Published: 25 March 2022
A first-in-human treatment designed specifically for Creutzfeldt–Jakob disease (CJD) has shown encouraging early results and may contribute to the development of new treatments for other neurodegenerative diseases.
CJD is a rare and fatal disease that causes brain damage and for which there is currently no licensed treatment. Most patients die within a few months of diagnosis.
Researchers at the NIHR UCLH Biomedical Research Centre (BRC) developed a monoclonal antibody, called PRN100, that was given to six patients with CJD between October 2018 and July 2019.
The results, published in 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. None of the six patients experienced side effects while receiving the treatment but all sadly died as a result of their condition.
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.
Professor John Collinge, Director of the MRC Prion Unit at UCL who led the development of the PRN100 treatment, said: “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.”
Professor Bryan Williams, director of the NIHR UCLH 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.”