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gangliosides (Nervomax / gangliosides, Fidia / Neurosido)

✓ Approved

Gramon · 治疗药物

什么是 gangliosides？

gangliosides 是一种治疗药物，由Gramon研发。该药已获批，用于治疗相关适应症，给药途径：Unknown。

药物档案

商品名Nervomax, gangliosides, Fidia, Neurosido
公司Gramon
给药途径Unknown
状态Approved
来源： ClinicalTrials.gov, Gramon

治疗适应症

gangliosides 针对 4 个适应症，涉及 3 个治疗领域。

治疗领域疾病/病症分期
Nervous system disordersDiabetic neuropathy✓ Approved
Nervous system disordersNeuropathy peripheral✓ Approved
Blood and lymphatic system disordersNeutropenia✓ Approved
Congenital, familial and genetic disordersRetinitis pigmentosa✓ Approved

相关研究文献

PubMedbioRxiv : the preprint server for biology2026-10-25

Autoantibodies from recovered Guillain-Barré syndrome patients exhibit altered effector functions that prevent subsequent neuron degeneration.

Rogers Ashley M AM, McAlpine Jessica L JL, Yang Xu X, Jahan Israt I et al.

Guillain-Barré syndrome (GBS) is an autoimmune polyneuropathy that is the leading cause of nonpoliovirus-associated acute flaccid paralysis worldwide. In most cases, GBS occurs following an infection, most commonly Campylobacter jejuni , through the induction of antibodies recognizing bacterial ganglioside-mimicking lipooligosaccharides that cross-react with human neuronal gangliosides. This post-infectious autoimmune cascade causes neuropathy, from which patients can recover as their anti-ganglioside antibody titers diminish and immunostimulation decreases. In this study, we find 10% of clinically confirmed GBS patients maintain high titers of circulating anti-ganglioside antibodies more than one decade after recovery. These antibodies no longer cause neuropathy compared to acute sera from the same patients using a human pluripotent stem cell-derived sensory neuron model with human complement. We found both IgG subclass and glycoform differences between paired acute and recovered GBS patient sera, including anti-inflammatory modifications on isolated anti-GM1 ganglioside antibodies. Together, these data suggest that patients with GBS select for non-pathogenic variants of autoantibodies that are no longer capable of damaging their neurons, but may still protect against C. jejuni infection.

PMID 42922744
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PubMedChemphyschem : a European journal of chemical physics and physical chemistry2026-10-25

Phospholipid Monolayers and Vesicular Membranes Show Only Minor Structural Changes After Interaction With α -Synuclein at Low and Physiological Concentrations.

Major András A, von Campenhausen Eva E, Hilger Riko R, Bruns Hendrik H et al.

We study how the lipid membrane structure changes upon interaction with α -synuclein (aSyn) at low and physiological concentrations, using three different X-ray scattering methods and two well-established model systems, the Langmuir film balance and small unilamellar vesicles in bulk solution. While aSyn is highly membrane active, we find that at physiological protein concentration it induces only rather small changes in lipid membrane structure and vesicle shape. The vertical density profile of Langmuir lipid monolayers shows no observable changes for DPPC:DPPS and DOPC:DOPS, and moderate changes only for a model system containing gangliosides. Contrarily, the protein induces more pronounced changes in the bilayer density profile of highly curved anionic lipid vesicles, but without rupture and major remodeling of vesicular shapes as reported previously for pathological concentrations. In fact, the shape of the vesicles showed even a slight reduction in ellipticity. In addition to aSyn only, we also study the structure of the monolayer and vesicular membranes in the presence of two major synaptic proteins, aSyn and synapsin 1, at concentrations where the latter induces lipid-protein condensates.

PMID 42921993
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PubMedBiophysical reviews2026-10-25

Dynamics and recognition of glycosphingolipids in membranes: insights from different scales.

