Gene therapy funding a 'drop in the ocean'

Experts worry what impact the Government's White Paper on genetics can have if it's not matched with money. Katrina Lythgoe reports

When it unveiled its White Paper on genetics, the Government said that the NHS should use the new knowledge to the benefit of all patients, and that Britain should lead the world in genetics research and development. Today, we reveal the verdict of the genetics community: the paper marks a step in the right direction but they will need more money if it's to have an impact.Ê

This snapshot of their opinion has come from an exclusive poll commissioned by The Daily Telegraph, conducted with the help of Government adviser Prof John Burn and the British Society for Human Genetics . From a diverse range of fields and laboratories, 73 experts gave us their opinion on the extra £50 million the Government will spend. Nine out of every 10 polled had reservations about whether the additional investment would allow Britain to keep up with the rest of the world in the area of gene therapy. A quarter categorically said that we would fall behind.

The Government has said that it will invest an additional £3 million in gene therapy for the 4,000 single-gene disorders, and a further £2.5 million for cystic fibrosis, the most common of them. It has also allocated £4 million to pay for the vectors - viruses and other means to carry healthy genes into patients' bodies - that make gene therapy possible.

These amounts may sound like a fortune to the parents of a child suffering from a genetic disorder, but genetics research is not cheap. This amount of funding is a "drop in the ocean", as one expert put it. "Peanuts compared to charity work recently funded," said another senior geneticist. "This will make no difference."

To put these figures in perspective, consider the cost of gene therapy trials for the "bubble babies" who have X-linked severe combined immunodeficiency, X-SCID, which strips the body of the ability to fight infection.

Developing gene therapy to fight this disease cost Great Ormond Street children's hospital £1 million, and it costs £100,000 to treat a single child. The hospital will receive some extra funding from the Government but will still rely heavily on charities.

"Amounts like £3 million for gene therapy and £2.5 million for cystic fibrosis are almost insignificant on a national level," said Dr Stephen Hart, a molecular immunologist at the Institute of Child Health.

Not everyone agrees. Many experts questioned whether more money should be put into gene therapy at all. "In terms of progress towards treating genetic disease," said one, "I don't believe gene therapy is the most cost-effective area to target resources."

Although gene therapy has been a fashionable topic for a decade, success has been limited. The Great Ormond Street team is one of only three worldwide to have reported unambiguous positive results, and there are concerns about side effects.

"I am sceptical about gene therapy as research has been ongoing for many years with very little success," said one molecular geneticist. "I am concerned that the hopes of families with single-gene disorders, such as cystic fibrosis, are raised with the increased publicity of a 'cure'."

The second controversial issue concerns the NHS's ability to diagnose those who have faulty genes. As Professor John Burn, director of the Northern Genetics Knowledge Park, mentions in his accompanying article, the Government has set impressive targets for the testing of genetic disease. But more than eight out of 10 of our experts said they had reservations that the targets could be met.

"The target for identifying unknown mutations in genes seems unrealistic," said one. "This paper will fuel unrealistic patient expectation with no prospect of delivery by an already overstretched laboratory workforce," another senior geneticist added.

And therein lies the problem. You can pump all the money you like into new buildings and new equipment, but if there are not enough people processing the tests they will not be able to cope. The Government has allocated money to train 90 new laboratory scientists in recognition of this. What many of our experts wonder, however, is who will train them?

"There is an acute problem with shortages of trained staff, to the point that there is limited capacity for existing staff to train new individuals even if the promised funding becomes available," said one.

A consultant clinical geneticist added: "The real problem is in staff retention in the labs and that can only be addressed by improving pay." The trouble is, improving pay for laboratory technicians is not going to do much to improve the Government's profile.

Despite their reservations, most experts welcomed the White Paper - if only because it will put genetics in the spotlight, giving hope for better funding in the future. "Clearly it demonstrates a raised profile for genetics, at least until the next hot topic comes along," one geneticist replied.

"The UK is ahead of the United States and the rest of the world in many areas of genetic science and its clinical application," said Prof David Porteous, head of medical genetics at the University of Edinburgh.

"This is despite relatively low investment in basic science and a dreadful record on biotech investment. The Government investment is fully justified, but only sufficient to make modest changes in UK effectiveness. We could be global leaders. We have the knowledge base and we have the NHS, an unrivalled combination."

These words are at odds with the Government's gung-ho pronouncements: "Britain's academic and industrial research prowess means that we are already among the leading players in genetics research and development," writes John Reid, the Health Secretary, in his forward to the White Paper. "We are determined to harness that potential and ensure the benefits of genetics are realised throughout the NHS."

But one of our respondents, from the front line, remains gloomy: "I hope the Government doesn't believe its own publicity material, or we are in a worse mess than I thought."

23 July 2003