BDNF insufficiency exacerbates ALS progression

Cell Reports Medicine 2026 Open access

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Authors: Yihua Xu, Ji He, Shudan Wang, Yijun Ge, Yichang Jia, Wei Guo, Lv Chen, Xinmeng Yao, Yingying Mao, Fan Mei, Dongsheng Fan, Peng Yuan
+1 more Bai Lu
DOI: 10.1016/j.xcrm.2026.102758
Cited by: 1
References: 54

Abstract

, we demonstrate that BDNF haploinsufficiency leads to shortened lifespan, accelerated motor dysfunctions, and exacerbated motor neuron death. Importantly, activation of the BDNF receptor TrkB by an agonistic antibody effectively rescues these ALS-associated phenotypes. In additional ALS mouse models, TrkB activation antibody also shows superior therapeutic effects compared to current ALS medication riluzole. Our data indicate that insufficient BDNF could be a crucial contributing factor for ALS progression, and activation of BDNF-TrkB pathway may represent a promising therapeutic strategy against ALS.

Fields of Study & Subjects

Amyotrophic Lateral Sclerosis Research Nerve injury and regeneration Neurogenetic and Muscular Disorders Research

Publication details

Journal / source
Cell Reports Medicine
Publisher
Elsevier BV
Volume
7
Issue
5
Pages
102758–102758
Publication date
20 April 2026
Language
EN