Analysis: Judgment reflects desperation of struggle against vCJD

Six years after the new strain of Creutzfeldt-Jakob Disease, variant CJD, was identified and linked with BSE in beef, it continues to be a threat, despite the low numbers – 119 to date – that have fallen victim to it. Scientists, however, still have no way of knowing what the final toll may be.

The grip the disease has on public life was reflected by two developments yesterday. The Government announced it had bought an American supplier of blood plasma, Life Resources Incorporated, for £50m to ensure the NHS has a secure long-term supply of blood products such as albumin for burns victims and Factor VIII for haemophilia patients.

The NHS has obtained plasma for blood products from foreign sources since 1998 to minimise the risk of transmission of CJD. Although there is no documented case of CJD transmitted through blood, plasma is pooled from thousands of donations, increasing the risk over the transfusion of red blood cells which comes from a single donor.

The second, and more important development is a High Court legal ruling which has allowed an experimental drug to be injected into the brains of two teenagers in a last-ditch effort to save their lives – despite medical advice to the contrary.

The case not only sets a legal precedent for the use of experimental treatments in people with terminal illnesses but shows the devastating impact of vCJD on its victims and their families.

Don Simms, the father of one of the teenagers, learnt that his 18-year-old son Jonathan had vCJD and did what any parent would do. He refused to give up hope until he had exhausted every possibility of finding a treatment for him.

The first signs of the disease were clumsiness and lack of balance. Mr Simms noticed that when Jonathan walked through a doorway late in the evening he would have to hang onto either side of it. Within weeks he was unable to stand without help.

When the diagnosis came, it was shattering. "My wife and I were just numb. I knew the consequences, I knew that it was serious. I knew that it was fatal," Mr Simms said.

Doctors said there was nothing that could be done. But on the internet Mr Simms found details of pentosan polysulphate, a drug used for the treatment of bladder and bowel inflammation in the United States but which has no current licence in this country. Experiments on rats in the late 1990s had shown that it could delay the progression of CJD, following research in the 1980s which suggested it could be effective against scrapie, the disease in sheep similar to BSE in cattle.

Mr Simms approached the doctors caring for his son but the hospital trust refused to allow the procedure. Undaunted, he found a hospital in Germany prepared to do it and hired an air ambulance to take his son there. But as they were preparing to make the trip, the German hospital pulled out.

Appeals to the Government came to nothing. The Committee on Safety of Medicines first considered the role of pentosan polysulphate in February 1999 and rejected it on the grounds that there was no information that could "form a rational basis for prescribing" the drug.

The issue was examined again in 2001 and in October 2002 and the committee reached the same conclusion – that further research in animals was necessary before the treatment was tried in humans.

The CJD Therapy Advisory Group reached a similar conclusion, that there was insufficient data on which to base a treatment regime. But the group added a crucial rider: "Members acknowledged that healthcare professionals may be more risk averse than patients or their families when considering potential treatments for incurable disease such as CJD."

The added clause led Mr Sims to take his case to the High Court, together with the parents of the 16-year-old girl, whose name has not been released. Doctors needed the sanction of the courts to deliver the treatment because it was untried, dangerous and the patients were not capable of giving consent.

Mr Simms said before the hearing: "My son would not only have wanted me to do what I am doing now – he would have expected me to do it. If he has the treatment and it doesn't work at least I know I have done everything I can to help him."

In reaching her decision to allow the experimental treatment, Dame Elizabeth had to confront several ethical dilemmas. Because the treatment is outside mainstream medicine – the drug is not licensed – consent is crucial. But neither patient is capable of giving consent and under the law no one is able to consent on the part of an adult over 16.

Where a mentally incapacitated patient requires treatment that could be controversial – an abortion, for example – doctors have to seek the permission of the courts. In those cases the courts would normally expect the treating hospital to have obtained a second opinion, such as the agreement of a specialist outside the medical team.

Vivienne Nathanson, head of science and ethics at the British Medical Association, said: "It is about protecting patients from treatments that are not in their best interests. This is especially true in research involving experimental treatments. The courts rightly say you must be very careful to ask whether the treatment is for the benefit of the individual or for the benefit of others. It is not acceptable if it is only for the benefit of others."

When patients (or their relatives) are desperate they will try anything. As there is no treatment for CJD and it is invariably fatal they may think they have nothing to lose. But the CJD Therapy Advisory Group warned that there was a risk of side effects with pentosan polysulphate, including "bleeding events" and "hypersensitivity reactions". Nor is there information, it pointed out, on the "ideal route, dose, duration or timing" of the treatment.

Following the judgment, the two families must now hope they can find a neurosurgeon willing to carry out the procedure and a hospital trust that will allow it to proceed.

If they succeed, they will face an anxious wait to see the effect of the treatment. It is accepted that the drug cannot cure the disease. At best, it may slow down or arrest its progression. But even that would be a miracle.

The judge - Dame Elizabeth Butler-Sloss

Few judges can match the instinctive and sensitive judicial qualities of Dame Elizabeth Butler-Sloss, whose rulings often involve grappling with the most taxing moral issues of the day.

Earlier this year her handling of the case of Miss B won her praise from the medical and legal professions. In that judgment, she ruled that a patient had the right to refuse medical treatment even if it meant she would die.

Dame Elizabeth, the most senior female judge in England and Wales, has become the number one choice for deciding on cases for which there are often no right answers.

The pressure under which she works was plain to see during a revealing moment in the case of Miss B when she told the patient, via a video link to her bed, that the "tangled and conflicting emotions of the case could cloud her judgment". She wondered out loud if she "might be moved by emotion".

A judge of less experience or less confidence in their ability to separate the law from the highly-charged emotion might not have been quite so open about the fear of giving in to her feelings.

For almost 30 years, Dame Elizabeth – who was asked yesterday whether two vCJD patients could be treated with an unproven drug – has ruled on some very important cases of public policy.

She was appointed president of the Family Division of the High Court, one of the four most senior judicial posts, in June 1999. At the time, this promotion was recognised as a significant achievement for a woman in a profession where few make it to high office.

Dame Elizabeth, who is 68, first came to public attention when she was universally praised for her stewardship of the Cleveland child abuse inquiry in 1987 and 1988. The Children Act 1989 implemented her recommendations and was aimed at ensuring that children could not be summarily removed from parents to "places of safety".

Children, she has said, should not be removed from homes unless absolutely necessary and are "entitled to respect and consideration".

Her rulings have also included overriding the wishes of parents when their 15-year-old daughter, already a mother, sought an abortion.

Four years ago she issued landmark guidelines in a case in which a woman was forced to have a Caesarean delivery against her wishes, saying courts would no longer approve such surgery without a patient's consent.

Last year, she ruled that the killers of James Bulger should be granted life-long anonymity after their release.

Dame Elizabeth's career is nearing its end. A grandmother, she is expected to step down before reaching her compulsory retirement age in seven years. But who else has the immense judicial capabilities to step into her shoes when she bows out of public office?

Robert Verkaik