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Continuing Education Column Human Prion Diseases SangYun Kim, MD Department of Neurology, Clinical Neuroscience Center, Seoul National University Bundang Hospital E - mail : neuroksy@snu.ac.kr Hae-Kwan Cheong, MD Department of Social and Preventive Medicine, Sungkyunkwan University College of Medicine E - mail : hkcheong@skku.edu Seong Soo An, PhD Department of Bionanotechnology, KyungWon University Gachon Bionanotechnology Research Institute E - mail : seongaan@kyungwon.ac.kr J Korean Med Assoc 2008; 51(12): 1125-1138 Abstract Transmissible Spongiform Encephalopathy (TSE) or prion diseases are fatal neurodegenerative diseases, which are caused by transmissible abnormal prion proteins, converting the endogenous normal prion in the body to the infectious abnormal prions. The most common form of human prion diseases is Creutzfeldt - Jakob disease (CJD). Most of CJD are sporadic with unknown cause. Some familial or iatrogenic CJDs are reported in many countries, but there have been no formally reported case in Korea. Variant CJD (vcjd) is a new form of human prion disease, which revealed differentiated clinical presentations and laboratory diagnostic results. vcjd was thought to be originated from eating the beefs or other parts of bovine spongiform encephalopathy (BSE) infected cattle. The unpredictable species barriers, the underestimated distribution of prion infected tissues, the variable clinical courses, and uncertain disease progressions of many prion diseases, all made the prion related risk assessment very difficult. Korea needs our own surveillance system for various prion diseases of human and animals and to make plans for the risk assessment of the various prion disease transmissions for the minimal spread by maximizing the research capacities. Keywords: Prion; Creutzfeldt - Jakob disease; Variant Creutzfeldt - Jakob disease; Transmissible spongiform encephalopathy 1125

Kim SY Cheong HK An SS Table 1. Comparison between sporadic and variant Creutzfeldt-Jakob disease Sporadic CJD Variant CJD Median age at death 68 years 28 year (18~53 year) Median duration of illness 4~5 months 13~14 months (8~38 months) Main clinical features Onset with rapidly progressive dementia Onset with psychiatric features Cerebellar ataxia Cerebellar ataxia Cortical blindness Dystonia / Chorea Pyramidal signs Pain / other sensory symptoms Myoclonic jerks Oculomotor symptoms Pyramidal signs Dementia Myoclonus Distribution Worldwide Mainly UK Cause Unknown BSE infection in diet Familial cases 10~15% No EEG Generalized theta and delta slowing only or Nonspecific slow waves only normal at early stage Sometimes normal even with Characteristic 1~2Hz triphasic sharp waves neurological symptoms in more than 80%before death CSF Normal cytology and chemistry except mildly Positive 14-3-3 protein in increased protein less than 50% Usually positive 14-3-3 protein Brain CT No specific finding. Cortical atrophy with ventriculomegaly at late stage Brain MRI Asymmetric cortical high signal lesion on Pulvinar sign in more than 75% diffusion weighted MR images High signal lesion on both basal ganglia, caudate nucleus and thalamus Cortical atrophy and enlarged ventricles PRNP 129 codon genotype Relatively high rate of methioine homozygotes Methionine homozygotes in all tested vcjd cases Florid plaques in pathology Rare or absent Present in large numbers Immunohistochemistry Variable accumulation of protease-resistant Marked accumulation of proteaseprion protein resistant prion protein Presence of prion in lymphoid tissue Not readily detected Readily detected Tonsil biopsy as a diagnostic tool Not beneficial Highly recommended 1126

Human Prion Diseases Figure 1. Relationship of animal and human prion diseases. 1127

Kim SY Cheong HK An SS 1128

Human Prion Diseases 1129

Kim SY Cheong HK An SS 1130

Human Prion Diseases Figure 2. Diffusion weighted images (lower line) of sporadic Creutzfeldt-Jakob disease show asymmetrical high signal intensities along the gyrus, which were not so clear in T2W images (upper line). 1131

Kim SY Cheong HK An SS Figure 3. Periodic sharp waves at about 1.5/sec; diffuse, bilateral, slightly asymmetrical: and maximal over the anterior portions of the brain. 1132

Human Prion Diseases 1133

Kim SY Cheong HK An SS Figure 4. A Pulvinar sign (arrows) of variant Creutzfeldt-Jakob disease (MR flair image) (from internet). 1134

Human Prion Diseases 11. Kong Q, Huang S, Zou W, Vanegas D, Wang M, Wu D. Chronic wasting disease of elk: transmissibility to humans examined by transgenic mouse models. J Neurosci 2005; 25: 7944-7949. 12. Marsh RF, Kincaid AE, Bessen RA, Bartz JC, Interspecies transmission of chronic wasting disease prions to squirrel monkeys (Saimiri sciureus). J Virol 2005; 79: 13794-13796. 13. Global surveillance, diagnosis and therapy of human trans- 1135

