Gene therapy raises hopes of Parkinson`s sufferers

Last Updated: Friday, March 18, 2011 - 00:00

London: Raising hopes of millions of people suffering from Parkinson`s disease, a new study has found that gene therapy can significantly improve movement in patients with the degenerative brain disorder.

In the first proper clinical trial of the innovative treatment, in which genes are injected directly into the brain, was found to be safe and led to improvements in the condition for people who did not respond to drugs.

The findings, reported in The Lancet Neurology, have raised hope that the therapy can eventually be developed to treat sufferers of Parkinson`s disease and other brain conditions such as Alzheimer`s.

Parkinson`s develops when levels of important chemicals in the brain drop, and when protein deposits in the brain develop. It affects the ability to walk and eat while existing drugs can cause side-effects such as involuntary movements, the Telegraph reported.

One current treatment, called Deep Brain Stimulation, involves patients being fitted with an electronic device that blocks the nerve signals that cause tremors.

But four years ago, scientists disclosed a "milestone" in treating the disease as well as other degenerative conditions by injecting genes.

A small group of patients was injected with billions of copies of a genetically altered virus, into a part of the brain called the subthalamic nucleus.

The virus contained the human gene for an enzyme called glutamic acid decarboxylase (GAD) that helps to make a chemical lacking in Parkinson`s sufferers, called GABA.

In the initial trial, the patients` brain cells started to make the GABA chemical and were later found to have greatly improved mobility.

In the first randomised double-blind study of the gene therapy, 22 patients had the virus injected into their brains along with 23 who received a placebo.

Using an established scale to assess their mobility up to six months later, researchers at the Feinstein Institute for Medical Research, New York, found that those who had the gene therapy saw a 23.1 per cent improvement in their motor function compared with 12.7 in the placebo group.

The only "mild" side-effects noted were headache and nausea, the researchers said.

"This is the first double-blind randomised, controlled gene therapy trial in Parkinson?s patients and the fact that there are some positive results is most welcome encouragement for Parkinson`s patients," said Dr Thomas Foltynie at the Institute of Neurology, London.

However experts pointed out that the trial only involved a small number of patients, and may not represent an improvement on existing treatments.

Dr Michelle Gardner, from Parkinson`s UK charity, said: "This research shows the promise of gene therapy for neurological conditions like Parkinson`s. But further research is still needed."

PTI



First Published: Friday, March 18, 2011 - 00:00

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