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| Gene therapy treats Parkinson's | Gene therapy treats Parkinson's |
| (2 days later) | |
| An experimental form of gene therapy for Parkinson's disease has been shown to produce promising results. | An experimental form of gene therapy for Parkinson's disease has been shown to produce promising results. |
| US scientists treated 12 patients with a virus genetically modified to carry a human gene which dampens down the nerve cells over-excited by Parkinson's. | US scientists treated 12 patients with a virus genetically modified to carry a human gene which dampens down the nerve cells over-excited by Parkinson's. |
| Now brain scans have revealed significant improvements - which were still present a year later. | Now brain scans have revealed significant improvements - which were still present a year later. |
| The Feinstein Institute for Medical Research study features in Proceedings of the National Academy of Sciences. | |
| This study important as it suggests that it was the therapy itself, rather than a placebo effect, that was having a positive impact on patients' symptoms Dr Kieran BreenParkinson's Disease Society | This study important as it suggests that it was the therapy itself, rather than a placebo effect, that was having a positive impact on patients' symptoms Dr Kieran BreenParkinson's Disease Society |
| However, the work is still at an early stage. The main aim was to test whether the therapy was safe. | However, the work is still at an early stage. The main aim was to test whether the therapy was safe. |
| Scientists delivered the gene only to one side of the brain - that which controls movement on the side of the body most affected by Parkinson's - to reduce the potential risk. | Scientists delivered the gene only to one side of the brain - that which controls movement on the side of the body most affected by Parkinson's - to reduce the potential risk. |
| It makes an inhibitory chemical called GABA that turns down the activity in a key part of the pathway which controls movement. | It makes an inhibitory chemical called GABA that turns down the activity in a key part of the pathway which controls movement. |
| Motor network changes | Motor network changes |
| The US team tested the impact of the therapy by using a form of brain imaging known as positron emission tomography (PET) to track changes in the brain. | The US team tested the impact of the therapy by using a form of brain imaging known as positron emission tomography (PET) to track changes in the brain. |
| They focused on two discrete brain networks - one that regulates movement, and another that affects thinking processes. | They focused on two discrete brain networks - one that regulates movement, and another that affects thinking processes. |
| Only the motor networks were altered by the therapy - but this was all the researchers had hoped for. | Only the motor networks were altered by the therapy - but this was all the researchers had hoped for. |
| The scans showed that the motor network on the untreated side of the body got worse, and that on the treated side got better. | The scans showed that the motor network on the untreated side of the body got worse, and that on the treated side got better. |
| The improvement was reflected in an improvement in patients' symptoms. | The improvement was reflected in an improvement in patients' symptoms. |
| They began to show signs of improvement one month after starting therapy, and by six months movement had improved by an average of 30%. | They began to show signs of improvement one month after starting therapy, and by six months movement had improved by an average of 30%. |
| One patient registered an improvement of 65%. | One patient registered an improvement of 65%. |
| The brain scans also showed those patients who received the highest dose of the gene therapy registered the longest-lasting effect. | The brain scans also showed those patients who received the highest dose of the gene therapy registered the longest-lasting effect. |
| Lead researcher Dr David Eidelberg said: "Having this information from a PET scan allows us to know that what we are seeing is real. | Lead researcher Dr David Eidelberg said: "Having this information from a PET scan allows us to know that what we are seeing is real. |
| "This study demonstrates that PET scanning can be a valuable marker in testing novel therapies for Parkinson's disease." | "This study demonstrates that PET scanning can be a valuable marker in testing novel therapies for Parkinson's disease." |
| Parkinson's, which affects around 120,000 people in the UK, is caused by the loss of brain cells, which leads to a drop in chemicals required to regulate cell activity, and the connections they make with their neighbours. | Parkinson's, which affects around 120,000 people in the UK, is caused by the loss of brain cells, which leads to a drop in chemicals required to regulate cell activity, and the connections they make with their neighbours. |
| Dr Kieran Breen, of the Parkinson's Disease Society, said the disease was likely to be caused by a mix of genetic and environmental factors. | Dr Kieran Breen, of the Parkinson's Disease Society, said the disease was likely to be caused by a mix of genetic and environmental factors. |
| "Because of this, there are many potential ways to treat or cure Parkinson's, and gene therapy is one potential route holding a lot of promise," he said. | "Because of this, there are many potential ways to treat or cure Parkinson's, and gene therapy is one potential route holding a lot of promise," he said. |
| "This study is important as it suggests that it was the therapy itself, rather than a placebo effect, that was having a positive impact on patients' symptoms." | "This study is important as it suggests that it was the therapy itself, rather than a placebo effect, that was having a positive impact on patients' symptoms." |