18:37 17 December 02 NewScientist.com news service

The British High Court has given the go-ahead for two teenagers dying from vCJD to be the first to receive an experimental treatment. It would involve injections of a drug called pentosan polysulphate directly into their brains.

Pentosan polysulphate is currently used as an oral drug in people to treat cystitis and bladder pain. Research in the UK and Japan has shown that the drug can stop the progression of scrapie, a very similar disease, in mice. It worked by binding to a key site on cells and stopping abnormal prion proteins being created.

But research in the field is at an early stage. In October 2002, the UK's Committee on the Safety of Medicines reviewed all existing data on the drug and concluded it was too early to consider using it in human vCJD patients.

However, High Court Judge Dame Elizabeth Butler Sloss said on Tuesday: "Both families are very well informed about the disease and the proposed treatment and its risk and possible benefits." The parents of one of the teenagers, 18-year-old Jonathan Simms, "believe it is a worthwhile risk to try the treatment," she said.

Stephen Dealler, a microbiologist at the Royal Lancaster Infirmary, who has advocated testing the drug in human patients, says: "It would have been immoral if the decision had gone any other way."

"It works in the test tube and in mice, we know it's not incredibly toxic because it's been used in people for 40 years. But we don't know the dose to try in people and we don't know for sure it will work - that's what hurts most scientists," he told New Scientist.

No cure

The two patients are too ill to give consent, although in the case of the 15-year-old girl, who has not been named, her parents are legally allowed to do so.

There is very little hope that the drug could offer any kind of cure for vCJD, since it has only been shown to halt the progression of scrapie in mice. "These children will probably get very little advantage from it. But when this High Court trial started, they were walking and talking," says Dealler.

A UK hospital trust that had been willing to administer the treatment has since retracted its offer. The UK's Department of Health is now helping the parents search for doctors who will help.

The molecule is too large to cross the blood-brain barrier, so doctors will have to inject it directly. The toxicity of the drug in the brain has never been tested in people, but researchers working with mice have reported few side effects, Dealler says. Ethically acceptable

Ethically, the decision is acceptable, says Vivienne Nathanson, director of professional activities at the British Medical Association. "No doctor will be forced to do something they believe is inappropriate. And patients should always have the right to request that a treatment - no matter how odd it might seem - should be considered," she says.

After the judgement, Don Simms, Jonathan's father, said: "We regard this treatment as experimental treatment, but it is not experiment for experiment's sake. The fact is, there is nothing else on the horizon at present. We think this treatment has potential, but no one knows how much."

In 2001, Stanley Prusiner at the University of California, San Francisco, reported that a drug, quinacrine, had relieved symptoms in a handful of vCJD patients. But one of the patients died shortly afterwards, and most researchers now think the drug is not an effective treatment.

-- Anonymous, December 18, 2002