Makshakova Olga N ON, Römer Winfried W

Glycosphingolipids are the most complex molecules among mammalian lipids. They consist of a ceramide backbone and a glycan head, so their description combines the challenges of lipids and carbohydrates. The carbohydrate moiety (with high conformational flexibility) serves as a receptor for carbohydrate-binding proteins, e.g., lectins, including molecules of pathogenic origin. Glycosphingolipids exhibit inhomogeneous lateral diffusion, which supports the formation of nanoscale functional complexes during signal transduction. These complexes enriched with glycosphingolipids can also contain GPI-anchored proteins, transmembrane proteins, and phospholipids from both membrane leaflets via acyl chain interdigitation, thereby transmitting the signal from outside the cell to the cell interior. The formation process of such molecular complexes extends over a wide spatial and temporal range and still remains largely unexplored due to the resolution limits of existing observation techniques. In this review, we bring together insights into the dynamics of glycosphingolipids in membranes and their interactions with carbohydrate-binding proteins from high/super-resolution fluorescence microscopy and molecular modelling-illustrated with examples such as the gangliosides GM1 and GM3, and the globoside globotriaosylceramide (also known as Gb3, CD77, and Pk antigen)-to exemplify how the gap in spatial and temporal resolution between experimental and computational approaches is steadily narrowing. The selected case studies present investigations using both cells and membrane model systems, such as giant unilamellar vesicles and supported lipid bilayers, together with in silico lipid bilayers with the aim of reducing the environmental complexity to determine the key molecular determinants involved in functional complex formation, membrane bending, endocytosis, and signalling.

PMID 42927094
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PubMedJournal of lipid research2026-10-22

NEU3 at the Plasma Membrane: Context-Dependent Ganglioside Remodeling in Health and Disease.

Cirillo Federica F, Rota Paola P, Anastasia Luigi L, Piccoli Marco M

Cell-surface gangliosides tune receptor signaling, cell-state transitions, and responses to stress. NEU3 is a mammalian sialidase associated with the plasma membrane and endomembranes that preferentially removes terminal sialic acids from gangliosides. Here, we critically review biochemical studies, cultured-cell experiments, animal models, and observations in human tissues that link NEU3 to myogenesis, hypoxic adaptation, cardioprotection, ganglioside storage disorders, cancer, fibrosis, and intestinal inflammation. Across these settings, NEU3 can alter the local ganglioside environment of receptors and signaling complexes, but the strength and direction of the resulting effects vary with cell type, substrate availability, localization, expression level, and duration of activity. We therefore frame NEU3 as a context-dependent regulator rather than a uniformly beneficial or harmful enzyme. This evidence-weighted model helps reconcile apparently opposing findings while highlighting important uncertainties in membrane topology, extracellular delivery, substrate selection, and pharmacological specificity. Selective inhibitors, tissue-restricted delivery, and biomarker-guided approaches are promising, but clinical translation will require stronger target-engagement data and broader in vivo validation.

PMID 42906757
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PubMedAngewandte Chemie (International ed. in English)2026-10-15

Substrate Promiscuity of CHST1 Enables Efficient Synthesis of Galactose-6-O-Sulfated Glycans for Siglec Recognition Profiling.

Bao Shumin S, Shen Tangliang T, Shabahang MohammadHossein M, Fan Shuquan S et al.