Kim SY Cheong HK An SS missible spongiform encephalopathies: report of a WHO consultation Geneva, Switzerland, 9-11 February 1998. 14. Cosseddu GM, Agrimi U, Pinto J, Schuddel AA. Advances in scrapie research. Rev Sci Tech 2007; 26: 657-668. 15. Conti S, Masocco M, Toccaceli V, Vichi M, Ladogana A, Almonti S, Puopolo M, Pocchiari M, Mortality from human transmissible spongiform encephalopathies: a record linkage study, Neuropidemiology 2005; 24: 214-220. 16. Kristiansen M, Deriziotis P, Dimcheff DE, Jackson GS, Ovaa H, Naumann H, Clarke AR, van Leeuwen FWB, Menendez- Benito V, Dantuma NP, Portis JL, Collinge J, Tabrizi SJ. Disease-associated prion protein oligomers inhibit the 26S porteasome. Molecular Cell 2007; 26: 175-187. 17. Gambetti P, Dong Z, Yuan J, Xiao X, Zheng M, Alxhekhlee A, Castellani R, Cohen M, Barria M, Gonzalez-Romero D, Belay ED, Schonberger LB, Marder K, Harris C, Burke JR, Montine T, Wisniewski T, Dickson DW, Soto C, Hulette CM, Mastrianni JA, Kong Q, Zou WQ. A Novel human disease with abnormal prion protein sensitive to protease. Ann Neurol 2008; 63: 697-708. 18. Prusiner SB. Novel Proteinaceous Infectious Particles Cause Scrapie. Science 1982; 216: 136-144. 19. Roland Riek, Simone Hornemann, Gerhard Wider, Martin Billeter, Rudi Glockshuber & Kurt Wüthrich, NMR structure of the mouse prion protein domain PrP(121~231). Nature 1996; 382: 180-182. 10. Bernoulli C, Siegfried J, Baumgartner G. Danger of accidental person-to-person transmission of Creutzfeldt-Jacob disease by surgery. Lancet 1977; 1: 478-479. 11. Prusiner SB. Prions. Proc Natl Acad Sci 1998; 95: 13363-13383. 12. Chsea R, Piccardo P, Quaglio E. Molecular distinction between pathogenic and infectious properties of the prion protein. J Virol 2003; 77: 7611-7622. 13. Eric D. Ross, Herman K. Edskes, Michael J. Terry, and Reed B. Wickner, Primary sequence independence for prion formation. PNAS 2005; 102: 12825-12830. 14. Johnson RT, Gibbs CJ Jr. Creutzfeldt-Jakob disease and related transmissible spongiform encephalopathies. N Engl J Med 1998; 339: 1994-2004. 15. Zou W-Q, Capellari S, Parchi P, Sy M-S, Gambetti P, Chen SG. Identification of novel proteinase K-resistant C-terminal fragments of PrP in Creutzfeldt-Jakob Disease. J Biol Chem 2003; 278: 40429-40436. 16. Parkin ET, Watt NT, Hussain I, Eckman EA, Eckman CB, Manson JC, Baybutt HN, Turner AJ, Hooper NM. Cellular prion protein regulates beta-secretase cleavage of the Alzheimer s amyloid precursor protein. Proc Natl Acad Sci USA 2007; 104: 11062-11067. 17. Saborio GP, Permanne B, and Soto C. Sensitive detection of pathological prion protein by cyclic amplification of protein misfolding. Nature 2001; 411: 810-813. 18. Wells GAH, Scott AC, Johnson CT. A novel progressive spongiform encephalopathy in cattle. Vet Rec 1987; 121: 419-420. 19. Collins SJ, Lawson VA, Masters CL. Transmissible spongiform encephalopathies. Lancet 2004; 363: 51-61. 20. Bruce ME, Will RG, Ironside JW. Transmissions to mice indicate that new variant CJD is caused by the BSE agent. Nature 1997; 389: 498-501. 21. Scott MR, Perezt D, Hguyen HO, Dearmond SJ, Prusiner SB. Transmissions barriers for bovine, ovine and human prions in transgenic mice. J Virol 2005; 79: 5259-5271. 22. Brown P, Will RG, Bradley R, Asher DM, Detwiler L. Bovine spongiform encephalopathy and Creutzfeldt-Jakob disease: background, evolution, and current concerns. Emerg Infect Dis 2001; 7: 6-16. 23. Williams ES, Young S. Chronic wasting disease of captive mule deer: a spongiform encephalopathy. J Wildlife Dis 1980; 16: 89-98. 24. Belay ED, Maddox RA, Williams ES, Miller MW, Gambetti P, Schonberger LB. Chronic wasting disease and potential transmission to humans. Emerg Infect Dis 2004; 10: 977-984. 25. Mawhinney S, Pape WJ, Forster JE, Anderson CA, Bosque P, Miller MW. Human prion disease and relative risk associated with chronic wasting disease. Emerg Infect Dis 2006; 12: 1527-1535. 26. Sohn HJ, Kim JH, Choi KS, Nah JJ, Joo YS, Jean YH, Ahn SW, Kim OK, Kim DY, and Balachandran A. A case of chronic wasting disease in an elk imported to Korea from Canada. J Vet Med Sci 2002; 64: 855-858. 27. Classification of sporadic Creutzfeldt-Jakob disease revisited. Cali I, Castellani R, Yuan J, Al-Shekhlee A, Cohen ML, Xiao X, MoleresFJ, Parchi P, Zou WQ and Gambetti P. Brain 2006; 129: 2266-2277. 28. Cheong HK, et al. Epidemiological study on the establishment of surveillance system on Creutzfeldt-Jakob disease. Ministry of Health and Welfare. 2003 May. Report No. 01-PJ1-PG3-21900-0010. (Korean) 29. Korea Centers for Disease Control and Prevention. Reported cases of Creutzfelt-Jacob Disease by year. [cited 2008 July 10] Available from:http://sentinel.cdc.go.kr/cro/main/status.asp. 30. Zigas V. Laughing Death, the untold story of Kuru. New Je rsey; Humana Press, 1990: 197-228. 31. Sikorska B, Liberski PP, Sobow T, Budka H, Ironside JW, Ultrastructural study of florid plaques in variant Creutzfeldt- Jakob disease: a comparison with amyloid plaques in kuru, sporadic Creutzfeldt-Jakob disease and Gerstmann-Sträussler-Scheinker disease, Neuropathol Appl Neurobiol 2008; May 30 (online only). 32. Ladogana A, Puopolo M, Croes EA, Budka H, Jarius C, Collins S, Klug GM, Sutcliffe T, Giulivi A, Alperovitch A, Delasnerie- 1136

