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.

To show that the treatment was truly having an effect, the doctors injected the virus into the subthalamic nucleus of each of the 12 Parkinson's patients, but only on one side of their brains. One reason for this was out of concerns for the patients' safety, after deaths caused by gene therapy.

Three months after the injections, the patients had shown up to 30 per cent improvement. Several showed improvements of up to 65 per cent.

"Will these remarkable improvements persist? Only longer follow-up can tell, but prior studies in animals, including primates, suggest that the transplanted gene does stay active for years," Dr Kaplitt said.

"This breakthrough trial has implications that go far beyond Parkinson's research. It's taken us nearly two decades of hard work to get here, but the success of this trial lays the foundation for the use of gene therapy against neurological diseases generally."

The researchers are planning a larger Phase 2 study in Parkinson's disease this year and a preliminary trial with epilepsy sufferers.

"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.

However, in an accompanying comment in The Lancet, Dr Jon Stoessl, of the Pacific Parkinson's Research Centre at the University of British Columbia, Canada, questioned the advantages of gene therapy over deep-brain stimulation, a current method for treating Parkinson's disease by implanting ultra fine electrodes to stimulate the brain.

"During and colleagues should be congratulated," he said. "But much work should be done before neurologists and neuroscientists will regard this as an effective approach."

Prof During's laboratory grew copies of the human gene used in the therapy in bacteria from DNA isolated from a human sample and the gene multiplied with the bacteria.

Sat Jun 23, 2007 1:23 pm
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