Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease

Abstract Histamine H3 receptor (H3R) antagonists regulate histamine release that modulates neuronal activity and cognitive function. Although H3R is elevated in Alzheimer’s disease (AD) patients, whether H3R antagonists can rescue AD-associated neural impairments and cognitive deficits remains unkno...

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Main Authors: Yang Zou, Linhan Yang, Jiahui Zhu, Jihua Fan, Hanrun Zheng, Xiang Liao, Zhiqi Yang, Kuan Zhang, Hongbo Jia, Arthur Konnerth, Yan-jiang Wang, Chunqing Zhang, Yun Zhang, Sunny C. Li, Xiaowei Chen
Format: Article
Language:English
Published: Nature Publishing Group 2025-04-01
Series:Translational Psychiatry
Online Access:https://doi.org/10.1038/s41398-025-03358-8
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author Yang Zou
Linhan Yang
Jiahui Zhu
Jihua Fan
Hanrun Zheng
Xiang Liao
Zhiqi Yang
Kuan Zhang
Hongbo Jia
Arthur Konnerth
Yan-jiang Wang
Chunqing Zhang
Yun Zhang
Sunny C. Li
Xiaowei Chen
author_facet Yang Zou
Linhan Yang
Jiahui Zhu
Jihua Fan
Hanrun Zheng
Xiang Liao
Zhiqi Yang
Kuan Zhang
Hongbo Jia
Arthur Konnerth
Yan-jiang Wang
Chunqing Zhang
Yun Zhang
Sunny C. Li
Xiaowei Chen
author_sort Yang Zou
collection DOAJ
description Abstract Histamine H3 receptor (H3R) antagonists regulate histamine release that modulates neuronal activity and cognitive function. Although H3R is elevated in Alzheimer’s disease (AD) patients, whether H3R antagonists can rescue AD-associated neural impairments and cognitive deficits remains unknown. Pitolisant is a clinically approved H3R antagonist/inverse agonist that treats narcolepsy. Here, we find that pitolisant reverses AD-like pathophysiology and cognitive impairments in an AD mouse model. Behavioral assays and in vivo wide-field Ca2+ imaging revealed that recognition memory, learning flexibility, and slow-wave impairment were all improved following the 15-day pitolisant treatment. Improved recognition memory was tightly correlated with slow-wave coherence, suggesting slow waves serve as a biomarker for treatment response and for AD drug screening. Furthermore, pitolisant reduced amyloid-β deposition and dystrophic neurites surrounding plaques, and enhanced neuronal lysosomal activity, inhibiting which blocked cognitive and slow-wave restoration. Our findings identify pitolisant as a potential therapeutic agent for AD treatments.
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series Translational Psychiatry
spelling doaj-art-2a19e58089024101910a66c8d359ad4c2025-08-20T02:08:11ZengNature Publishing GroupTranslational Psychiatry2158-31882025-04-0115111510.1038/s41398-025-03358-8Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s diseaseYang Zou0Linhan Yang1Jiahui Zhu2Jihua Fan3Hanrun Zheng4Xiang Liao5Zhiqi Yang6Kuan Zhang7Hongbo Jia8Arthur Konnerth9Yan-jiang Wang10Chunqing Zhang11Yun Zhang12Sunny C. Li13Xiaowei Chen14Guangxi Key Laboratory of Special Biomedicine/Advanced Institute for Brain and Intelligence, School of Medicine, Guangxi UniversityGuangxi Key Laboratory of Special Biomedicine/Advanced Institute for Brain and Intelligence, School of Medicine, Guangxi UniversityGuangxi Key Laboratory of Special Biomedicine/Advanced Institute for Brain and Intelligence, School of Medicine, Guangxi UniversityGuangxi Key Laboratory of Special Biomedicine/Advanced Institute for Brain and Intelligence, School of Medicine, Guangxi UniversityState Key Laboratory of Structural Chemistry, Fujian Institute of Research on the Structure of Matter, Chinese Academy of SciencesCenter for Neurointelligence, School of Medicine, Chongqing UniversityBrain Research Center and State Key Laboratory of Trauma and Chemical Poisoning, Third Military Medical UniversityBrain Research Center and State Key Laboratory of Trauma and Chemical Poisoning, Third Military Medical UniversityGuangxi Key Laboratory of Special Biomedicine/Advanced Institute for Brain and Intelligence, School of Medicine, Guangxi UniversityInstitute of Neuroscience and Munich Cluster for Systems Neurology, Technical University MunichInstitute of Brain and Intelligence, Third Military Medical UniversityInstitute of Brain and Intelligence, Third Military Medical UniversityState Key Laboratory of Structural Chemistry, Fujian Institute of Research on the Structure of Matter, Chinese Academy of SciencesLFC Laboratory and Chongqing Institute for Brain and Intelligence, Guangyang Bay LaboratoryBrain Research Center and State Key Laboratory of Trauma and Chemical Poisoning, Third Military Medical UniversityAbstract Histamine H3 receptor (H3R) antagonists regulate histamine release that modulates neuronal activity and cognitive function. Although H3R is elevated in Alzheimer’s disease (AD) patients, whether H3R antagonists can rescue AD-associated neural impairments and cognitive deficits remains unknown. Pitolisant is a clinically approved H3R antagonist/inverse agonist that treats narcolepsy. Here, we find that pitolisant reverses AD-like pathophysiology and cognitive impairments in an AD mouse model. Behavioral assays and in vivo wide-field Ca2+ imaging revealed that recognition memory, learning flexibility, and slow-wave impairment were all improved following the 15-day pitolisant treatment. Improved recognition memory was tightly correlated with slow-wave coherence, suggesting slow waves serve as a biomarker for treatment response and for AD drug screening. Furthermore, pitolisant reduced amyloid-β deposition and dystrophic neurites surrounding plaques, and enhanced neuronal lysosomal activity, inhibiting which blocked cognitive and slow-wave restoration. Our findings identify pitolisant as a potential therapeutic agent for AD treatments.https://doi.org/10.1038/s41398-025-03358-8
spellingShingle Yang Zou
Linhan Yang
Jiahui Zhu
Jihua Fan
Hanrun Zheng
Xiang Liao
Zhiqi Yang
Kuan Zhang
Hongbo Jia
Arthur Konnerth
Yan-jiang Wang
Chunqing Zhang
Yun Zhang
Sunny C. Li
Xiaowei Chen
Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease
Translational Psychiatry
title Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease
title_full Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease
title_fullStr Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease
title_full_unstemmed Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease
title_short Pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of Alzheimer’s disease
title_sort pitolisant alleviates brain network dysfunction and cognitive deficits in a mouse model of alzheimer s disease
url https://doi.org/10.1038/s41398-025-03358-8
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