Human Prion Diseases Laupretre N, Brandel JP, Poser S, Kretzschmar H, Rietveld I, Mitrova E, Cuesta Jde P, Martinez-Martin P, Glatzel M, Aguzzi A, Knight R, Ward H, Pocchiari M, van Duijn CM, Will RG, Zerr I. Mortality from Creutzfeldt-Jakob disease and relaed disorders in Europe, Australia, and Canada. Neurology 2005; 64: 1586-1591. 33. EUROCJD. Total cases of CJD: definite and probable cases: sporadic, familial/genetic, vcjd and iatrogenic deaths. http://www.eurocjd.ed.ac.uk/totalcasesneuro.htm viewed on July 10, 2008. 34. National CJD Surveillance Unit. Fifteenth Annual Report 2006: Creutzfeldt-Jakob disease surveillance in the UK. Edinburgh, National CJD Surveillance Unit, 2007. 35. National Prion Disease Pathology Surveillance Center (NPDPSC). National Prion Disease Pathology Surveillance Center cases examined. http://www.cjdsurveillance.com / resources-casereport.html viewed on July 10, 2008 36. Brown P, Gibbs CJ Jr, Rodgers-Johnson P, Asher DM, Sulima MP, Bacote A, Goldfarb LG, Gajdusek DC. Human spongiform encephalopathy: the National Institutes of Health Series of 300 cases of experimentally transmitted disease. Ann Neurol 1994; 35: 513-529 37. Bernoulli CC, Masters CL, Gajdusek DC, Gibbs CJ Jr, Harris JO. Early clinical features of Creutzfeldt-Jakob disease (subacute spongiform encephalopathy) In: Prusiner SB, Hadlow WJ, eds. Slow transmissible diseases of the nervous system, vol 1: clinical, epidemiological, genetic and pa-thological aspects of the spongiform encephalopathies. New York: Academic Press, 1979: 229-241. 38. Brown P, Gibbs CJ Jr, Rodgers-Johnson P, Asher DM, Sulima MP, Bacote A, Goldfarb LG, Gajdusek DC. Human spongiform encephalopathy: the National Institutes of Health Series of 300 cases of experimentally transmitted disease. Ann Neurol 994; 35: 513-529. 39. Uro-Coste E, Cassard H, Simon S, Lugan S, Bilheude JM, Perret-Liaudet A, Ironside JW, Haik S, Basset-Leobon C, Lacroux C, Peioch K, Streichenberger N, Langeveld J, Head MW, Grassi J, Hauw JJ, Schelcher F, Delisle MB, Andreoletti O. Beyond PrPres type 1/type 2 dichotomy in Creutzfeldt- Jakob disease. PLoS Pathogens 2008; 4(e 1000029): 1-9. (online onle; www.plospathogens.org) 40. Ludewigs H, Zuber C, Vana K, Nikles D, Zerr I, Weiss S. Therapeutic approaches for prion disorders. Expert Rev Anti Infect Ther 2007; 5 : 613-630. 41. Capellari S, Cardone F, Notari S, SchininàME, Maras B, Sità D, Baruzzi A, Pocchiari M, Parchi P. Creutzfeldt-Jakob disease associated with the R208H mutation in the prion protein gene. Neurology 2005; 64: 905-907. 42. Mastrianni JA. Prion diseases and dementia. In: Power C, Johnson RT, eds. Emerging neurological infections. Boca Raton: Taylor and Francis, 2005: 77-113. 43. 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