Experimental treatments for human transmissible spongiform encephalopathies: Is there a role for pentosan polysulfate?

N. G. Rainov, Y. Tsuboi, P. Krolak-Salmon, A. Vighetto, K. Doh-ura

Research output: Contribution to journalReview articlepeer-review

46 Citations (Scopus)


Human transmissible spongiform encephalopathies (TSEs), also known as prion diseases, are caused by the accumulation of an abnormal isoform of the prion protein in the CNS. Creutzfeldt-Jakob disease in its sporadic form is the most frequent type of human TSE. At present, there is no proven specific or effective treatment available for any form of TSE. Pentosan polysulfate (PPS) has been shown to prolong the incubation period when administered to the cerebral ventricles in a rodent TSE model. Cerebroventricular administration of PPS has been carried out in 26 patients with TSEs and has been shown to be well tolerated in doses ≤ 220 μg/kg/day. Proof of efficacy has been difficult because the specific and objective criteria for measurement of response have not been established yet. Preliminary clinical experience confirms extended survival in patients with variant Creutzfeldt-Jakob disease receiving intraventricular PPS; however, it is still not clear if this is due to PPS itself. Further prospective investigations of long-term intraventricular PPS administration are essential for the assessment of its effects.

Original languageEnglish
Pages (from-to)713-726
Number of pages14
JournalExpert Opinion on Biological Therapy
Issue number5
Publication statusPublished - 2007 May


  • Gerstmann-Straussler-Scheinker syndrome
  • Pentosan polysulfate
  • Prion disease
  • Quinacrine
  • Sporadic Creutzfeldt-Jakob disease
  • Transmissible spongiform encephalopathy
  • Variant Creutzfeldt-Jakob disease

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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