Delving into the Latest Updates on GM1 ganglioside with Synapse

Overview

Basic Info

Drug Type

Chemical drugs

Synonyms

Shenjie

Target-

Action-

Mechanism-

Therapeutic Areas

Nervous System DiseasesEndocrinology and Metabolic Disease

Active Indication-

Inactive Indication

Parkinson Disease

Originator Organization

LZ Therapeutics, Inc.

Active Organization-

Inactive Organization

LZ Therapeutics, Inc.

License Organization-

Drug Highest PhasePendingPhase 2

First Approval Date-

Regulation-

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Structure/Sequence

Molecular FormulaC73H131N3NaO31

InChIKeySCNDOMAFNZIBOJ-POYTVKKJSA-N

CAS Registry1694670-82-0

To evaluate the efficacy and safety of GM1 for preventing cognitive impairment related to whole brain radiotherapy in breast cancer patients with brain metastases. And explore the clinical and molecular parameter for predicting severe cognitive impairment induced by WBRT and gaining benefit from GM1.
Primary Endpoint: the change of Hopkins Verbal and Learning Test-Revised Delayed Recall,HVLT-R DR,before and after WBRT Secondary ENDPOINT: the change of Alzheimer's Disease Assessment Scale-Cognitive，ADAS-Cog before and after WBRT;severe cognitive impairment percentage and onset time; Design:204 patients will be randomly assigned to exp.group,102 cases,and 102 cases of control group.

Start Date15 May 2020

Sponsor / Collaborator

Sun Yat-Sen University

100 Clinical Results associated with GM1 ganglioside

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100 Translational Medicine associated with GM1 ganglioside

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100 Patents (Medical) associated with GM1 ganglioside

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547

Literatures (Medical) associated with GM1 ganglioside

01 Jan 2025·Food Research International

Mechanisms by which Ganglioside GM1, a specific type of glycosphingolipid, ameliorates BMAA-induced neurotoxicity in early-life stage of zebrafish embryos

Article

Author: Xue, Changhu ; Wang, Xiaoxu ; Li, Hongyan ; Zhu, Yuhe ; Song, Yu ; Wang, Zhigao ; Yang, Shuaiqi ; Dong, Bo ; Liu, Yanjun ; Xu, Jie ; Li, Yiyang

The neurotoxin β-methylamino-L-alanine (BMAA) produced by cyanobacteria is widely present in foods and dietary supplements, posing a significant threat to human health. Ganglioside GM1 (GM1) has demonstrated potential for treating neurodegenerative diseases; however, its ability to prevent BMAA-induced neurotoxicity remains uncertain. In this study, zebrafish embryos were treated with Ganglioside GM1 to investigate its neuroprotective effects against BMAA exposure and the underlying mechanisms. Exposure to BMAA (400 μM) resulted in increased malformation rate and heart rates in zebrafish embryos at 72 h post-fertilization, along with the decreased survival rates. Conversely, GM1 intervention rescued BMAA-induced movement disorders and brain cell apoptosis, and oxidative stress was alleviated. In addition, GM1 inhibited the neurotoxic effects of BMAA in zebrafish embryos, as indicated by the up-regulation of genes related to neuron development (gpx1a, bdnf, ngfb, and islet-1) and the down-regulations of neurodegeneration-related genes (cdk5, gfap, and nptxr). GM1 treatment restored 261 differentially expressed genes (DEGs) identified through RNA sequencing, with the most enriched DEGs related to the mitogen-activated protein kinase (MAPK) signaling pathway (P < 0.05, 47 genes). GM1 modulated MAPK-targeted gene expression at the mRNA level. These findings suggest that GM1 alleviates BMAA-induced neurotoxicity in the early-life stage of zebrafish embryos. The neuroprotective mechanism may involve the MAPK pathway, offering new insights into lipid signaling for the prevention of neurotoxic hazards to biological health.

