Parkinson's gene therapy step closer

Surgical gene therapy for Parkinson's disease - one of a series of breakthroughs in Professor Matthew During's research career - has taken a big step towards becoming an established treatment.

A recently reported phase two trial found reduced symptoms in patients who received the treatment, compared with others who had sham surgery.

There were no serious adverse events from the gene therapy or the surgical procedure, says the United States biotech company Neurologix, which is working towards commercialising the treatment.

Performed under local anaesthetic, the treatment involves boring a hole through the skull to deliver the genes via a safe virus in a liquid.

Professor During, who divides his time between Auckland University and Ohio State University in the US, led development of the therapy.

He said that although the trial had not yet been reported in a peer-reviewed journal, it was a milestone.

"That's the first proof of any gene therapy in the brain. We are going to go to a pivotal phase three [trial] to get licensure in the US so it can be developed as a viable commercial treatment."

Around 8000 New Zealanders have Parkinson's, which is characterised by trembling, rigid posture, slow movements and a shuffling gait. It is caused by loss of the nerve cells making dopamine, a chemical messenger.

The trial delivers a gene called GAD to calm certain brain cells whose overactivity produces the symptoms of the disease.