Prion protein monoclonal antibody (PRN100) therapy for Creutzfeldt-Jakob disease
MRC Prion Unit and Institute of Prion diseases – UCL – University College London
A world-first treatment for Creutzfeldt-Jakob disease (CJD), developed by scientists at the Medical Research Council (MRC) Prion Unit at UCL, has shown “very encouraging” early results following its use in six patients at University College London Hospitals (UCLH) NHS Foundation.
CJD is a rare but devastating disease that causes brain damage and for which there is currently no licensed treatment. It is always fatal and most patients sadly die within a few months of diagnosis.
Researchers at the MRC Prion Unit at UCL have developed a monoclonal antibody, called PRN100, which was given to six UCLH patients with CJD between October 2018 and July 2019.
The results, published in the Lancet Neurology, show the treatment is safe and able to access the brain. In three patients, disease progression appeared to stabilise when dosing levels were in target range.
Given the small number of patients treated, researchers say the findings should be regarded as preliminary and further studies are needed to draw more comprehensive conclusions.
None of the six patients experienced side effects while receiving the treatment but all sadly died as a result of their condition.
Professor John Collinge, Director of the MRC Prion Unit at UCL and UCLH consultant neurologist, who led the development of the PRN100 treatment, said: “Drugs used to treat other diseases have been tried experimentally in treating CJD in the past but none has had an impact on disease progression or mortality.
“This is the first time in the world a drug specifically designed to treat CJD has been used in humans and the results are very encouraging.
“While the number of patients we treated was too small to determine whether the drug altered the course of the disease, this is nevertheless an important step forward in targeting prion infections.
“It has been a huge challenge to reach this milestone and we still have a long way to go but we have learned a great deal and these results now justify developing a formal clinical trial in a larger number of patients.”
Looking further into the future, Professor Collinge added: “We hope the drug may also have the potential to prevent the onset of symptoms in people at risk of prion disease due to genetic mutations or accidental prion exposure and may contribute to the development of therapies for more common dementias, such as Alzheimer’s disease.”
In a comment piece published alongside the results in the Lancet Neurology, Professor Inga Zerr, from the Department of Neurology at Georg-August University of Gottingen, Germany, also called for further studies in this area.
“These outcomes are very encouraging and long awaited but, in light of the limitations, such as the small number of patients included and the use of historical controls, these results must be considered preliminary,” she said.
UCLH provided the PRN100 drug to patients under a “Specials” exemption, rather than a regulated clinical trial. A “Specials” exemption permits a healthcare professional to treat an individual patient with an unlicensed drug when their special clinical needs cannot be met by a licensed product on the market.
Three of the six patients were able to consent to receiving the PRN100 antibody themselves. The other three did not have the capacity to consent, so with the support of their families, UCLH sought the opinion of a judge in the Court of Protection in order to proceed.
UCLH created an oversight group, independent of the MRC Prion Unit at UCL and treating clinicians, to consider the numerous and complex clinical, safety, legal and ethical issues arising from the potential use of this unlicensed treatment. The group comprised world-leading experts from a range of disciplines and met regularly with lawyers and patient advocates from the Cure CJD Campaign.
Professor Bryan Williams, director of the National Institute for Health Research (NIHR) UCLH Biomedical Research Centre (BRC), said: “UCLH is a bold healthcare institution which, along with its academic partner UCL, is always seeking to push the frontiers of medicine and science to deliver innovative treatments to patients.
“Creutzfeldt-Jakob disease (CJD) is a rare and cruel disease which rapidly destroys the brain and for which there is currently no cure or licensed treatment. It was extremely important to us to find a way through the many challenges arising from the potential use of this novel treatment in order to offer it to a small group of patients.
“We are encouraged by these results which demonstrate the treatment is safe and there is some signal of benefit. The hope is that this could pave the way for new treatments for other neurodegenerative diseases.”
Patient story
Carole Kiralyfi was one of the six patients to receive the PRN100 antibody.
Her husband, Laszlo, said a fall during a game of tennis in January 2019 was one of the first signs that something was not right with Carole, who was 70 years old at the time. Her vision then began to deteriorate and she had difficulties managing everyday tasks.
