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| Version 5 | Version 6 |
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| 'Bubble boy' develops leukaemia | 'Bubble boy' develops leukaemia |
| (30 minutes later) | |
| One of the boys with no immune system being treated with pioneering gene therapy at Great Ormond Street has developed leukaemia, his doctors say. | One of the boys with no immune system being treated with pioneering gene therapy at Great Ormond Street has developed leukaemia, his doctors say. |
| They said cancer was an "acknowledged risk" of this treatment for X-SCID, which is commonly known as "boy in the bubble syndrome". | They said cancer was an "acknowledged risk" of this treatment for X-SCID, which is commonly known as "boy in the bubble syndrome". |
| A trial in France of a similar therapy was halted in 2002 after four of eleven children developed leukaemia. | A trial in France of a similar therapy was halted in 2002 after four of eleven children developed leukaemia. |
| Ten children with X-SCID have so far been treated at the London hospital. | Ten children with X-SCID have so far been treated at the London hospital. |
| Gene therapy appears to offer a less intrusive treatment, for those patients without a good bone marrow donor, and if we continue to make advances, may become the treatment of choice. Great Ormond Street Q&A: Gene therapy cancer case | Gene therapy appears to offer a less intrusive treatment, for those patients without a good bone marrow donor, and if we continue to make advances, may become the treatment of choice. Great Ormond Street Q&A: Gene therapy cancer case |
| All of these children "have seen clinical benefit", Professor Adrian Thrasher and Professor Bobby Gaspar, consultant immunologists on the gene therapy programme, said in a statement. | All of these children "have seen clinical benefit", Professor Adrian Thrasher and Professor Bobby Gaspar, consultant immunologists on the gene therapy programme, said in a statement. |
| "This unfortunate event is the first such development on our programme." | "This unfortunate event is the first such development on our programme." |
| The child, who is three, was treated two years ago. He has not been named. | The child, who is three, was treated two years ago. He has not been named. |
| X-SCID is caused by mutations in the IL2RG gene, which governs the behaviour of a protein involved in the development of a number of immune system cells. | X-SCID is caused by mutations in the IL2RG gene, which governs the behaviour of a protein involved in the development of a number of immune system cells. |
| Without the protein, the cells cannot develop normally, and are unable to protect the body. | Without the protein, the cells cannot develop normally, and are unable to protect the body. |
| The gene therapy works by replacing a defective gene. | The gene therapy works by replacing a defective gene. |
| Prior to treatment, the outlook for children with X-CID who did not have a suitable bone marrow donor was bleak. | Prior to treatment, the outlook for children with X-CID who did not have a suitable bone marrow donor was bleak. |
| They had to live in sterile conditions or risk picking up a life-threatening infection. They often died very young. | They had to live in sterile conditions or risk picking up a life-threatening infection. They often died very young. |
| French findings | French findings |
| But there had been warnings that there was a risk of cancer. | But there had been warnings that there was a risk of cancer. |
| A US study last year published last year looked at the long-term effect of infecting the IL2RG gene into mice: A third of the animals developed a form of cancer, with most doing so when they were about 10 months old. | A US study last year published last year looked at the long-term effect of infecting the IL2RG gene into mice: A third of the animals developed a form of cancer, with most doing so when they were about 10 months old. |
| Doctors at Great Ormond Street dismissed the findings of the US study as "unhelpful". | Doctors at Great Ormond Street dismissed the findings of the US study as "unhelpful". |
| A few years previously a French trial was halted prematurely after four of 11 boys treated were diagnosed with T-Cell leukaemia. | A few years previously a French trial was halted prematurely after four of 11 boys treated were diagnosed with T-Cell leukaemia. |
| Ten of them had been cured of their original condition. Three of those who developed leukaemia went into remission, but one died. | Ten of them had been cured of their original condition. Three of those who developed leukaemia went into remission, but one died. |
| It is thought the implanted gene was planted next to, and switched on, an oncogene, which is gene which causes cancer. | It is thought the implanted gene was planted next to, and switched on, an oncogene, which is gene which causes cancer. |
| The UK used a different vector to that in the French trial. Both now seem to pose a leukaemia risk. | |
| Making improvements | Making improvements |
| Great Ormond Street said what they hoped would be safer formulations of the genetic medicine were being prepared for clinical trials next year at several centres. | Great Ormond Street said what they hoped would be safer formulations of the genetic medicine were being prepared for clinical trials next year at several centres. |
| "Every child matters," they said in a statement. | "Every child matters," they said in a statement. |
| "Families are counselled very carefully before taking part in these treatments. Gene therapy appears to offer a less intrusive treatment, for those patients without a good bone marrow donor, and if we continue to make advances, may become the treatment of choice. | "Families are counselled very carefully before taking part in these treatments. Gene therapy appears to offer a less intrusive treatment, for those patients without a good bone marrow donor, and if we continue to make advances, may become the treatment of choice. |
| "All patients are monitored carefully as part of their care plan." | "All patients are monitored carefully as part of their care plan." |