Treatment of Prion Disease with Heterologous Prion Proteins

PLoS One. 2015 Jul 2;10(7):e0131993. doi: 10.1371/journal.pone.0131993. eCollection 2015.

Abstract

Prion diseases such as Creutzfeldt-Jakob disease in humans, bovine spongiform encephalopathy in cattle, and scrapie in sheep are fatal neurodegenerative diseases for which there is no effective treatment. The pathology of these diseases involves the conversion of a protease sensitive form of the cellular prion protein (PrPC) into a protease resistant infectious form (PrPsc or PrPres). Both in vitro (cell culture and cell free conversion assays) and in vivo (animal) studies have demonstrated the strong dependence of this conversion process on protein sequence homology between the initial prion inoculum and the host's own cellular prion protein. The presence of non-homologous (heterologous) proteins is often inhibitory to this conversion process. We hypothesize that the presence of heterologous prion proteins from one species might therefore constitute an effective treatment for prion disease in another species. To test this hypothesis, we infected mice intracerebrally with murine adapted RML-Chandler scrapie and treated them with heterologous prion protein (purified bacterially expressed recombinant hamster prion protein) or vehicle alone. Treated animals demonstrated reduced disease associated pathology, decreased accumulation of protease-resistant disease-associated prion protein, with delayed onset of clinical symptoms and motor deficits. This was concomitant with significantly increased survival times relative to mock-treated animals. These results provide proof of principle that recombinant hamster prion proteins can effectively and safely inhibit prion disease in mice, and suggest that hamster or other non-human prion proteins may be a viable treatment for prion diseases in humans.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Cells, Cultured
  • Cricetinae
  • Disease Models, Animal
  • Disease Progression
  • Female
  • Gliosis / physiopathology
  • Gliosis / therapy
  • Mice
  • Mice, Inbred C57BL
  • PrPC Proteins / chemistry
  • PrPC Proteins / therapeutic use*
  • Prion Diseases / genetics
  • Prion Diseases / therapy*
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / therapeutic use*
  • Scrapie / therapy*
  • Treatment Outcome

Substances

  • PrPC Proteins
  • Recombinant Proteins

Grants and funding

This work was supported by a seed grant from the University of Minnesota Academic Health Center, PJS, and by the Intramural Research Program of the National Institute of Allergy & Infectious Disease, National Institutes of Health, SAP. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.