Go-ahead for gene therapy offers hope to thousands

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The Independent Online
THE FIRST trial in Britain involving the revolutionary technique of gene therapy - which holds out hope to hundreds of thousands of children and adults suffering from genetic diseases and cancers - will be approved by the Government this week.

In gene therapy, healthy genes are inserted into a patient with a genetic disorder. Approval of trials will launch a new chapter in British medical science.

The Government is expected to ask British doctors and researchers to bring forward for approval a range of proposals designed to lead to effective treatment and possibly cures for up to 4,000 genetic diseases, such as cystic fibrosis, that are caused by single defective genes. Gene therapy can also be used to treat cancers, with cells genetically modified to release a toxic compound inside the tumours. Gene therapy trials have already begun in the United States.

Soon, probably this week, the Government will announce that a permanent advisory body will be set up to review applications to conduct gene therapy. The body is expected to consist of a small committee of the 'great and good', and a larger group of specialist advisers. No names have been mentioned, but it is understood that some people have been approached. They could include leaders of the calibre of Dr John Habgood, Archbishop of York.

This would implement the main recommendation of the Committee on the Ethics of Gene Therapy, chaired by Sir Cecil Clothier, the former National Health Service ombudsman, which a year ago recommended the use of gene therapy subject to strict safeguards.

Scientists want to take a cautious approach because as a new and unknown treatment there may be hidden dangers for patients. Commentators are also worried that one day there may be pressure to practise gene therapy on the reproductive cells - sperm, eggs and embryos - which would irrevocably alter the genetic makeup of future generations. The Clothier committee is firmly opposed to such practice.

David Shapiro, a spokesman for the Nuffield Council on Bioethics, an independent body studying the ethical issues, said: 'Until now there has been no machinery to regulate gene therapy. There has been a growing feeling among researchers that it would make sense to be allowed to get on with some experiments in cases of grave illness. This will be a very welcome and desirable step.'

On Tuesday, the committee is expected to give the go-ahead to Britain's first gene therapy trial, involving a child who inherited a rare immune-deficiency disease. Doctors led by Professor Roland Levinsky, director of the Institute of Child Health at the Great Ormond Street Hospital in London, have been waiting for permission to give the child an injection of genetically-engineered blood cells.

The medical team is likely to follow closely the clinical protocol used two years ago in the US where doctors at the National Institutes of Health in Bethesda, Maryland, injected two girls with genetically modified cells containing a healthy gene for an enzyme called ADA - adenosine deaminase - which is vital to the correct functioning of the immune system.

Both girls have responded well to treatment. However, the technique is not a cure; it requires repeated injections. The next stage is to try to replace the cells lying deep within the bone marrow that are responsible for the continuous production of the cells of the immune defences. If successful, the doctors may be able to cure the genetic disorder and in so doing reverse nature's fate.

A second gene therapy proposal is being considered by the Clothier committee although this is 'further down the line', a Department of Health source said. This involves inserting healthy genes into the lungs of children suffering from cystic fibrosis, the most common genetic disorder of north Europeans. Professor Bob Williamson at St Mary's Hospital in London expects to begin clinical trials later this year.

In a further move this week, the Medical Research Council will unveil plans for research into gene therapy at its new Clinical Sciences Centre at the Royal Postgraduate Medical School in Hammersmith, London.

Next week in the 'Sunday Review': the start of a major series on the new genetics