New hope was offered to sufferers of Parkinson's disease today by a study that found that a type of gene therapy can significantly improve motor function in patients.
The trial showed that injection of the glutamic acid decarboxylase (GAD) gene directly into the brain was safe and could lead to improvements in the condition of those who are not responsive to drug treatment.
It is thought that the findings, published in medical journal The Lancet Neurology, show the potential of gene therapy to alleviate the symptoms of other brain disorders as well.
The GAD gene produces a brain chemical called GABA, which is responsible for coordinating movement and is severely reduced in Parkinson's patients. In the treatment being tested, the gene is inserted into a part of the brain called the subthalamic nucleus with the aim of increasing GABA production and thus restoring motor function.
Significant improvements in motor function were observed in patients who underwent the gene therapy and who had been off their medication for 12 hours.
Six months after surgery, patients who had received gene therapy had an average 23.1pc improvement in motor function compared with 12.7pc in a group that had received placebos.
The scientists behind the trial, Andrew Feigin and colleagues from the Feinstein Institute for Medical Research in New York, said: "This study ... justifies the continued development of AAV2-GAD for treatment of Parkinson's disease ... and shows the promise of gene therapy for other neurological disorders."