By Rob Waters

(Bloomberg) -- Parkinson's Disease symptoms were reduced in 12 patients who were among the first to get gene therapy for the incurable disorder that robs some 1.5 million Americans of the ability to control body movements.

The research, sponsored by Neurologix Inc. of Fort Lee, New Jersey, involves one of two such therapies tested in Parkinson's. In the treatment, the genes were inserted into patient's brains using inactive viruses as carriers. Six to 12 months afterwards, researchers using imaging scans found that the therapy calmed overexcited brain cells tied to Parkinson's.

The report, published today in the Proceedings of the National Academy of Sciences, proves the principle that genes carried by viruses can provide safe therapy, said Jon Stoessl, director of the Pacific Parkinson's Research Centre at the University of British Columbia in Vancouver.

Using imaging scanning ``provides an independent line of evidence that the circuitry involved in Parkinson's has improved following the surgery,'' said Stoessl, who wasn't involved in the study. He responded to questions in an e-mail.

People with Parkinson's gradually lose neurons that make the brain chemical dopamine. The lack of dopamine leads to increasing activity in a part of the brain called the subthalamic nucleus that influences movement. The gene injected into patients' brains ramps up production of another chemical, GABA, which reins in the excess activity and reduces abnormal movements.

Expanded Trials

Neurologix, which has no marketed products, has applied to the FDA to conduct expanded clinical trials testing the gene therapy or a sham control in about 40 patients. The doctors who developed the treatment and performed the surgery, Michael Kaplitt and Matthew During, are company founders.

No gene therapy has yet been approved by the U.S. Food and Drug Administration, though potential treatments have been tested for years for treating diseases including cystic fibrosis, rheumatoid arthritis and hemophilia. The only success to date has been in treating the condition known as bubble boy disease, a severe impairment of the immune system that renders people susceptible to infections.

Some treatments are already available to treat early stage Parkinson's symptoms, including Boehringer Ingelheim GmbH's Mirapex drug, GlaxoSmithKline Plc's Requip and a generic medication called levodopa.

Brain Stimulation

Once these treatments stop working, patients receive electrical current shot into their brains using a technique called deep brain stimulation, or DBS. While brain stimulation helps some, it also requires them to have equipment installed in their bodies, an uncomfortable choice for some patients, according to senior author David Eidelberg.

``A lot of people don't want hardware and wires inserted into their brains,'' said Eidelberg, director of the Center for Neurosciences at the Feinstein Institute for Medical Research, in Manhasset, New York.

While the initial study suggested the treatment was safe and patients reported improvements, ``there was no objective proof that this was anything other than a placebo effect,'' Eidelberg said. The scans provided physical evidence of changes in brain networks known to be involved in Parkinson's.

Nathan Klein, who has suffered from Parkinson's for almost 20 years, says his symptoms have improved since he was one of the first patients treated in the trial four years ago.

`Like a Statue'

Prior to the gene therapy, ``I did a lot of shaking and my body was very stiff,'' Klein said in a Nov. 16 phone interview. ``My facial expression was just like a statue, nothing there.'' When he was called to say a blessing at his son's bar mitzvah, the wine in the glass he was holding ``went all over the place. It was very embarrassing,'' he recalled.

Klein, 59, started noticing improvements about six months after the surgery, and today, he says, ``I feel better and I don't shake anymore.'' Without the therapy, ``I'd probably be wheelchair-bound and miserable,'' he said.

Eidelberg and Stoessl say that while Parkinson's research is progressing rapidly, no cure is in sight.

``There's a lot of momentum now from different strategies using really cool and modern technology,'' Eidelberg said. ``Patients with Parkinson's can only be optimistic. I think we're on the right path.''

Source: Bloomberg.com

Date:- November 19 2007