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Gene therapy 'treats' Parkinson's disease | Gene therapy 'treats' Parkinson's disease |
(1 day later) | |
By James Gallagher Health reporter, BBC News | By James Gallagher Health reporter, BBC News |
Treating Parkinson's disease with gene therapy has been shown to be successful in clinical trials for the first time, say US researchers. | Treating Parkinson's disease with gene therapy has been shown to be successful in clinical trials for the first time, say US researchers. |
The illness causes uncontrolled shaking, stiffness and slow movement as part of the brain dies. | The illness causes uncontrolled shaking, stiffness and slow movement as part of the brain dies. |
The small study in The Lancet Neurology used a virus to add genes to brain cells, which resulted in reduced symptoms for half of patients. | The small study in The Lancet Neurology used a virus to add genes to brain cells, which resulted in reduced symptoms for half of patients. |
Parkinson's UK welcomed the study, but said further research was needed. | Parkinson's UK welcomed the study, but said further research was needed. |
The disease affects 120,000 people in the UK, mostly in the over-50s. | The disease affects 120,000 people in the UK, mostly in the over-50s. |
There is no cure, although drugs and deep brain stimulation have been shown to reduce symptoms. | There is no cure, although drugs and deep brain stimulation have been shown to reduce symptoms. |
Gene treatment | Gene treatment |
Patients with Parkinson's have reduced levels of a chemical - GABA - in part of the brain known as the subthalamic nucleus. | Patients with Parkinson's have reduced levels of a chemical - GABA - in part of the brain known as the subthalamic nucleus. |
The researchers created a virus which "infects" cells with a gene to increase GABA production. | |
In the trial, 22 patients had the virus injected into their brains while 23 patients had "sham surgery", to make them think they had the virus injected. | In the trial, 22 patients had the virus injected into their brains while 23 patients had "sham surgery", to make them think they had the virus injected. |
Their motor function was then scored over six months. | Their motor function was then scored over six months. |
Patients who had gene therapy showed a 23.1% improvement in their motor score, those with sham surgery improved by 12.7%. | Patients who had gene therapy showed a 23.1% improvement in their motor score, those with sham surgery improved by 12.7%. |
The report's authors say this "offers a novel alternative to conventional pharmacological or surgical treatment" and that it "shows the promise of gene therapy for other neurological disorders." | The report's authors say this "offers a novel alternative to conventional pharmacological or surgical treatment" and that it "shows the promise of gene therapy for other neurological disorders." |
Professor Nicholas Mazarakis, who is a specialist in gene therapy at Imperial College London, told the BBC that the positive result was "very encouraging." | Professor Nicholas Mazarakis, who is a specialist in gene therapy at Imperial College London, told the BBC that the positive result was "very encouraging." |
He added: "This result should be taken with some caution though, as it constitutes a rather small mean improvement, only 10.4%, in the clinical rating scale motor scores between those patients receiving the gene therapy and the placebo group." | He added: "This result should be taken with some caution though, as it constitutes a rather small mean improvement, only 10.4%, in the clinical rating scale motor scores between those patients receiving the gene therapy and the placebo group." |
In addition the absence of significant improvements in other secondary outcome measures such as dyskinesia and quality of life between the two groups, warrants further long-term evaluation of this treatment in more patients." | In addition the absence of significant improvements in other secondary outcome measures such as dyskinesia and quality of life between the two groups, warrants further long-term evaluation of this treatment in more patients." |
There have been concerns about the safety of gene therapy. In 1999, Jesse Gelsinger died during a trial in the US and there were cases of leukaemia after treatment in France. | There have been concerns about the safety of gene therapy. In 1999, Jesse Gelsinger died during a trial in the US and there were cases of leukaemia after treatment in France. |
The authors say this procedure is safe. | The authors say this procedure is safe. |
Dr Michelle Gardner, research development manager at Parkinson's UK, said: "This research shows the promise of gene therapy for neurological conditions like Parkinson's, but further research is still needed. | Dr Michelle Gardner, research development manager at Parkinson's UK, said: "This research shows the promise of gene therapy for neurological conditions like Parkinson's, but further research is still needed. |
"We still don't know for how long the benefits of this treatment may last, or whether there may be long-term problems due to introducing viruses into the brain. | "We still don't know for how long the benefits of this treatment may last, or whether there may be long-term problems due to introducing viruses into the brain. |
"In addition, any new treatment must be shown to be more effective than those currently available for Parkinson's, which this treatment has not yet been shown to do." | "In addition, any new treatment must be shown to be more effective than those currently available for Parkinson's, which this treatment has not yet been shown to do." |
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