NR AWMJ
AU Rainov,N.G.; Doh-ura,K.; Tsuboi,Y.; Krolak-Salmon,P.; Heidecke,V.
TI Experimental treatments for human prion diseases
QU International Conference - Prion 2006: Strategies, advances and trends towards protection of society - 3.10.-6.10.2006, Torino, Italy, Lingotto Conference Centre - Poster sessions THE-14
PT Konferenz-Poster
AB Human prion diseases, also known as transmissible spongiform encephalopathies (TSE), are caused by accumulation of the abnormal isoform of the prion protein in the central nervous system. At present there is no proven specific or effective treatment available for any forms of TSE. Some oral agents, such as quinacrine or flupirtine, have shown promising results in vitro and are currently being investigated in clinical trials. Pentosan polysulphate (PPS), a polysulphonated polysaccharide, has been shown to significantly prolong the incubation period when administered to the cerebral ventricles in preclinical trials with rodent TSE models. Cerebroventricular administration of PPS has been carried out in patients with different forms of TSE and was shown to be well tolerated in doses up to 220 µg/kg/day. Currently, a total of 22 patients have received PPS for periods of time ranging from 2 to 39 months. Proof of efficacy has been difficult because of the lack of specific and objective criteria for measurement of response to treatment. However, preliminary clinical experience demonstrated extended survival in some patients receiving long-term PPS. A young man with vCJD is surviving for a total of 5 years after initial symptoms, and for 39 months after start of PPS infusion. Other vCJD patients have also shown overall survival times well above the mean values expected with their disease. Further prospective investigation of the cerebroventricular administration of PPS is essential to clarify the observed trends and to ascertain possible clinical benefits in patients with TSE.
AD N.G. Rainov, V. Heidecke: Department of Neurosurgery, Zentralklinikum Augsburg, Augsburg, Germany, and Department of Neurosurgery, Martin-Luther-University Halle-Wittenberg, Halle, Germany; K. Doh-ura: Department of Prion Research, Tohoku University Graduate School of Medicine, Sendai, Japan; Y. Tsuboi: Department of Neurology, Fukuoka University School of Medicine, Fukuoka, Japan; P. Krolak-Salmon: Department of Neurology, Hopital Neurologique, Lyon, France
SP englisch
PO Italien