19 Mar 2024·Glycobiology

GM1 ganglioside protects against LPS-induced neuroinflammatory and oxidative responses by inhibiting the activation of Akt, TAK1 and NADPH oxidase in MG6 microglial cells

Article

Author: Yeh, Ting-Yin ; Chu, Wen-Jui ; Huang, Yuahn-Sieh

AbstractGM1 is a major brain ganglioside that exerts neurotrophic, neuroprotective and antineuroinflammatory effects. The aim of this study was to obtain insights into the antineuroinflammatory mechanisms of exogenous GM1 in lipopolysaccharide (LPS)-stimulated MG6 mouse transformed microglial cell line. First, we found that GM1 prevented the LPS-induced transformation of microglia into an amoeboid-like shape. GM1 treatment inhibited LPS-induced expression of inducible nitric oxide synthase, cyclooxygenase-2 (COX-2), and proinflammatory cytokines such as TNF-α, IL-1β and IL-6 in MG6 cells. In LPS-treated mice, GM1 also reduced striatal microglia activation and attenuated COX-2 expression. Subsequent mechanistic studies showed that GM1 suppressed LPS-induced nuclear translocation of nuclear factor κB (NF-κB) and activator protein-1 (AP-1), two critical transcription factors responsible for the production of proinflammatory mediators. GM1 exhibited antineuroinflammatory properties by suppressing Akt/NF-κB signaling and the activation of mitogen-activated protein kinases (MAPKs), including p38 MAPK, extracellular signal-regulated kinase 1/2 (ERK1/2) and c-Jun N-terminal kinase (JNK). Furthermore, GM1 suppressed LPS-induced activation of transforming growth factor-β-activated kinase 1 (TAK1) and NADPH oxidase 2 (NOX2), upstream regulators of the IκBα/NF-κB and MAPK/AP-1 signaling pathways. GM1 also inhibited NOX-mediated reactive oxygen species (ROS) production and protected against LPS-induced MG6 cell death, suggesting an antioxidant role of GM1. In conclusion, GM1 exerts both antineuroinflammatory and antioxidative effects by inhibiting Akt, TAK1 and NOX2 activation.

01 Dec 2023·FEBS Open Bio

GM1 ganglioside exerts protective effects against glutamate‐excitotoxicity via its oligosaccharide in wild‐type and amyotrophic lateral sclerosis motor neurons

Article

Author: Aureli, Massimo ; Sonnino, Sandro ; Carsana, Emma Veronica ; Chiricozzi, Elena ; Di Biase, Erika ; Mari, Luigi ; Spedding, Michael ; Ciampa, Maria Grazia ; Fazzari, Maria ; Henriques, Alexandre ; Loberto, Nicoletta ; Mauri, Laura ; Lunghi, Giulia ; Callizot, Noelle

Alterations in glycosphingolipid metabolism have been linked to the pathophysiological mechanisms of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease affecting motor neurons. Accordingly, administration of GM1, a sialic acid‐containing glycosphingolipid, is protective against neuronal damage and supports neuronal homeostasis, with these effects mediated by its bioactive component, the oligosaccharide head (GM1‐OS). Here, we add new evidence to the therapeutic efficacy of GM1 in ALS: Its administration to WT and SOD1G93A motor neurons affected by glutamate‐induced excitotoxicity significantly increased neuronal survival and preserved neurite networks, counteracting intracellular protein accumulation and mitochondria impairment. Importantly, the GM1‐OS faithfully replicates GM1 activity, emphasizing that even in ALS the protective function of GM1 strictly depends on its pentasaccharide.

100 Deals associated with GM1 ganglioside

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External Link

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-	GM1 ganglioside	-	-

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Parkinson Disease	Phase 2	-	LZ Therapeutics, Inc.	-

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


SFN2015	Not Applicable	-	-	GM1 ganglioside	ayfdydkxtt(mscxusrwon) = sjkuwrvqpf yhkkheetpp (ahstnzwxjn )	Positive	20 Oct 2015
				Placebo	ayfdydkxtt(mscxusrwon) = jlxyjlqygg yhkkheetpp (ahstnzwxjn )
delayed start clinical trial (SFN2011)	Not Applicable	Parkinson Disease	77	GM1 ganglioside	yhuhejwohb(pjatbtqtrb) = ifxwjsewxp nhalksuprz (smptwanwnk, 0.50)	Positive	15 Nov 2011
				Placebo	yhuhejwohb(pjatbtqtrb) = rvluyzwryq nhalksuprz (smptwanwnk, 0.49)