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Tuesday, 17 December, 2002, 14:29 GMT
Teenagers get CJD drug go-ahead
Scales of Justice
The case was heart in the High Court
Two teenagers dying from vCJD should be given an experimental treatment which might help them, a court has ruled.

However, a hospital trust which had been lined up to carry out the treatment is still refusing to do so, even though doctors there are happy to go ahead.

The landmark decision by the High Court means that the pair - Jonathan Simms, 18, from Belfast in Northern Ireland, and a 15-year-old girl, who cannot be named for legal reasons - will become the first in the world to receive the drug, pentosan polysulphate, injected directly into their brains.

We regard this treatment as experimental treatment, but it is not experiment for experiment's sake

Don Simms, father of 18-year-old patient
Some scientists are hopeful that it could stall the progress of the disease.

The case sets a legal precedent that could radically alter the way that unproven treatments are used in people with a terminal illness.

High Court Judge Dame Elizabeth Butler Sloss decided it would not be either "irresponsible or unethical" to treat the teenagers.

She ruled: "Where there is no alternative treatment available and the disease is progressive and fatal, it seems to me to be reasonable to consider experimental treatment with unknown benefits and risks, but without significant risks of increased suffering to the patient, in cases where there is some benefit to the patient."

About Jonathan, she added:"They would rather have him in his present condition than not at all, and believe it is a worthwhile risk to try the treatment."

She said she was "concerned" that the trust were still planning to refuse to carry out the treatment.

She said: "In the case of the families of those suffering from as tragic an illness would be an unbelievably cruel blow to have the High Court say yes to the treatment and the two committees of the hospital trust to say no."

Jonathan Simms
Jonathan Simms may receive the treatment
The treatment could now commence within weeks if a new hospital can be found - and could prompt a surge in demand for the drug to be given to other vCJD patients.

The High Court case was the culmination of months of behind-the-scenes pressure for the treatment to be carried out inside the NHS.

Although the families had found doctors willing to deliver the treatment, others were concerned that the treatment has not been tested properly in humans, and might actually cause harm.

But the families insist that it is their last hope.

The previous advice from vCJD experts in the UK was not to use pentosan - but that is now to be reviewed.

In each case the children have changed from normal, energetic teenagers into helpless invalids lying in bed and with a severely limited enjoyment of life

Dame Elizabeth Butler Sloss, High Court judge
After the hearing, Don Simms said: "We have come a long way from where we started and now it is up to the Health Secretary, Alan Milburn, to get my son treated.

"My son remains quite strong, although certainly there is a neurological difficulty. If we had got this treatment sooner, it would not have been so great.

"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."

Foreign failure

After an NHS hospital refused to perform the operation, unsuccessful attempts were made to transfer it abroad.

Don Simms
Jonathan's father Don has campaigned for the drug
The Department of Health is now helping the families find another venue.

However, David Body, the families' solicitor, confirmed that no hospital could be compelled to carry out the treatment.

Studies in animals have given some reassurance about safety and even boosted hopes that it might have an impact on the disease.

vCJD is thought to be caused by "rogue" proteins called prions, which corrupt other proteins in the brain and form clumps which slowly kill brain cells.

At present there is no available treatment that can reverse or even slow down this decline.

Pentosan, it is hoped, may be able to latch onto prions, rendering them less harmful, and perhaps allowing them to be flushed out of brain cells.

Unknown quantity

The drug molecule has to be injected directly into the brain because it is too big to pass from the bloodstream across a protective filter called the "blood brain barrier".

What doctors are unsure about is how much pentosan would have to be given to have an impact on vCJD in the human brain, and how much it is safe to give.

Some studies have already suggested that it could have a role in patients who have been "infected" with vCJD prions, but have not yet developed clinical signs of the disease.

Statistical experts say that although the number of people who have so far developed vCJD, probably as a result of exposure to BSE infected meat during the 1980s and early 1990s, is currently only in the hundreds, the true numbers likely to go on to develop it are unknown, as it may incubate for decades in the body.

The BBC's Karen Allen
"The families never wanted this to end up in court"
Don Simms, father of 18-year-old patient
"I cannot stand idly by and watch Jonathan die"
See also:

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