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    CANCER:

    Neuroblastoma

    Related cancers:
    3d
    Identification of novel markers for neuroblastoma immunoclustering using machine learning. (PubMed, Front Immunol)
    Six characteristic genes (BATF, CXCR3, GIMAP5, GPR18, ISG20, and IGHM) were identified by machine learning, and these genes are associated with multiple immune-related pathways and immune cells in neuroblastoma. BATF, CXCR3, GIMAP5, GPR18, ISG20, and IGHM may serve as biomarkers that reflect the conditions of the immune microenvironment of neuroblastoma and hold promise in guiding neuroblastoma treatment.
    Journal • Machine learning
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • CXCR3 (C-X-C Motif Chemokine Receptor 3) • BATF (Basic Leucine Zipper ATF-Like Transcription Factor) • ISG20 (Interferon Stimulated Exonuclease Gene 20)
    3d
    Selective Noradrenergic Activation of BDNF Translation by Mirtazapine. (PubMed, Mol Neurobiol)
    These findings provide a new perspective on the critical role of the noradrenergic system in mediating mirtazapine's effects on BDNF translation. We propose a novel mechanism of action in which mirtazapine promotes norepinephrine release and noradrenergic responses by upregulating ADRA2A and ADRB2 while downregulating serotonergic receptors.
    Journal
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    ADRB2 (Adrenoceptor Beta 2) • BDNF (Brain Derived Neurotrophic Factor)
    3d
    Phenotypic and oncological insights in ANNA1 autoimmunity: Age stratification and biomarker analysis. (PubMed, Ann Clin Transl Neurol)
    Young adults with ANNA1 autoimmunity phenotypically resembled older adults but rarely had an underlying cancer. Pediatric patients frequently presented with limbic encephalitis and neuroblastoma and often responded favorably to immunotherapy. Distinct antigenic signatures may underlie differences in clinical presentations. Serum NfL levels may be a biomarker of poor long-term outcomes in ANNA1 autoimmunity.
    Journal • IO biomarker
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    SOX2 • ELAVL4 (ELAV Like RNA Binding Protein 4) • NEFL (Neurofilament Light Chain)
    3d
    Combinatorial immunotherapy of anti-MCAM CAR-modified expanded natural killer cells and NKTR-255 against neuroblastoma. (PubMed, Mol Ther Oncol)
    NKTR-255, a polymer-conjugated recombinant human interleukin-15 agonist, significantly stimulated NK cell proliferation and expansion and further enhanced the in vitro cytotoxic activity and in vivo anti-tumor efficacy of anti-MCAM-CAR-NK cells against NB. Our preclinical studies demonstrate that ex vivo expanded and modified anti-MCAM-CAR-NK cells alone and/or in combination with NKTR-255 are promising novel alternative therapeutic approaches to targeting MCAMhigh malignant NB.
    Journal • IO biomarker
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    MCAM (Melanoma Cell Adhesion Molecule) • IL15 (Interleukin 15)
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    MCAM expression
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    avipendekin pegol (NKTR-255)
    3d
    Cell-based assay to detect small molecules restoring levels of let-7 miRNAs. (PubMed, Am J Cancer Res)
    This novel strategy provides an additional cell-based assay for candidate cancer drug screening in a high throughput setting and will facilitate the identification of anti-cancer drugs. Moreover, this assay could be used to screen shRNA and CRISPR libraries to identify novel components of the LIN28-let-7 axis which may provide new therapeutic targets.
    Journal
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • LIN28B (Lin-28 Homolog B)
    4d
    Metanephrine mirage: distinguishing the phaeocopies, a case report and literature review. (PubMed, Clin Diabetes Endocrinol)
    Neuroblastomas may be misdiagnosed as phaeochromocytomas given the ability to secrete catecholamines and similarities in radiological appearance. Differentiating neuroblastomas from phaeochromocytomas and paragangliomas (PPGL) is critical, but clinically difficult. Genomics are central for management; diagnosing ALK-positive neuroblastoma triggers consideration of ALK-targeted therapy, which is not relevant for PPGL. A critical eye is required for the accurate diagnosis and management of malignant adrenal incidentalomas.
