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Neurologix Gene Therapy Helps Parkinsonís Patients Walk, Move in Study

Note from PDF: The article below details the results of the study showing the first successful double-blind trial of gene therapy for Parkinson's disease.  According to Robin Elliott, Executive Director of the Parkinson's Disease Foundation,

"This is an important study because it is the first controlled clinical trial to show a statistically significant improvement in people with Parkinson's treated with gene therapy.  Although the results appear promising and merit further investigation, this study involved a small number of people with Parkinson's and the findings will need to be confirmed in a larger trial.  It will also be important to continue to follow these individuals to look for long-term benefits and potential risks.

PDF is committed to keeping its members and readership up to date on developing results in this and all other important clinical trials in PD.  Please visit for new information.


By Rob Waters - Mar 16, 2011

A gene injected deep into the brains of people with Parkinson’s disease eased their jerking motions and tendency to freeze, according to a study sponsored by Fort Lee, New Jersey-based Neurologix Inc. (NRGX)

The inserted gene acts like a factory, pumping out one of the brain chemicals lacking in people with the disorder to rebalance substances that stimulate and inhibit activity, said Michael Kaplitt, a founder of Neurologix and co-creator of the therapy. The study was published today in the journal Lancet Neurology.

The therapy appeared safe and seemed to improve motor control, compared with subjects who received a placebo through a sham surgical procedure, said Jon Stoessl, director of the Pacific Parkinson’s Research Centre at the University of British Columbia in Vancouver. Stoessl, who wasn’t involved in today’s findings, said the finding should be evaluated in larger trials.

“This is the first controlled trial to show a significant improvement in the active group of a gene therapy trial,” Stoessl said in a telephone interview. “It appears promising and merits further investigation.”

Neurologix, which has no marketed products, will apply to the FDA to begin an expanded trial next year that may be used to win regulatory approval, Marc Panoff, the company’s chief financial officer, said yesterday in a telephone interview.

Seeking Investors

The company needs to raise $30 million to $40 million to conduct the study and is talking to both investors and potential partners from the pharmaceutical industry to obtain funding, Panoff said.

Walter Liskiewicz, 60, a retired oral surgeon who has been living with Parkinson’s for 18 years, said he was in “terrible” shape before taking part in the study.

“I wiggled all the time, I had trouble walking, life sucked,” Liskiewicz said.

After being treated in July, 2009, Liskiewicz started noticing slow, steady improvement, he said in a telephone interview yesterday. Now he walks a mile or two once or twice a week and can carry groceries into his house.

“This is not a neuroprotective treatment, which would be the holy grail,” said Blair Ford, a professor of clinical neurology at Columbia University in New York. “This is aimed at ameliorating the immediate symptoms of Parkinson’s, not reversing its course.”

Gene Therapy Revival

About 1.5 million people in the U.S. have Parkinson’s, according to the Parkinson’s Disease Foundation. People with the neurological disease 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, which influences movement.

While no gene therapy has yet been approved by the U.S. Food and Drug Administration, recent successes have revived a field that was almost ended when a patient in such a trial died in 1999. Cambridge, Massachusetts-based Bluebird Bio, a closely held company, is developing therapies for a vision disorder and blood conditions including beta thalassemia and sickle cell disease after clinical trials reported positive results. Bluebird is backed by Genzyme Corp. (GENZ), also in Cambridge, and by venture capital firms.

Early-stage Parkinson’s patients now are treated with medications including Boehringer Ingelheim GmbH’s Mirapex, GlaxoSmithKline Plc’s Requip and a generic medication called levodopa. All aim to increase dopamine in the brain.

Effectiveness Wanes

These treatments generally lose effectiveness after several years. Patients may then get a treatment called deep-brain stimulation, which shoots electrical current into the subthalamic area of the brain. While the procedure helps many patients, they must have batteries and wires installed in their bodies, said Neurologix’s Kaplitt, a neurosurgeon at Weill Cornell Medical College in New York.

In the Neurologix therapy, an ultra-thin wire is inserted through the skull and passes through brain tissue to reach the subthalamic area. The gene, carried by a harmless virus, journeys into brain cells to ramp up production of the chemical GABA, which limits excess activity to reduce abnormal movements.

The therapy was first tested in 12 patients in a 2007 study. While those findings were encouraging, no control group was used so researchers couldn’t be certain about the findings.

In the latest study, 22 patients received an injection with the gene, called GAD. For comparison, 23 people underwent a sham procedure in which surgeons pretended to drill holes in their skulls and imitated sounds of the actual procedure.

Catheter Inserted

A catheter designed by Minneapolis-based Medtronic Inc. (MDT) to lock in place after being inserted allowed patients to leave the operating room and have the gene injected in a recovery room, making the procedure less invasive and more affordable. Afterward, the catheter was removed and a small cap was used to cover the hole in the skull.

Both groups of patients showed improvement on a scale that rates stiffness, tremors, speed of motion and other symptoms. After six months, patients who got the treatment improved by 8.1 points, or 23 percent, while the placebo patients had a 4.7 point, or 11 percent, improvement, Kaplitt said yesterday in a telephone interview. Some patients did even better, he said.

“All but two of the patients in the treatment group had some improvement and half of them had dramatic improvement,” Kaplitt said. There were no serious adverse events related to the treatment, he said.

The therapy didn’t appear to alleviate the cognitive problems, depression and impaired balance many patients suffer, Stoessl said.

Gene therapy has several advantages over deep-brain stimulation, said Kaplitt.

“It’s a single operation instead of two, patients don’t need general anesthesia and no hardware is left in the brain,” he said.

To contact the reporter on this story: Rob Waters in San Francisco at

To contact the editor responsible for this story: Reg Gale at

Source Date: Mar 17 2011
Source Publication: Bloomberg
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