Creutzfeldt-Jakob Disease Treatment Shows Promising Early Results

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MRI Creutzfeldt-Jakob Disease
Credit: Pract Neurol. 2017 Apr; 17(2): 113–121

Scientists report encouraging early results from an experimental study on the world’s first in-human treatment for Creutzfeldt-Jakob disease.

Creutzfeldt-Jakob disease (CJD) is a rare, but fatal, neurodegenerative disease. It occurs due to the presence of prion proteins within the brain, damaging neurons and resulting in degeneration of the brain tissue. This defective protein is often transmitted into humans through the consumption of contaminated meat, infected brain products or even contaminated surgical instruments. Although researchers have experimented with several therapeutic agents for treating CJD, none have shown promise. Nor does there exist an authorized treatment for the disease. Now, researchers at University College London (UCL) have developed a monoclonal antibody for the treatment of Creutzfeldt-Jakob disease.

Recently, the team of researchers completed the first in-human treatment programme for the experimental therapy. According to the peer-reviewed study, the early results are ‘very encouraging’.

Called PRN100, the monoclonal antibody targets cellular prion proteins. Under a Specials License, researchers administered the drug to six patients with CJD. None of the patients developed any adverse events while undergoing treatment. Moreover, researchers reported a good concentration of the drug within the brain and cerebrospinal fluid of the patient. Thus, revealing the treatment is safe and easily transported to the brain.

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.

Professor John Collinge, lead developer of the PRN100 treatment

In three of the patients, the researchers noted a stabilization in their disease progression. However, all six patients passed away due to their condition later on. Nevertheless, the study demonstrates the safety of the new treatment and its potential for treating prion diseases. Due to the small sample size of the study, the study authors are regarding the results as preliminary. They now hope to conduct a larger study to assess its efficacy and safety.

Reference:

Prion protein monoclonal antibody (PRN100) therapy for Creutzfeldt–Jakob disease: evaluation of a first-in-human treatment programme, Lancet Neurology (2022). DOI:https://doi.org/10.1016/S1474-4422(22)00082-5

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