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| Version 1 | Version 2 |
|---|---|
| '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 the same therapy was halted in 2002 after three of ten children developed leukaemia. | A trial in France of the same therapy was halted in 2002 after three of ten 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 | |
| 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." |
| 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. | |
| 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. |
| A few years previously a French trial was halted prematurely after three of ten boys treated were diagnosed with T-Cell leukaemia. | A few years previously a French trial was halted prematurely after three of ten boys treated were diagnosed with T-Cell leukaemia. |
| Nine of them had been cured of their original condition. Three went on to be cured from leukaemia, but one died. | |
| Last year, doctors at Great Ormond Street dismissed the findings of the US study as "unhelpful". | Last year, doctors at Great Ormond Street dismissed the findings of the US study as "unhelpful". |
| Professor Thrasher said then: "This is a very preliminary study published in an incomplete form. | Professor Thrasher said then: "This is a very preliminary study published in an incomplete form. |
| "The researchers have taken artificially high doses of these genes and given them to animals. | "The researchers have taken artificially high doses of these genes and given them to animals. |
| "I'm not sure how it is relevant to human treatment. | "I'm not sure how it is relevant to human treatment. |
| "We know already that, in lower doses, the gene therapy does not have that effect." | "We know already that, in lower doses, the gene therapy does not have that effect." |
| 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. | |
| "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. | |
| "All patients are monitored carefully as part of their care plan." |