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| Therapy for 'Lorenzo's oil' boys | Therapy for 'Lorenzo's oil' boys |
| (about 22 hours later) | |
| Doctors have for the first time used gene therapy to treat two boys with the rare nervous disorder made famous through the film "Lorenzo's Oil". | Doctors have for the first time used gene therapy to treat two boys with the rare nervous disorder made famous through the film "Lorenzo's Oil". |
| Treatment for adrenoleukodystrophy has mainly involved bone marrow transplant, but this can be problematic due to lack of donors and rejection by the body. | Treatment for adrenoleukodystrophy has mainly involved bone marrow transplant, but this can be problematic due to lack of donors and rejection by the body. |
| French doctors have now managed to modify the boys' own bone marrow. | French doctors have now managed to modify the boys' own bone marrow. |
| They unveiled their findings at a summit of the European Society of Gene and Cell Therapy in Rotterdam. | They unveiled their findings at a summit of the European Society of Gene and Cell Therapy in Rotterdam. |
| Adrenoleukodystrophy (ALD) is a rare inherited disorder that leads to progressive brain damage, failure of the adrenal gland and eventually death. | Adrenoleukodystrophy (ALD) is a rare inherited disorder that leads to progressive brain damage, failure of the adrenal gland and eventually death. |
| It occurs because a flawed gene on the child's X chromosome means his body cannot produce an enzyme needed to break down fatty acids which can accumulate in the brain. | It occurs because a flawed gene on the child's X chromosome means his body cannot produce an enzyme needed to break down fatty acids which can accumulate in the brain. |
| There is no cure. "Lorenzo's Oil", a mixture of oleic acid and euric acid, is only really effective before the onset of symptoms. | There is no cure. "Lorenzo's Oil", a mixture of oleic acid and euric acid, is only really effective before the onset of symptoms. |
| A team led by Patrick Aubourg, a professor at the Saint Vincent de Paul Hospital in Paris, started to look at whether it was possible to correct the faulty gene. | A team led by Patrick Aubourg, a professor at the Saint Vincent de Paul Hospital in Paris, started to look at whether it was possible to correct the faulty gene. |
| Virus employed | Virus employed |
| They used a harmless version of HIV as a vector to carry a corrected version of the gene into the bone marrow cell. | |
| Of course it's early days yet, but parents really can start to feel hopeful, even if more patience is needed Paula Brazeal United Leukodystrophy Foundation | Of course it's early days yet, but parents really can start to feel hopeful, even if more patience is needed Paula Brazeal United Leukodystrophy Foundation |
| HIV, which can lead to Aids, is the only virus known to be capable of carrying a gene into the nucleus of non-dividing cells, such as stem cells - the process needed if the treatment is to have a long-term effect. | HIV, which can lead to Aids, is the only virus known to be capable of carrying a gene into the nucleus of non-dividing cells, such as stem cells - the process needed if the treatment is to have a long-term effect. |
| The treated bone marrow was then reinjected back into the boys. | The treated bone marrow was then reinjected back into the boys. |
| Results at both six months and a year suggested the production of the missing enzyme had noticeably improved. | Results at both six months and a year suggested the production of the missing enzyme had noticeably improved. |
| "But we need to be cautious, especially as we need to know if it will be a sufficient and long term effect, and if no complications will arise due to the use of this vector," said Professor Aubourg. | "But we need to be cautious, especially as we need to know if it will be a sufficient and long term effect, and if no complications will arise due to the use of this vector," said Professor Aubourg. |
| But Paula Brazeal, the head of the United Leukodystrophy Foundation, said this was undoubtedly "the future". | But Paula Brazeal, the head of the United Leukodystrophy Foundation, said this was undoubtedly "the future". |
| "We've been waiting for this breakthrough for a long time. This is the way treatment is heading. | "We've been waiting for this breakthrough for a long time. This is the way treatment is heading. |
| "Of course it's early days yet, but parents really can start to feel hopeful, even if more patience is needed." | "Of course it's early days yet, but parents really can start to feel hopeful, even if more patience is needed." |