Continuous intraventricular infusion of pentosan polysulfate: clinical trial against prion diseases

Neuropathology. 2009 Oct;29(5):632-6. doi: 10.1111/j.1440-1789.2009.01058.x.


Prion diseases are progressive neurological disorders due to abnormal prion protein (PrP(Sc)) deposition in the central nervous system. At present, there is no effective treatment available for any form of prion disease. Pentosan polysulfate (PPS) has been shown to prolong significantly the incubation period in mice with PrP(Sc) infection when administered to the cerebral ventricles in preclinical trials. In human studies conducted in European countries and Japan, intraventricular PPS was administered to patients with different forms of prion disease and was well tolerated. We report 11 patients with prion disease treated with intraventricular PPS at Fukuoka University from 2004. Cases included three familial CJD (two with V180I mutation, one GSS with P102L mutation), two iatrogenic CJD, and six sporadic CJD cases. At present, average survival period after treatment was 24.2 months (range, 4-49). Seven cases died of sepsis and pneumonia. Subdural effusion with various degrees was seen on CT scan in most cases. Except for these, adverse effects did not occur in the treatment period. Although our preliminary study of the new treatment with PPS by continuous intraventricular infusion showed no apparent improvement of clinical features in patients with prion disease, the possibility of extended survival in some patients receiving long-term PPS was suggested.

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Anti-Infective Agents / administration & dosage
  • Anti-Infective Agents / therapeutic use*
  • Brain / diagnostic imaging
  • Brain / surgery
  • Catheterization
  • Female
  • Follow-Up Studies
  • Humans
  • Infusion Pumps, Implantable / adverse effects
  • Infusions, Parenteral / adverse effects
  • Male
  • Middle Aged
  • Pentosan Sulfuric Polyester / administration & dosage
  • Pentosan Sulfuric Polyester / therapeutic use*
  • Pneumonia / etiology
  • Pneumonia / mortality
  • Prion Diseases / diagnostic imaging
  • Prion Diseases / drug therapy*
  • Prion Diseases / surgery
  • Prospective Studies
  • Sepsis / etiology
  • Sepsis / mortality
  • Time Factors
  • Tomography, X-Ray Computed
  • Treatment Outcome


  • Anti-Infective Agents
  • Pentosan Sulfuric Polyester