TY - JOUR
T1 - Clinical trials of prion disease therapeutics
AU - Forloni, Gianluigi
AU - Roiter, Ignazio
AU - Tagliavini, Fabrizio
N1 - Copyright © 2019. Published by Elsevier Ltd.
PY - 2019/2
Y1 - 2019/2
N2 - Prion-related encephalopathies or transmissible spongiform encephalopathies (TSEs) are a group of rare progressive neurodegenerative disorders that are invariably fatal with often only six months elapsing from diagnosis to patient death. This makes the development of effective therapeutic strategies challenging. Nonetheless, compounds have been identified in animal models of TSE that prolong survival and, in some instances, eradicate the disease. These have been tested in the clinic, although with modest or negative outcomes. While little progress has been made over the last decade, new findings that include the ability to identify prion aggregates at low levels in biological fluids and cells may lead to the development of early-stage biomarkers for TSE. An increased focus on immunotherapeutic approaches to TSE may result in the development of novel preventive approaches for TSE.
AB - Prion-related encephalopathies or transmissible spongiform encephalopathies (TSEs) are a group of rare progressive neurodegenerative disorders that are invariably fatal with often only six months elapsing from diagnosis to patient death. This makes the development of effective therapeutic strategies challenging. Nonetheless, compounds have been identified in animal models of TSE that prolong survival and, in some instances, eradicate the disease. These have been tested in the clinic, although with modest or negative outcomes. While little progress has been made over the last decade, new findings that include the ability to identify prion aggregates at low levels in biological fluids and cells may lead to the development of early-stage biomarkers for TSE. An increased focus on immunotherapeutic approaches to TSE may result in the development of novel preventive approaches for TSE.
U2 - 10.1016/j.coph.2019.04.019
DO - 10.1016/j.coph.2019.04.019
M3 - Review article
C2 - 31108459
VL - 44
SP - 53
EP - 60
JO - Current Opinion in Pharmacology
JF - Current Opinion in Pharmacology
SN - 1471-4892
ER -