Title : Therapeutic approaches for prion diseases: A systematic review
Abstract:
Background: Prionic diseases, such as Transmissible Spongiform Encephalopathies (TSEs) and Creutzfeldt-Jakob disease (CJD), are rare and fatal neurodegenerative disorders caused by the abnormal buildup of misfolded prion protein (PrPsc). Currently there are no efficient therapies available when it comes to prionic diseases, which highlights the importance of evaluating the impact and possible repurpose of many available drugs against these Encephalopathies.
Objective: This systematic review intends to compare pharmaceutical possibilities using medication available (such as Flupirtine, Doxycycline, Quinacrine, Monoclonal antibody PRN100 and Pentosan Polysulfate).
Methodology: The research consisted of searching through Clinical trials in PUBMED, SCIELO, Cochrane and BVS databases.
Results: Initially were found 12395 articles, which resulted in 8 articles after the application of inclusion and exclusion criteria.
Discussion: Flupirtine demonstrated relevant cognitive benefits (improving ADAS-Cog and MMSE scores), while monoclonal antibody PRN100 showed potential in modulating disease progression, despite inevitable advancement. Doxycycline showed a significant increase in survival in one of the studies. Quinacrine had inconsistent results, and Pentosan Polysulfate Sodium was associated with significant safety concerns. Common limitations were small samples, heterogeneous disease stages and the lack of early biomarkers.
Conclusion: Flupirtine and PRN100 emerge as the most promising approaches for managing CJD. Future research must prioritize early intervention, randomized controlled trials along with possible combined therapy.
Keywords: Prions. Transmissible Spongiform Encephalopathies. Creutzfeldt-Jakob disease. Anti-prion therapy. Clinical trials.
