Four patients given new drug at UCLH to treat incurable brain disease die

The world-first treatment for sporadic Creutzfeldt-Jakob disease (sCJD) was first administered to a married father last October
PA
Ross Lydall @RossLydall9 October 2019

Four of the six patients given an unlicensed drug never before used in humans to treat an incurable brain disease have died, it can be revealed today.

The world-first treatment for sporadic Creutzfeldt-Jakob disease (sCJD) was first administered to a married father last October after University College London Hospitals NHS Trust won permission from the High Court.

The hospital had to apply to the Court of Protection because the man, whose family backed the move, did not have the mental capacity to give consent.

Today it was revealed that the drug, an antibody known as PRN100 that was created in a laboratory at the Medical Research Council’s prion unit at University College London, had been given to six patients at different stages of sCJD.

Of the four who died, two had been able to give personal consent to receive the treatment and two received it after court approval. A legal ruling prevents the patients being identified.

UCLH said it would not be treating any more patients with PRN100 “at this time” as supplies were almost exhausted. It had always planned to offer the drug only for a short period. None of the six patients suffered side-effects from the drug, which is infused into the bloodstream via a vein.

UCLH chief executive Professor Marcel Levi said: “Our sincere condolences go to the families of these four patients. CJD is a rare but cruel disease which is devastating for loved ones to witness. We are very sorry for their loss.”

Professor John Collinge, director of the Medical Research Council’s prion unit at UCL, said it would now analyse data “to determine if, or to what extent, the drug has had an impact on the patients’ conditions”.

SCJD is the most common of four strains of CJD, the most notorious of which is variant CJD, which is caused by infection from eating beef from cows with BSE or mad cow disease.

About 120 cases of sCJD are diagnosed in the UK each year. A few patients live for more than a year but most die within six months of symptoms, and typically within six weeks of being diagnosed.

At present, there is no cure for CJD.

SCJD is caused when healthy proteins become spontaneously misshapen and build up in the brain. These misshapen proteins, called prions, stick to healthy proteins, enabling the disease to spread.

The body does not create antibodies to fight the prions because they are not recognised as “foreign invaders”.

The build-up of prions causes brain cells to die, affecting memory, thinking, talking, balance, movement and behaviour. It leads to death. The aim of the treatment was to bind to normal proteins in the brain to form a defensive shield against the march of the prions.

The treatment was not expected to reverse brain damage that had already occurred, but it was hoped that it could slow or stop the progression of sCJD.

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