Child likely to be first gene therapy patient

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The Independent Online
The first gene therapy trial in Britain won approval from the Government's regulatory authority yesterday.

A child suffering from a rare inherited disorder of the immune system is likely to become the first patient in the UK to receive injections of genetic material designed in the laboratory as a substitute for a defective gene.

After a day-long meeting, the Committee on the Ethics of Gene Therapy, chaired by Sir Cecil Clothier, the former National Health Service ombudsman, said that it had no ethical objections.

A spokesman for the Department of Health said that although the medical researchers involved must provide further technical details of how they intended to conduct the gene therapy trial, there were now no substantial hurdles in their way. 'The principle has been approved,' he said.

The patient concerned suffers from an immune disorder resulting from the inheritance of a defective gene for an enzyme called adenosine deaminase, ADA, which is vital for the proper functioning of the immune defences. Without ADA, children are at risk from life- threatening infections such as pneumonia and cannot lead a normal, active life.

Doctors led by Roland Levinsky, director of the Institute of Child Health at the Hospotal for Sick Children Great Ormond Street in London hope to take the patient's own bone marrow cells, genetically engineer them with healthy copies of the gene, and then inject the cells back into the child's bone marrow, where cells of the immune system are made. If successful, the child should then produce enough ADA.

Gareth Morgan, senior lecturer in clinical immunology at the hospital, said the approval from the committee 'opens the way for use of this form of therapy as an alternative to current treatments which, although of value, are not totally successful'.

The hospital emphasised that the risk to the child was probably less than the risk of other forms of treatment. The patient will be kept in isolation until the outcome of the trial is known.

Kay Davies, the director of the new Clinical Sciences Centre at Hammersmith Hospital in west London, which will conduct research into gene therapy, welcomed the ethical approval. 'It's a big step forward. It's essentially the green light for more proposals,' she said.

The Clothier committee, which is reviewing the ethics of gene therapy trials until the Government establishes a permanent regulatory authority, also looked at a second proposal but did not reach a decision. This trial will attempt to substitute the defective genes of children suffering from cystic fibrosis, the most common genetic disorder among north Europeans.

Doctors at the Royal Brompton and St Mary's Hospitals in London hope to insert healthy copies of the cystic fibrosis gene into cells lining the lungs in an attempt to stop the production of a sticky mucus that blocks respiratory passages.

Professor Bob Williamson, at St Mary's Hospital, said the proposal was experimental and so the safety issues had to be looked at carefully.

Gene therapy has begun in the United States on two children suffering from ADA deficiency.