When Carole was diagnosed with sporadic CJD in March 2019, Laszlo said the whole family was “absolutely devastated”.
“It was such a shock, everything happened so quickly. Carole had always been so healthy and active – I had always thought she would outlive me.”
With Laszlo’s support, Carole decided to receive the PRN100 drug after being thoroughly assessed by neurologists at UCLH’s National Prion Clinic.
“Carole came to terms with her diagnosis a lot better than we (her family) did – she was not afraid. The drug was our only option so we decided to go ahead.”
Carole sadly died of her condition in April 2019 before the target level of PRN100 was achieved but Laszlo takes comfort in the fact that she may have contributed to the development of a potential treatment of the future.
“Obviously, I wish there could have been a treatment for Carole but if we are one step closer to achieving that now, it means her death was not in vain.
“The team that looked after her was so caring and compassionate – they have dedicated their lives to finding a cure for this terrible disease so I want this for them too.
“CJD may be rare but it is devastating and that is why it is so important there is more research in this area.”
Paying tribute to his wife of 28 years, Laszlo, who is now 74, added: “She was an extraordinary person with a big heart; a wonderful wife and fantastic mother. She was very popular and had many friends – when she walked into a room it lit up and when she left the light remained. She will remain forever in our hearts.”
Original Article: World-first CJD treatment shows promising early results
More from: University College London
The Latest Updates from Bing News & Google News
Go deeper with Bing News on:
Creutzfeldt-Jakob disease
- Humans not at risk of deadly chronic wasting disease: Study
A substantial species barrier is preventing the spread of deadly chronic wasting disease (CWD) seen in deer, elk, and moose to humans, found a study.
- NIH study shows chronic wasting disease unlikely to move from animals to people
A new study of prion diseases, using a human cerebral organoid model, suggests there is a substantial species barrier preventing transmission of chronic wasting disease (CWD) from cervids—deer, elk ...
- Study suggests chronic wasting disease unlikely to move from animals to people
Over the next decade, 178 people in the U.K. who were thought to have eaten BSE-infected beef developed a new form of a human prion disease, variant Creutzfeldt-Jakob Disease, and died. Researchers ...
- Study: Human barrier to chronic wasting disease substantial
The infectious agent that causes chronic wasting disease – an always fatal affliction infecting moose, deer and elk – did not infect human neural tissue, a newly published study found. “Overall, the ...
- Human barrier to chronic wasting disease substantial, Hamilton lab study finds
The information provides a temporary sigh of relief for many hunters who fear contracting the infection, which causes holes in infected animals’ brain tissue.
Go deeper with Google Headlines on:
Creutzfeldt-Jakob disease
[google_news title=”” keyword=”Creutzfeldt-Jakob disease” num_posts=”5″ blurb_length=”0″ show_thumb=”left”]
Go deeper with Bing News on:
PRN100 drug
- Drug safety articles from across Nature Portfolio
Drug safety relates to the potential for adverse effects related to the administration of drugs. Efforts to establish the safely profile of drugs begin early in their development, with in vitro ...
- U.S. drug deaths declined slightly in 2023 but remained at crisis levels
Preliminary data released Wednesday from the Centers for Disease Control and Prevention found fatal drug overdoses in the U.S. fell by roughly 3% in 2023. That is a significant reversal from ...
- US drug overdose deaths decreased in 2023 for the first time in five years
After a steep rise during the Covid-19 pandemic, preliminary data shows that drug overdose deaths in the United States ticked down in 2023 for the first time in five years. About 107,500 people ...
- In a decade of drug overdoses, more than 320,000 American children lost a parent
More than 320,000 children across the United States lost a parent due to a drug overdose between 2011 and 2021. That's according to a new study published in JAMA Psychiatry Wednesday. "It's a call ...
- British Columbia to back off drug decriminalisation project
But it has faced pushback over concerns around public disorder and drug use. Police will soon have the power to again address drug use in all public places, including hospitals, restaurants ...
Go deeper with Google Headlines on:
PRN100 drug
[google_news title=”” keyword=”PRN100 drug” num_posts=”5″ blurb_length=”0″ show_thumb=”left”]