British man dying with human variant of ‘mad cow disease’ to become first to be given the only cure for the disease
A British man dying with CJD is to become the first person in the world to receive the only treatment for the disease.
A judge at the Court of Protection last night gave the go-ahead for the pioneering treatment.
Until now there has been no treatment for Creutzfeldt-Jakob disease – the human variant of bovine spongiform encephalopathy (BSE), known as ‘mad cow disease’.
The disease is rare but fatal, usually killing patients within six weeks.
Scientists at University College London’s Prion Unit, which was established by the Government in the wake of the 1990s BSE outbreak, have spent 20 years working on the treatment.
Scientists at University College London’s Prion Unit, which was established by the Government in the wake of the 1990s BSE outbreak, have spent 20 years working on the treatment, a synthetic antibody called PRN100 [File photo]
The treatment – called PRN100 – is a synthetic antibody they hope will stop CJD attacking the brain.
Laboratory tests have been encouraging but until now no human has received the treatment.
The difficulty has been that the treatment is experimental, unproven and may prove dangerous.
Because CJD strikes so quickly, by the time patients are diagnosed they often do not have the mental capacity to consent to receiving the treatment.
But last night Mr Justice Cohen gave specialists the go-ahead to treat a man with sporadic CDJ after analysing evidence at a hearing in London.
The family of the man, who is married and comes from eastern England, requested the treatment.
Mr Justice Cohen, who ruled the patient could not be identified or his age given, said: ‘Every day he spends with his family is special to him.
‘He wants that to continue as long as possible.
‘It seems to me it is plainly in his best interests that he should have treatment and that I should approve it.’
Professor John Collinge, director of the Prion Unit, said: ‘This is a very important milestone for us and for patients.
Until now there has been no treatment for CJD – the human variant of bovine spongiform encephalopathy, known as ‘mad cow disease’ [File photo]
WHAT IS CREUTZFELDT-JAKOB DISEASE?
Creutzfeldt-Jakob disease (CJD) is a rare, fatal condition that causes irreversible brain damage.
It affects around three in every one million people annually in the US. Its UK prevalence is unknown.
Symptoms, which worsen rapidly over time, include:
Most sufferers die within a year of symptom onset, usually due to an infection caused by immobility.
CJD occurs due to abnormal proteins known as prions, which cause nerve-cell damage.
Prions can occur spontaneously, be inherited or transmitted on contaminated surgical equipment.
There is no cure.
Treatment focusces of relieving symptoms and making sufferers comfortable via painkillers and antidepressants.
Source: NHS Choices
‘As this is the first time this treatment has been used in humans we cannot predict what the outcome will be but laboratory testing has shown the potential to treat prion infection.
‘A key issue will be whether a sufficient quantity of the drug is able to cross the blood brain barrier to reach the brain tissue where it needs to work.
‘We will proceed with extreme caution in very tightly controlled conditions. A team of experts from a range of disciplines will make collective decisions in the best interests of the patient.’
Sporadic CJD, the most common form of the disease in humans affects roughly 90 people in the UK every year.
It is similar to the variant CJD form, which is thought to be caused by eating meat from cows with BSE.
CJD is caused when healthy proteins which exist normally in the human body become spontaneously misshapen and build up in the brain.
These misshapen proteins, which are called prions, stick to other healthy proteins causing them to become misshapen too and the disease spreads through the brain.
Normally the human immune system produces antibodies to fight infections which invade the body.
But because abnormal prions are made of one of the body’s own proteins, the immune system cannot recognise or fight them.
The PRN100 treatment uses an artificial antibody which stops prions sticking to healthy proteins.
The man will initially receive the treatment by a drip into a vein in his arm and will be monitored around the clock by a team of experts from University College London Hospital.
The treatment is not expected to reverse any brain damage that has already occurred – but the team hopes it will halt further damage.
Professor Marcel Levi, chief executive of University College London Hospitals NHS Foundation Trust, said: ‘CJD is a rare and cruel disease which rapidly destroys the brain, affecting memory, thinking, speech, balance, movement and behaviour.
‘There is currently no cure or treatment for CJD. At present, caring for patients with CJD involves trying to use medicines to alleviate symptoms only but sadly, the disease always results in the rapid death of the patient.
‘The court’s confirmation today is an important step forward in tackling this devastating illness.’
Colin Beatty, of the Cure CJD campaign, said: ‘It’s true that the treatment carries potential risks, and the benefits are not yet certain, but without it, there is no hope. The only certainty with CJD is death.’