The galactose-6-O-sulfation (Gal-6-su) is a prevalent glycan modification that influences Siglec recognition across diverse glycan structures. In this study, we achieved high-level expression of an active truncated CHST1 (carbohydrate sulfotransferase 1, a Gal-6-O-sulfotransferase) and conducted systematic substrate specificity studies. We identified remarkable substrate promiscuity toward β-galactosides with activity significantly enhanced by α2,3-sialylation. Most other modifications on Gal are tolerated by CHST1 except for those at its C4-hydroxyl position, and the activity is strongly influenced by proximal residues and linkage type. We also observed an unexpected activity toward β-N-acetyl-galactosamine (GalNAc) glycosides including chondroitin, resembling the activity of CHST3. Exploiting this substrate flexibility, chemoenzymatic modular strategies were developed to prepare novel sulfated gangliosides and O-glycans for profiling Siglec-glycan interactions. Glycan microarray analyses, using synthesized sulfated glycans alongside their non-sulfated counterparts, demonstrate that Gal-6-su broadly enhances binding across Siglec-1, -3, -5, -7, -8, -15, and murine Siglec-E and -F, while inhibiting binding to Siglec-9. A clear Siglec preference to α2,3-sialylated and 6-O-sulfated β1,4-galactosides was also observed. Together, these findings define key determinants of CHST1 specificity, expand the glycan synthesis toolbox, and provide new insight into how Gal-6-su modulates Siglec recognition, enabling future therapeutic and biochemical applications.

PMID 42879309
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PubMedClinical and translational medicine2026-10-15

Graph attention network-driven single-cell omics analysis reveals how B4GALNT1-mediated glycosylation remodelling of the ferroptosis-pyroptosis network promotes drug resistance in hepatocellular carcinoma.

Bie Lihan L, Xu Biao B, Shen Haitao H, Ren Shuwen S et al.

Acquired resistance to sorafenib in hepatocellular carcinoma (HCC) is accompanied by coordinated changes in oxidative-stress handling and regulated cell-death pathways, yet how membrane organisation connects these adaptations is not fully resolved. We therefore examined whether B4GALNT1, a ganglioside biosynthetic glycosyltransferase, couples changes in ganglioside composition and lipid-raft architecture to ferroptosis regulation and caspase-1/GSDMD-associated signalling in sorafenib-resistant HCC. We combined TCGA-LIHC analysis, single-cell transcriptomics, pathway-level analyses, and graph attention network modelling with a treatment-annotated local cohort of 92 patients with HCC who received sorafenib. Functional experiments were conducted in Huh7/Huh7-SR cells and additional liver-tumour models using B4GALNT1 gain- and loss-of-function, an independent second-sgRNA knockout clone, GM1 rescue, and re-expression of either wild-type B4GALNT1 or the catalytic-loss D358A variant. Validation included ganglioside profiling, detergent-resistant membrane isolation, lipid-raft disruption/repletion, ferroptosis-directed pharmacology, cell-death measurements, and xenograft studies. B4GALNT1 was enriched in tumour-associated hepatocyte populations and was upregulated in Huh7-SR cells. In the local cohort, higher B4GALNT1 protein abundance tracked with less favourable response to sorafenib and poorer survival outcomes. Increasing B4GALNT1 shifted ganglioside abundance toward GM2/GM1/GD2 and increased GM1-enriched raft-associated fractions, whereas knockout produced reciprocal changes. B4GALNT1-high cells showed increased GPX4 and SLC7A11, reduced lipid peroxidation and labile Fe2 +, and lower ferroptotic injury. At the same time, caspase-1/GSDMD-associated signalling and IL-1β/IL-18 release increased without widespread terminal membrane lysis. Exogenous GM1 partially rescued knockout-associated changes, and wild-type B4GALNT1 restored the phenotype more effectively than the D358A mutant. Concordant directional effects were observed in additional cell models and in xenografts. The data are consistent with a model in which B4GALNT1 reshapes ganglioside composition and GM1-enriched membrane microdomains, thereby supporting a drug-adapted state that combines greater ferroptosis resistance with restricted caspase-1/GSDMD-associated membrane permeabilisation. This integrated phenotype accompanies reduced sorafenib responsiveness and offers a mechanistic basis for acquired resistance in HCC. B4GALNT1 is associated with sorafenib resistance and poorer outcomes in treated HCC. B4GALNT1 remodels gangliosides and lipid rafts, limiting ferroptotic injury. Caspase-1/GSDMD signalling increases without widespread terminal membrane lysis. Genetic rescue and cross-model studies support B4GALNT1 specificity.

PMID 42878060
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