A gene therapy treatment for Parkinson’s disease really does make a difference, according to the findings of a major trial published last night.
The treatment involves injecting the GAD gene into the brain and early trials had suggested it might work.
But the latest study provides proof – suggesting the gene contributes an improvement of about ten per cent in patients’ ability to control their movements.
Just 45 patients were involved in the latest research, published in The Lancet Neurology.
Half had the gene injection while the other half had a similar brain procedure – but without the gene.
Researchers found that without the gene patients experienced an improvement of about 12 per cent – suggesting a powerful "placebo effect."
But the gene treatment, known as NLX-P101, added further improvements – which lasted for at least six months.
The GAD gene – glutamic acid decarboxylase – is meant to produce a chemical called GABA, which as responsible for co-ordinating movement.
Researcher Dr Michael Kaplitt, of New York-Presbyterian Hospital/Weill Cornell Medical Center, USA, said: "Patients who received NLX-P101 showed a significant reduction in the motor symptoms of Parkinson’s, including tremor, rigidity and difficulty initiating movement."
Fellow researcher Professor Matthew During described the findings as "great news" for people with Parkinson’s disease.
He added: "Since this is also the first gene therapy study for a neurological disease to achieve success in a rigorous randomised, double-blind design compared with a sham group, this is also a crucial step forward toward finally bringing gene therapy into clinical practice for patients with debilitating brain disorders."
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