Tsuboi Y, Doh-ura K, Yamada T. 2009. Continuous intraventricular infusion of pentosan polysulfate: clinical trial against prion diseases (Neuropathology 29:632-636)

This is the Japanese report on continuous infusion of pentosan polysulfate (PPS) into the brain ventricles of people with prion disease. It describes a series of 11 patients treated at Fukuoka University from 2004, covering sporadic, genetic and iatrogenic forms. The authors found no clear clinical improvement, a mean survival of 24.2 months after starting treatment, and frequent subdural fluid collections, and they could only suggest that long-term infusion might have lengthened survival in some patients. Reviews place it among the uncontrolled studies that gave no proof of benefit. Information only, not medical advice. Aggregate findings only.

Key facts

  • Citation verified against PubMed: Neuropathology, October 2009, volume 29 issue 5, pages 632 to 636, PMID 19788637, DOI 10.1111/j.1440-1789.2009.01058.x, authors Yoshio Tsuboi, Katsumi Doh-ura and Tatsuo Yamada.
  • The series covers 11 patients treated with intraventricular PPS at Fukuoka University from 2004, drawn from sporadic, genetic and iatrogenic prion disease, with sporadic cases the largest group.
  • Mean survival after starting treatment was 24.2 months, with a range of 4 to 49 months; seven patients died of sepsis and pneumonia.
  • Most patients developed subdural effusion of varying degree on CT and the abstract reports no other adverse effects; a review counted catheter-related complications in 10 of the 11 patients.
  • The authors reported no apparent clinical improvement and said only that long-term PPS may have extended survival in some patients.
  • The clinical work followed mouse experiments in which intraventricular PPS delayed disease when started early or late, but with effects confined to the infused side of the brain and with brain bleeding at higher doses.
  • A 2011 review noted that survival was longer than in historical controls without any change in the underlying disease, and called for standardised prospective studies with post-mortem examination.
  • A 2013 review lists the paper among the human PPS reports, states that small numbers, absent controls and short survival make rigorous trials hard, but misdescribes the series as four patients, which conflicts with the abstract.

Timeline

  1. 2004Fukuoka University begins infusing PPS in patients with prion disease.
  2. 2004Doh-ura and colleagues publish the mouse study of intraventricular PPS in the Journal of Virology.
  3. 2009The 11-patient series is published in Neuropathology.
  4. 2010The Appleby and Lyketsos review summarising the Japanese series appears online.

Last checked 1 November 2026

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