Pioneering treatment for CJD to be given to a UCLH patient 

08/10/2018 00:00 
In a world-first, a patient of University College London Hospitals NHS Foundation Trust (UCLH) is set to be given a pioneering treatment for Creutzfeldt-Jakob disease (CJD).

CJD is a rare but devastating disease that causes brain damage and for which there is currently no treatment. It is always fatal and most patients sadly die within six weeks of diagnosis.

Researchers at the Medical Research Council (MRC) Prion Unit at University College London (UCL) have developed an antibody, called PRN100, for treating CJD. Laboratory testing of the antibody has been encouraging but it has not yet been used in patients.

UCLH is set to give the antibody to a patient for the first time in the world after a judge in the Court of Protection today confirmed that it is lawful and in the patient’s best interests to receive the unlicensed treatment.

UCLH’s chief executive Professor Marcel Levi said: “Creutzfeldt-Jakob disease (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.”

Professor Levi added: “UCLH is an innovative healthcare institution committed to ensuring our patients have access to the most cutting edge treatments available.”

Sporadic CJD, the most common form of the disease in humans, 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.

Our immune system produces antibodies to fight infections which invade the body. However, as abnormal prions are made of one of the body’s own proteins, our immune system does not make antibodies to fight them.

Professor John Collinge, director of the MRC Prion Unit at UCL, who led the development of the PRN100 treatment, said: “The treatment is an artificially manufactured antibody which has been created in the laboratory.

“The antibody has been designed to bind tightly to normal proteins in the brain. The aim is to prevent abnormal prions from being able to attach themselves to healthy proteins, meaning that they cannot grow and cause devastation throughout the brain.

“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.”

The patient and their family expressed their wish to receive this treatment and supported UCLH’s court application.

The patient will initially receive the treatment by a drip into a vein in the arm. They will be monitored around the clock by a team of experts from UCLH.

We are preparing for a range of possible outcomes including the treatment having no measurable effect and the treatment slowing or halting the progression of the disease. The treatment is not expected to reverse any brain damage that has already occurred.

The patient may also experience side effects which could lead the clinical team to limit or halt the doses of the treatment given.

In order to provide this treatment to a patient, UCLH created an oversight group, independent of the MRC Prion Unit and treating clinicians. The group comprises world-leading experts from a range of disciplines and it has met regularly with lawyers and patient advocates from the charity Cure CJD Campaign. The group considered the numerous and complex clinical, safety, legal and ethical issues arising from the potential use of this unlicensed treatment for CJD.

We will await the response of the first patient to this treatment before we consider a second patient.

A Q&A about this treatment

For further information please visit our frequently asked questions section on Prion diseases and Creutzfeldt-Jakob disease (CJD) on our website.


Patient advocate: Colin Beatty, Cure CJD Campaign charity

Colin Beatty lost his wife, Annie (pictured right), to sporadic CJD in 2010. Annie was 70-years-old and the couple had been married for 40 years.

“The diagnosis was devastating. It was like a bomb had gone off in our family,” said Colin.

“Annie was a former nurse so she knew what CJD meant and she was very frightened.

“It was a very difficult time. We cried many tears but I felt like it was my job to keep the family strong.”

Colin, 75, from Dorset, said the illness made Annie vacant in the beginning. Then her speech became muddled and she would wander off without warning, which he said was terrifying.

“It was heartbreaking to watch Annie deteriorate.

“I nursed her at home initially but she became very difficult to care for so we had to admit her to a nursing home.”

Colin said if the PRN100 antibody had been available at the time, he would have wanted Annie to have had the opportunity to be treated with it.

“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.

“For us, the decision about whether or not to have the treatment would’ve been a no brainer.”

Following Annie’s death, Colin became involved with the Cure CJD Campaign charity. He has represented the charity on UCLH’s PRN100 oversight committee.

“I got to know the team at UCLH’s National Prion Clinic very well when Annie was ill.

“Since we lost Annie, I have been committed to helping the team find a cure for this terrible disease.

“I don’t want other people to experience what we went through.”

Colin and Annie met in 1967 while they were working on a cruise ship which travelled between New York and Bermuda. Annie was the ship’s nursing sister and Colin was an electrical engineer.

“I was besotted with Annie. She later told me that as soon as she saw me, she knew I was the man she wanted to marry.

“She was a bubbly, popular person who was always laughing.

“She was a wonderful and supportive wife. We did everything together – we were so close as a couple and as a family.”

Colin and Annie have two sons, Chris and Nick, and four granddaughters.

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