    Review • Journal
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    ALK (Anaplastic lymphoma kinase) • ATRX (ATRX Chromatin Remodeler) • SSTR (Somatostatin Receptor)
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    ALK positive • SSTR Expression
    4d
    MIBG With Dinutuximab +/- Vorinostat (clinicaltrials.gov)
    P1, N=45, Active, not recruiting, New Approaches to Neuroblastoma Therapy Consortium | Trial primary completion date: Jun 2023 --> Feb 2024
    Trial primary completion date
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    CD34 (CD34 molecule)
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    Zolinza (vorinostat) • Unituxin (dinutuximab) • Azedra (iobenguane I 131) • Leukine (sargramostim)
    4d
    Immune-oncology targets and therapeutic response of cell pyroptosis-related genes with prognostic implications in neuroblastoma. (PubMed, Discov Oncol)
    The results suggest that PRGs may serve as a novel prognostic marker for NB patients to provide new immunotherapeutic targets for the clinical treatment of NB patients.
    Journal • IO biomarker
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    IL18 (Interleukin 18) • NLRP3 (NLR Family Pyrin Domain Containing 3)
    4d
    Short and long-term blockades of adenosine 2A, 5-HT2A, and 5-HT7 receptors induce apoptosis, reduce proliferation, and show differential effects on miR-27b-3p expression in neuroblastoma cell lines. (PubMed, Neuroscience)
    Our study provides the first evidence for the relationships between ketanserin/miR-27b-3p/pERK, MSX3/miR-27b-3p/pAKT, and SB269970/miR-27b-3p/pAKT in neuroblastoma cells. Ketanserin, MSX3, and SB269970 drug combinations may be promising therapeutic agents in neuroblastoma cells.
    Preclinical • Journal
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    MIR27B (MicroRNA 27b)
    4d
    PRDX6 contributes to selenocysteine metabolism and ferroptosis resistance. (PubMed, Mol Cell)
    Moreover, we demonstrate the functional significance of this alternative route in human cancer cells, revealing a notable association between elevated expression of PRDX6 and human MYCN-amplified neuroblastoma subtype. Our study sheds light on a previously unrecognized aspect of Sec metabolism and its implications in ferroptosis, offering further possibilities for therapeutic exploitation.
    Journal
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
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    MYCN amplification
    5d
    Protective effect of Apelin-13 on D-glutamic acid-induced excitotoxicity in SH-SY5Y cell line: An in-vitro study. (PubMed, Neuropeptides)
    It highlights Apelin-13's potential as a therapeutic agent against excitotoxicity-induced neuronal damage, emphasizing its ability to modulate key molecular pathways involved in inflammation and oxidative stress. Further in-vivo studies are warranted to explore the long-term neuroprotective effects of Apelin-13 in treating neurodegenerative diseases.
    Preclinical • Journal
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    TNFA (Tumor Necrosis Factor-Alpha) • IL10 (Interleukin 10) • TGFB1 (Transforming Growth Factor Beta 1) • IL1B (Interleukin 1, beta)
    5d
    Amyloid beta-activated alpha-1-syntrophin has ramifications on Rac1 activation, ROS production and neuronal cell death. (PubMed, Eur J Neurosci)
    Our study provides new insight into the mechanism of Aβ-mediated cell death in AD and suggests that MKK6-mediated activation of alpha-1-syntrophin promotes ROS production in neuronal cells, resulting in cell death. This study presents a mechanistic insight into Aβ-mediated cell death and could serve as a paradigm for reducing neuronal cell death in AD.
    Journal
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    RAC1 (Rac Family Small GTPase 1)
    5d
    Molecular characterization of adult non-glioblastoma central nervous system (CNS) tumors to identify potential targettable alterations (AIOM 2024)
    4 pts received TT at recurrence, within clinical trials: one with grade 3 meningioma and ALK rearrangement treated with alectinib, one with PTCH1 mutant medulloblastoma treated with vismodegib, and two with high TMB treated with nivolumab/ipilumumab. The incidence of targettable molecular alterations in adult CNS tumor patients was lower than in GBM. Nevertheless, in a few selected cases TT have the potential to increase treatment options at recurrence and improve outcomes.
