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Last Updated: Thursday, 25 September, 2003, 23:00 GMT 00:00 UK
Q&A: vCJD treatment
An experimental vCJD drug may have succeeded in slowing the advance of the disease in one teenage patient.

Jonathan Simms has still been left heavily disabled by his illness - but is alive, despite doctors' predictions he would be dead by last Christmas.

BBC News Online, which broke the story of his family's struggle for the drug last year, examines the prospects for this revolutionary drug treatment.

What is the drug treatment involved?

The treatment is a drug called pentosan polysulphate.

This drug has been around for years, but has never been recommended as a drug to treat people with CJD.

In fact, although it has been used in the past as a treatment for bowel or bladder inflammation, it has no licence for any use on humans at the moment in the UK.

Some vets use it as an effective drug for arthritis in dogs.

Because vCJD affects the brain, any drug treatment for people already showing signs of the disease must reach that organ.

However, if an injection of pentosan was given to the body, the natural filter between the bloodstream and the brain would stop it entering.

So in order for pentosan to have any chance of working, it must be injected directly into the brain itself.

How does it work?

Increasing evidence from animal and laboratory experiments suggests that pentosan seems to have the ability to latch onto "prions" - tiny body proteins which appear to be the cause of vCJD damage.

There is the suggestion that it may be able to actively transport these prions out of brain cells.

In animal studies, giving pentosan appeared to lengthen the incubation period of the disease.

Scientists say that pentosan is an established drug, and appears to be relatively non-toxic.

Of course, animal studies are not always a reliable indicator of success in humans, but scientists who support pentosan say there is enough evidence to justify its use in human patients, particularly those with advanced disease, for whom there is no alternative.

Why is this controversial?

The family of Jonathan Simms has had extreme difficulty finding a hospital prepared to carry out the procedure needed to pump pentosan into his brain.

The objections they encountered were that the risks of injecting the drug into the human brain were unknown, and although Jonathan was 18, and should make his own decision whether to go ahead or not, in his condition he was incapable of giving the go-ahead.

Attempts to get the treatment carried out abroad foundered.

Eventually, an application was made to the High Court asking a judge to decide whether it was in his best interests to proceed.

Dame Elizabeth Butler Sloss concluded that, given the terminal nature of Jonathan's illness, the risks involved with pentosan treatment were acceptable.

What progress has he made since?

Jonathan has now been on the drug for nine months - with no ill effects.

The delays involved in obtaining the drug meant that his condition had deteriorated greatly.

He was bedridden, heavily restricted in his movements, and barely aware of his surroundings.

The first positive sign is that Jonathan is alive - he has now lived more than 10 months longer than the average lifespan of a vCJD patient following diagnosis.

His condition has not got any worse, as might be expected in such a normally rapidly-progressing illness.

In fact, his family has actually noticed important improvements.

His swallowing reflex has returned - a sign of improvement in a key area of the brain stem.

This is vital because most vCJD patients die because they can't swallow their own saliva properly, the liquid ends up in their lungs, where it contributes to pneumonia.

In addition, he has become more aware of his surroundings, and interacts more with his family - even responding to verbal commands.

Doctors say that, given the level of probably irreversible brain damage Jonathan had suffered prior to his pentosan treatment, it is too much to expect much more from the drug.

Do any tests suggest an improvement?

It is hard to perform blood tests or brain activity tests looking for an improvement, because doctors simply do not know what they are looking for, as Jonathan is the first patient to undergo this treatment.

However, an innovative test which looks for variations in heart rate may offer a clue to the effects of the drug.

Damage to a part of the central nervous system called the brain stem is thought to cause a "variability" in heart rate common to vCJD patients.

After the drug was given, there were distinct changes in this variability measured by doctors in Manchester.

This suggests that the drug may be having an effect on the brain stem.

What about the future?

No-one has given up hope that Jonathan may recover further, although it may take breakthroughs in other areas, such as stem cell research, to repair the damage vCJD has done to his brain.

What experts are hoping is that the drug has "bought him some time", halting the progress of the disease for months or perhaps even years.

It is too early to tell whether this is the case.

However, this research does offer hope that patients at earlier stages of vCJD may have an option which could slow down their own decline.

CJD drug gamble 'helps teenager'
25 Sep 03  |  Health


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