TY - JOUR
T1 - Experimental treatments for human transmissible spongiform encephalopathies
T2 - Is there a role for pentosan polysulfate?
AU - Rainov, N. G.
AU - Tsuboi, Y.
AU - Krolak-Salmon, P.
AU - Vighetto, A.
AU - Doh-ura, K.
N1 - Funding Information:
101. http://www.ctu.mrc.ac.uk/studies/cjd.asp Medical Research Council, Clinical Trials Unit (MRC CTU). PRION-1: Randomised trial of quinacrine in human prion disease.
PY - 2007/5
Y1 - 2007/5
N2 - 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.
AB - 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.
KW - Gerstmann-Straussler-Scheinker syndrome
KW - Pentosan polysulfate
KW - Prion disease
KW - Quinacrine
KW - Sporadic Creutzfeldt-Jakob disease
KW - Transmissible spongiform encephalopathy
KW - Variant Creutzfeldt-Jakob disease
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UR - http://www.scopus.com/inward/citedby.url?scp=34248180644&partnerID=8YFLogxK
U2 - 10.1517/14712598.7.5.713
DO - 10.1517/14712598.7.5.713
M3 - Review article
C2 - 17477808
AN - SCOPUS:34248180644
VL - 7
SP - 713
EP - 726
JO - Expert Opinion on Biological Therapy
JF - Expert Opinion on Biological Therapy
SN - 1471-2598
IS - 5
ER -