    Clinical • Tumor mutational burden • BRCA Biomarker • PD(L)-1 Biomarker • IO biomarker
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    BRAF (B-raf proto-oncogene) • ALK (Anaplastic lymphoma kinase) • PIK3CA (Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha) • TMB (Tumor Mutational Burden) • MET (MET proto-oncogene, receptor tyrosine kinase) • BRCA1 (Breast cancer 1, early onset) • BRCA2 (Breast cancer 2, early onset) • RET (Ret Proto-Oncogene) • ROS1 (Proto-Oncogene Tyrosine-Protein Kinase ROS) • FGFR1 (Fibroblast growth factor receptor 1) • PDGFRA (Platelet Derived Growth Factor Receptor Alpha) • NF1 (Neurofibromin 1) • POLE (DNA Polymerase Epsilon) • MDM2 (E3 ubiquitin protein ligase) • PTCH1 (Patched 1) • NF2 (Neurofibromin 2) • BRCA (Breast cancer early onset) • NTRK (Neurotrophic receptor tyrosine kinase)
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    BRAF V600E • BRCA2 mutation • BRCA1 mutation • TMB-H • PIK3CA mutation • MET amplification • ALK rearrangement • FGFR1 amplification • POLE mutation • NF1 mutation • MDM2 amplification • RET mutation • PTCH1 mutation • NF2 mutation • ROS1 mutation • BRCA mutation • ALK rearrangement + PIK3CA mutation • PTCH1 rearrangement • NTRK fusion
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    FoundationOne® CDx
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    Opdivo (nivolumab) • Yervoy (ipilimumab) • Alecensa (alectinib) • Erivedge (vismodegib)
    5d
    The Wnt/Pyruvate kinase, Muscle axis plays an essential role in the differentiation of mouse neuroblastoma cells. (PubMed, Neurochem Int)
    The Wnt inhibitor ICG-001 and PKM activator ML-265 inhibited ATRA-induced Neuro-2a and N1E-115 differentiation, whereas RNA interference-mediated Pkm silencing promoted Neuro-2a and N1E-115 differentiation, which was reversed by PKM overexpression...These results indicate that Wnt/β-catenin signaling promotes Neuro-2a and N1E-115 differentiation by inhibiting PKM-mediated glycolysis during ATRA-induced differentiation. These findings may provide a new theoretical basis for the role of glycolysis in nerve differentiation.
    Preclinical • Journal
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    PKM (Pyruvate Kinase M1/2)
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    foscenvivint (PRI724)
    5d
    Olfactory Neuroblastoma With Divergent Differentiation: Contemporary Management of Unusual Pathology and Literature Review. (PubMed, Ear Nose Throat J)
    We are the first group to report next-generation sequencing of this tumor, which revealed a pathogenic mutation in PIK3CA and a likely pathogenic variant in RUNX1 (AML1). ON with divergent differentiation is very rare, and more robust studies characterizing molecular drivers and pathology may aid in clinical management.
    Review • Journal
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    PIK3CA (Phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha) • RUNX1 (RUNX Family Transcription Factor 1) • SYP (Synaptophysin)
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    PIK3CA mutation • ROS1 positive
    6d
    Modeling of TDP-43 proteinopathy by chronic oxidative stress identifies rapamycin as beneficial in ALS patient-derived 2D and 3D iPSC models. (PubMed, Exp Neurol)
    In a preliminary drug screening approach with autophagy-promoting drugs, namely rapamycin, lithium carbonate and metformin, only rapamycin prevented ARS-induced loss of TDP-43 splicing activity. Collectively, we established different human cell models of TDP-43 proteinopathy which recapitulate TDP-43 gain and loss of function, prevented by rapamycin administration. Human neuroblastoma cells and patient-derived fibroblasts and 2D- and 3D-iPSC models exposed to chronic oxidative stress represent therefore suitable in vitro platforms for future drug screening approaches in ALS.
    Journal
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    POLD1 (DNA Polymerase Delta 1) • TARDBP (TAR DNA Binding Protein)
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    sirolimus • metformin
    6d
    Simultaneous single-nucleus RNA sequencing and single-nucleus ATAC sequencing of neuroblastoma cell lines. (PubMed, Sci Data)
    Preliminary analysis reveals significant diversity in chromatin landscape and gene expression across neuroblastoma cell lines. This dataset is a valuable resource for studying the transcriptional and epigenetic mechanisms of this deadly childhood disease.
