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@ARTICLE{McDade:163359,
author = {McDade, Eric and Voytyuk, Iryna and Aisen, Paul and
Bateman, Randall J and Carrillo, Maria C and De Strooper,
Bart and Haass, Christian and Reiman, Eric M and Sperling,
Reisa and Tariot, Pierre N and Yan, Riqiang and Masters,
Colin L and Vassar, Robert and Lichtenthaler, Stefan F},
title = {{T}he case for low-level {BACE}1 inhibition for the
prevention of {A}lzheimer disease.},
journal = {Nature reviews / Neurology},
volume = {17},
number = {11},
issn = {1759-4766},
address = {London},
publisher = {Macmillan Publishers Limited, part of Springer Nature},
reportid = {DZNE-2022-00122},
pages = {703 - 714},
year = {2021},
abstract = {Alzheimer disease (AD) is the most common cause of dementia
in older individuals (>65 years) and has a long
presymptomatic phase. Preventive therapies for AD are not
yet available, and potential disease-modifying therapies
targeting amyloid-β plaques in symptomatic stages of AD
have only just been approved in the United States.
Small-molecule inhibitors of β-site amyloid precursor
protein (APP)-cleaving enzyme 1 (BACE1; also known as
β-secretase 1) reduce the production of amyloid-β peptide
and are among the most advanced drug candidates for AD.
However, to date all phase II and phase III clinical trials
of BACE inhibitors were either concluded without benefit or
discontinued owing to futility or the occurrence of adverse
effects. Adverse effects included early, mild cognitive
impairment that was associated with all but one inhibitor;
preliminary results suggest that the cognitive effects are
non-progressive and reversible. These discontinuations have
raised questions regarding the suitability of BACE1 as a
drug target for AD. In this Perspective, we discuss the
status of BACE inhibitors and suggest ways in which the
results of the discontinued trials can inform the
development of future clinical trials of BACE inhibitors and
related secretase modulators as preventative therapies. We
also propose a series of experiments that should be
performed to inform 'go-no-go' decisions in future trials
with BACE inhibitors and consider the possibility that low
levels of BACE1 inhibition could avoid adverse effects while
achieving efficacy for AD prevention.},
subtyp = {Review Article},
keywords = {Aged / Aged, 80 and over / Alzheimer Disease: prevention
$\&$ control / Amyloid Precursor Protein Secretases:
antagonists $\&$ inhibitors / Amyloid Precursor Protein
Secretases: genetics / Amyloid beta-Protein Precursor:
metabolism / Aspartic Acid Endopeptidases: antagonists $\&$
inhibitors / Aspartic Acid Endopeptidases: genetics /
Clinical Trials, Phase II as Topic / Clinical Trials, Phase
III as Topic / Enzyme Inhibitors: therapeutic use / Female /
Humans / Male / Middle Aged / Plaque, Amyloid: prevention
$\&$ control / Research Design / APP protein, human (NLM
Chemicals) / Amyloid beta-Protein Precursor (NLM Chemicals)
/ Enzyme Inhibitors (NLM Chemicals) / Amyloid Precursor
Protein Secretases (NLM Chemicals) / Aspartic Acid
Endopeptidases (NLM Chemicals) / BACE1 protein, human (NLM
Chemicals)},
cin = {AG Haass / AG Lichtenthaler},
ddc = {610},
cid = {I:(DE-2719)1110007 / I:(DE-2719)1110006},
pnm = {352 - Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:34548654},
doi = {10.1038/s41582-021-00545-1},
url = {https://pub.dzne.de/record/163359},
}