First-in-Human Immunotherapy Treatment Trial in Creutzfeldt-Jakob Disease

Humanized monoclonal antibodies targeting PrP^c achieved encouraging brain and CSF concentrations with a favorable safety profile.

In Creutzfeldt-Jakob disease (CJD), host-encoded normal prion protein (PrP^c) is an attractive therapeutic target given the central role its misfolding into the pathogenic form (PrP^Sc) plays in prion propagation. Following cell-culture and animal-immunotherapy studies (Nature 2001; 412:739), researchers developed an investigational, fully humanized monoclonal antibody (PRN100) that targets and stabilizes PrP^c. PRN100 binds tightly to PrP^c to prevent its misfolding and combination with prions, halting prion propagation. In a first-in-human study of humanized monoclonal antibody treatment in CJD, researchers assessed the effect of PRN100 on the progression of CJD while monitoring for adverse events. Six patients (5 with sporadic and 1 with iat…

Reviewing Author

John C. Probasco, MD

Disclosures

Grant/Research Support Genetech/Roche

Editorial BoardsThe Neurohospitalist

Leadership Positions in Professional SocietiesNeurohospitalist Society (President; Chair, Research Subcommittee)

John C. Probasco, MD

Disclosures

Grant/Research Support Genetech/Roche

Editorial BoardsThe Neurohospitalist

Leadership Positions in Professional SocietiesNeurohospitalist Society (President; Chair, Research Subcommittee)

First-in-Human Immunotherapy Treatment Trial in Creutzfeldt-Jakob Disease

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Reviewing Author