    Preclinical • Journal
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
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    MYCN amplification
    6d
    Artificial intelligence-based morphologic classification and molecular characterization of neuroblastic tumors from digital histopathology. (PubMed, NPJ Precis Oncol)
    A deep learning model using attention-based multiple instance learning (aMIL) and self-supervised learning (SSL) was developed to perform pathologic classification of neuroblastic tumors and assess MYCN-amplification status using H&E-stained whole slide images from the largest reported cohort to date. The model showed promising performance in identifying diagnostic category, grade, mitosis-karyorrhexis index (MKI), and MYCN-amplification with validation on an external test dataset, suggesting potential for AI-assisted neuroblastoma classification.
    Journal
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
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    MYCN amplification
    6d
    Immunomodulatory properties of extracellular vesicles isolated from bone marrow of patients with neuroblastoma: role of PD-L1 and HLA-G. (PubMed, Front Immunol)
    Finally, a high expression of CD56, HLA-G and PD-L1 on BM-derived EVs may represent a good prognostic factor. We described the presence of HLA-G and PDL1-bearing EVs in the BM of NB patients, which may represent a mechanism performed by resident BM cells to counteract the inflammation occurring in the BM microenvironment of NB patients.
    Journal • PD(L)-1 Biomarker • IO biomarker • Immunomodulating
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    PD-L1 (Programmed death ligand 1) • IL6 (Interleukin 6) • NCAM1 (Neural cell adhesion molecule 1) • CSF2 (Colony stimulating factor 2) • HLA-G (Major Histocompatibility Complex, Class I, G)
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    NCAM1 expression
    7d
    Spatial and Single Cell Analyses Reveal Heterogeneity of DNAM-1 Receptor-Ligand Interactions that Instructs Intratumoral γδT-Cell Activity. (PubMed, Cancer Res)
    By interacting with tumor cells differentially expressing CD112 and CD155, intratumoral γδT cells exhibited varying degrees of activation and DNAM-1 expression, representing three major functional subsets. This study underscores the complexity of tumor-immune crosstalk, offering insights into how tumor heterogeneity shapes the immune landscape.
    Journal • IO biomarker
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    PVR (PVR Cell Adhesion Molecule) • NECTIN2 (Nectin Cell Adhesion Molecule 2) • TRIM21 (Tripartite Motif Containing 21)
    7d
    STK33 as the functional substrate of miR-454-3p for suppression and apoptosis in neuroblastoma. (PubMed, Mol Cells)
    Overexpression of miR-454-3p or knockdown of STK33 mRNA promoted autophagy, and at the same time, increased the apoptotic markers in the tested NB cells, indicating a mechanism for the suppressive effect of the agents. Given the difficult-to-drug targets such as STK33 and the recent successes in RNA delivery methods for cancer treatment, it is thought that targeting cancer cells with a suppressive miRNA such as miR-454-3p for STK33-dependent cancer types may be an alternative means of NB therapy.
    Journal
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    MIR454 (MicroRNA 454)
    7d
    Membrane-Targeted Quantum Dot-Based BACE1 Activity Sensors for In Vitro and In Cellulo Assays. (PubMed, ACS Appl Mater Interfaces)
    In vitro, the sensor demonstrated stability under acidic conditions, and using high-throughput plate reader assays, we determined BACE1 activity in-line with literature values and enabled the obtainment of the inhibitor constant of verubecestat, a small molecule inhibitor. The sensor was also transitioned to cellular experiments, where it demonstrated sensitivity to BACE1 activity and its modulation upon inhibitor treatment in a neuroblastoma cell line.
    Preclinical • Journal
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    APP (Amyloid Beta Precursor Protein)
    7d
    Leveraging Neural Crest-Derived Tumors to Identify NF1 Cancer Stem Cell Signatures. (PubMed, Cancers (Basel))
    By focusing on the molecular and cellular dynamics within these tumors, we summarize CSC signatures in tumor maintenance, progression, and treatment resistance. A review of these signatures in the context of NF1 will provide insights into NF1 tumor biology and pave the way for developing targeted therapies and improving treatment outcomes for NF1 patients.
    Review • Journal • Cancer stem
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    NF1 (Neurofibromin 1)
    7d
    Accurate Measurement of Cell Number-Normalized Differential Gene Expression in Cells Treated With Retinoic Acid. (PubMed, Bio Protoc)
    • RNA-seq using control RNAs spiked-in on a per-cell basis more accurately reflects global expression changes, when comparing cell populations with substantially different MYCN expression levels. • In MYCN-amplified neuroblastoma, retinoic acid dramatically decreases MYCN expression levels, resulting in large changes in overall RNA expression levels per cell.
