Tested on six patients, this treatment would have stabilized the progression of Creutzfeldt-Jakob disease in half of them.
- Affecting humans, but also livestock and wild animals, prion diseases, including Creutzfedlt-Jakob disease, are characterized by rapid and fatal degeneration of the central nervous system.
- According to Santé Publique France, 28 cases of Creutzfeldt-Jakob disease were diagnosed in France between 1992 and 2019. The last known French case died in 2019.
A rare but devastating disease, Creutzfeldt-Jakob disease (CJD) is a neurodegenerative disease causing progressive neurological disorders with fatal outcome after a long and silent incubation period.
If there is currently no treatment, this could soon change. Scientists from the Prion Unit of the Medical Research Council (MRC) at University College London have just announced the preliminary results. “very encouraging” of a potential treatment. They are published in The Lancet Neurology.
Monoclonal antibody treatment
This potential treatment uses a monoclonal antibody called PRN100, which was administered to six CJD patients between October 2018 and July 2019.
As explained by Professor John Collinge, director of the MRC Prion Unit at UCL, who led the development of the PRN100 treatment “drugs used to treat other diseases have been tried experimentally to treat CJD in the past, but none have had an impact on disease progression or mortality”.
This is not the case with this new experimental treatment. The results show that in addition to being safe, it is able to access the brain. In three patients, disease progression even stabilized when dosage levels were within the target range.
“This is the first time in the world that a drug specifically designed to treat CJD has been used in humans and the results are very encouraging”continues the researcher.
While none of the six patients experienced side effects during treatment, all of them unfortunately died as a result of their illness.
A large clinical trial planned
Given the small number of patients treated, the researchers say the results should be considered preliminary and that further studies are needed to draw more complete conclusions. “Although the number of patients treated is too small to determine whether the drug modifies the course of the disease, it is nevertheless an important advance in the targeting of prion infections”they point out.
“It has been a huge challenge to reach this milestone and we still have a long way to go, but we have learned a lot and these results now justify developing a formal clinical trial in larger numbers of patients.”
“We hope that the drug can also prevent the onset of symptoms in people at risk for prion disease due to genetic mutations or accidental exposure to prions, and that it can contribute to the development of therapies for dementias. more common, such as Alzheimer’s disease”adds Professor Collinge.
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