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@ARTICLE{Chen:278999,
author = {Chen, Charles D and Franklin, Erin E and Li, Yan and
Joseph-Mathurin, Nelly and Burns, Aime L and Hobbs, Diana A
and McCullough, Austin A and Schultz, Stephanie A and Xiong,
Chengjie and Wang, Guoqiao and Masellis, Mario and Hsiung,
Ging-Yuek Robin and Gauthier, Serge and Berman, Sarah B and
Roberson, Erik D and Honig, Lawrence S and Clarnette, Roger
and Ringman, John M and Galvin, James E and Brooks, William
and Suzuki, Kazushi and Black, Sandra and Levin, Johannes
and Aggarwal, Neelum T and Jucker, Mathias and Frosch,
Matthew P and Kofler, Julia K and White, Charles and Keene,
C Dirk and Chen, Jie and Daniels, Alisha and Gordon, Brian A
and Ibanez, Laura and Karch, Celeste M and Llibre-Guerra,
Jorge and McDade, Eric and Morris, John C and Supnet-Bell,
Charlene and Allegri, Ricardo F and Lee, Jae-Hong and Day,
Gregory S and Lopera, Francisco and Roh, Jee Hoon and
Schofield, Peter R and Mills, Susan and Benzinger, Tammie L
S and Bateman, Randall J and Perrin, Richard J},
collaboration = {Team, DIAN-TU Study and Team, DIAN-Obs Study},
othercontributors = {Bateman, Randall and Daniels, Alisha J and Courtney, Laura
and Llibre-Guerra, Jorge J and Xiong, Chengie and Xu, Xiong
and Lu, Ruijin and Gremminger, Emily and Jerome, Gina and
Herries, Elizabeth and Stauber, Jennifer and Baker, Bryce
and Minton, Matthew and Cruchaga, Carlos and Goate, Alison M
and Renton, Alan E and Picarello, Danielle M and Benzinger,
Tammie and Hornbeck, Russell and Hassenstab, Jason and
Smith, Jennifer and Stout, Sarah and Aschenbrenner, Andrew J
and Marsh, Jacob and Holtzman, David M and Barthelemy,
Nicolas and Xu, Jinbin and Noble, James M and Ikonomovic,
Snezana and Nadkarni, Neelesh K and Graff-Radford, Neill R
and Farlow, Martin and Chhatwal, Jasmeer P and Ikeuchi,
Takeshi and Kasuga, Kensaku and Niimi, Yoshiki and Huey,
Edward D and Salloway, Stephen and Brooks, William S and
Bechara, Jacob A and Martins, Ralph and Fox, Nick C and
Cash, David M and Ryan, Natalie S and Laske, Christoph and
Hofmann, Anna and Kuder-Buletta, Elke and Gräber-Sultan,
Susanne and Obermueller, Ulrike and Roedenbeck, Yvonne and
Vӧglein, Jonathan and Sanchez-Valle, Raquel and Rosa-Neto,
Pedro and Mendez, Patricio Chrem and Surace, Ezequiel and
Vazquez, Silvia and Leon, Yudy Milena and Ramirez, Laura and
Aguillon, David and Levey, Allan I and Johnson, Erik C B and
Seyfried, Nicholas T and Ringman, John and Fagan, Anne M and
Mori, Hiroshi},
title = {{I}mmunohistochemical evaluation of a trial of gantenerumab
or solanezumab in dominantly inherited {A}lzheimer disease.},
journal = {Acta neuropathologica},
volume = {149},
number = {1},
issn = {0001-6322},
address = {Heidelberg},
publisher = {Springer},
reportid = {DZNE-2025-00663},
pages = {57},
year = {2025},
abstract = {Clinical trials of anti-amyloid-β (Aβ) monoclonal
antibodies in Alzheimer disease (AD) infer target engagement
from Aβ positron emission tomography (PET) and/or fluid
biomarkers such as cerebrospinal fluid (CSF) Aβ42/40.
However, these biomarkers measure brain Aβ deposits
indirectly and/or incompletely. In contrast, neuropathologic
assessments allow direct investigation of treatment effects
on brain Aβ deposits-and on potentially myriad 'downstream'
pathologic features. From a clinical trial of anti-Aβ
monoclonal antibodies in dominantly inherited AD (DIAD), in
the largest study of its kind, we measured
immunohistochemistry area fractions (AFs) for Aβ deposits
(10D5), tauopathy (PHF1), microgliosis (IBA1), and
astrocytosis (GFAP) in 10 brain regions from 10 trial
cases-gantenerumab (n = 4), solanezumab (n = 4), placebo/no
treatment (n = 2)-and 10 DIAD observational study cases.
Strikingly, in proportion to total drug received, Aβ
deposit AFs were significantly lower in the gantenerumab arm
versus controls in almost all areas examined, including
frontal, temporal, parietal, and occipital cortices,
anterior cingulate, hippocampus, caudate, putamen, thalamus,
and cerebellar gray matter; only posterior cingulate and
cerebellar white matter comparisons were non-significant. In
contrast, AFs of tauopathy, microgliosis, and astrocytosis
showed no differences across groups. Our results demonstrate
with direct histologic evidence that gantenerumab treatment
in DIAD can reduce parenchymal Aβ deposits throughout the
brain in a dose-dependent manner, suggesting that more
complete removal may be possible with earlier and more
aggressive treatment regimens. Although AFs of tauopathy,
microgliosis, and astrocytosis showed no clear response to
partial Aβ removal in this limited autopsy cohort, future
examination of these cases with more sensitive techniques
(e.g., mass spectrometry) may reveal more subtle
'downstream' effects.},
keywords = {Humans / Antibodies, Monoclonal, Humanized: therapeutic use
/ Alzheimer Disease: drug therapy / Alzheimer Disease:
pathology / Alzheimer Disease: genetics / Alzheimer Disease:
metabolism / Male / Female / Amyloid beta-Peptides:
metabolism / Aged / Brain: pathology / Brain: drug effects /
Brain: metabolism / Middle Aged / Immunohistochemistry /
Aged, 80 and over / Alzheimer disease (Other) /
Anti-amyloid-β monoclonal antibodies (Other) / CSF (Other)
/ Clinical trial (Other) / Digital pathology (Other) / PiB
PET (Other) / Antibodies, Monoclonal, Humanized (NLM
Chemicals) / Amyloid beta-Peptides (NLM Chemicals) /
gantenerumab (NLM Chemicals) / solanezumab (NLM Chemicals)},
cin = {AG Levin / Clinical Research (Munich) / AG Jucker},
ddc = {610},
cid = {I:(DE-2719)1111016 / I:(DE-2719)1111015 /
I:(DE-2719)1210001},
pnm = {353 - Clinical and Health Care Research (POF4-353) / 352 -
Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-353 / G:(DE-HGF)POF4-352},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40459787},
doi = {10.1007/s00401-025-02890-7},
url = {https://pub.dzne.de/record/278999},
}