    Journal
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    MYC (V-myc avian myelocytomatosis viral oncogene homolog) • MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
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    MYCN amplification • MYC expression • MYCN expression
    7d
    Evaluating the Biodegradability and Cellular Compatibility of Mg-Zn-Nd Alloy for Vascular Stent Application. (PubMed, Adv Biol (Weinh))
    Western blotting shows favorable expression levels of apoptotic and anti-apoptotic markers in the ZN20 group. ZN20 alloy demonstrates enhanced corrosion resistance, favorable endothelial compatibility, and reduced cytotoxicity, endorsing its safe application in vascular stent use.
    Journal
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    CD31 (Platelet and endothelial cell adhesion molecule 1) • PECAM1 (Platelet And Endothelial Cell Adhesion Molecule 1)
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    CD31 expression
    7d
    METTL3/MYCN cooperation drives neural crest differentiation and provides therapeutic vulnerability in neuroblastoma. (PubMed, EMBO J)
    In addition, we provide evidence that sustained overexpression of the MYCN oncogene in tNCC drives METTL3 recruitment to a specific subset of genes including posterior HOX genes creating an undifferentiated state. Moreover, METTL3 depletion/inhibition induces DNA damage and differentiation of MYCN overexpressing cells and increases vulnerability to chemotherapeutic drugs in MYCN-amplified patient-derived xenografts (PDX) in vivo, suggesting METTL3 inhibition could be a potential therapeutic approach for NB.
    Journal
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor) • METTL14 (Methyltransferase 14) • METTL3 (Methyltransferase Like 3) • WTAP (WT1 Associated Protein)
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    MYCN amplification • MYCN expression
    7d
    Conditional Activation of c-MYC in Distinct Catecholaminergic Cells Drives Development of Neuroblastoma or Somatostatinoma. (PubMed, Cancer Res)
    Thus, c-MYC overexpression results in different but related tumor types depending on the targeted cell. The GEMMs represent valuable tools for testing immunotherapies and targeted therapies for these diseases.
    Journal • IO biomarker
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    MYC (V-myc avian myelocytomatosis viral oncogene homolog) • PDX1 (Pancreatic And Duodenal Homeobox 1)
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    MYC overexpression • MYC expression
    7d
    Integrating multi-omics data reveals neuroblastoma subtypes in the tumor microenvironment. (PubMed, Life Sci)
    Drug sensitivity prediction analysis suggested that the MES subtype may respond favorably to MEK inhibitors, while the MYCN subtype may be susceptible to Bcl-2 inhibitors. Our integrative multi-omics approach enabled precise stratification of NB into biologically distinct subtypes, potentially facilitating the development of subtype-specific therapeutic strategies for improved patient management and survival outcomes.
    Journal
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    MYCN (MYCN Proto-Oncogene BHLH Transcription Factor)
    8d
    Phase 2 Study of Bintrafusp Alfa in Recurrent/Metastatic Olfactory Neuroblastoma (BARON). (clinicaltrials.gov)
    P2, N=11, Completed, National Cancer Institute (NCI) | Active, not recruiting --> Completed | Trial completion date: Aug 2025 --> Jul 2024 | Trial primary completion date: Aug 2025 --> Jul 2024
    Trial completion • Trial completion date • Trial primary completion date
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    PD-L1 (Programmed death ligand 1) • CD4 (CD4 Molecule)
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    bintrafusp alfa (M7824)
    9d
    GINAKIT2: GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children with Neuroblastoma (clinicaltrials.gov)
    P1, N=54, Recruiting, Baylor College of Medicine | N=36 --> 54
    Enrollment change
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    KUR-501
    10d
    A Phase II Study of Rivoceranib for Patients With Recurrent or Metastatic Olfactory Neuroblastoma (clinicaltrials.gov)
    P2, N=0, Withdrawn, M.D. Anderson Cancer Center | N=16 --> 0 | Trial completion date: Nov 2028 --> Nov 2024 | Initiation date: Apr 2025 --> Nov 2024 | Not yet recruiting --> Withdrawn | Trial primary completion date: Nov 2026 --> Nov 2024
    Enrollment change • Trial completion date • Trial initiation date • Trial withdrawal • Trial primary completion date • Metastases
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    AiTan (rivoceranib)
    10d
    A Study on the Efficacy of GD2-CAR T Cells in the Treatment of Neuroblastoma (clinicaltrials.gov)
    P1, N=30, Recruiting, The General Hospital of Western Theater Command
    New P1 trial • CAR T-Cell Therapy • IO biomarker
    16d
    Alpha/Beta CD19+ Depleted Haploidentical Transplantation + Zometa for Pediatric Hematologic Malignancies and Solid Tumors (clinicaltrials.gov)
    P1, N=22, Recruiting, University of Wisconsin, Madison | Suspended --> Recruiting | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2024 --> Dec 2025
    Enrollment open • Trial completion date • Trial primary completion date
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    melphalan • zoledronic acid
    16d
    NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial (clinicaltrials.gov)
    P2, N=62, Recruiting, New Approaches to Neuroblastoma Therapy Consortium | Not yet recruiting --> Recruiting
    Enrollment open • Combination therapy
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    temozolomide • irinotecan • Unituxin (dinutuximab) • Leukine (sargramostim)
    16d
    Combined inhibition of histone methyltransferases EZH2 and DOT1L is an effective therapy for neuroblastoma. (PubMed, Cancer Med)
    Our results support further investigation of HMT inhibitor combinations as a therapeutic approach in NB.
    Journal • Epigenetic controller
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    EZH2 (Enhancer of zeste 2 polycomb repressive complex 2 subunit) • DOT1L (DOT1 Like Histone Lysine Methyltransferase) • ATF4 (Activating Transcription Factor 4)
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    GSK343
    16d
    An automated pheochromocytoma and paraganglioma lesion segmentation AI-model at whole-body 68Ga- DOTATATE PET/CT. (PubMed, EJNMMI Res)
    The developed deep learning-based AI model showed reliable performance for automated segmentation of metastatic PPGL lesions on whole-body 68Ga-DOTATATE-PET/CT images, which may be beneficial for tumor burden estimation for objective evaluation during therapy follow-up. https://www.
    Journal
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    SSTR (Somatostatin Receptor)
    17d
    BRICC: Biomarkers of Resiliency In Childhood Cancer Surgery (clinicaltrials.gov)
    P=N/A, N=40, Not yet recruiting, Mayo Clinic
    New trial • Surgery
    17d
    NANT 2013-01: Immunotherapy of Relapsed Refractory Neuroblastoma With Expanded NK Cells (clinicaltrials.gov)
    P1, N=13, Active, not recruiting, New Approaches to Neuroblastoma Therapy Consortium | Trial completion date: Sep 2024 --> Sep 2025 | Trial primary completion date: Sep 2024 --> Sep 2025
    Trial completion date • Trial primary completion date
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    lenalidomide • Unituxin (dinutuximab)
    17d
    Pinus koraiensis essential oil enhances glucose uptake and proliferation in SH-SY5Y neuroblastoma cells. (PubMed, Sci Rep)
    Using gas chromatography-mass spectrometry, we confirmed the high levels of α-pinene, an inducer of GLUT4 expression, in PKSZ-EO. These results suggest that PKSZ-EO exerts a protective effect against glucose depletion stress, highlighting its potential as a therapeutic agent for NDDs.
    Journal
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    SLC2A4 (Solute Carrier Family 2 Member 4) • SLC2A2 (Solute Carrier Family 2 Member 2)
    17d
    Automated and closed clinical-grade manufacturing protocol produces potent NK cells against neuroblastoma cells and AML blasts. (PubMed, Sci Rep)
    Characterization of the NK cells before and after cytokine activation revealed a notable increase in the expression of activation markers, particularly CD69, consistent with enhanced functionality. Intriguingly, the NK cells exhibited increased killing efficacy against patient-derived CD33 + AML blasts and NBL cells in vitro, suggesting a potential therapeutic benefit in AML and NBL.
    Journal • IO biomarker
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    CD33 (CD33 Molecule) • CD69 (CD69 Molecule) • IL2 (Interleukin 2) • NCAM1 (Neural cell adhesion molecule 1) • IL15 (Interleukin 15)
    18d
    FOXR2 targets LHX6+/DLX+ neural lineages to drive CNS neuroblastoma. (PubMed, Cancer Res)
    These data indicate that NB-FOXR2 originate from LHX6+/DLX+ interneuron lineages, a lineage-of-origin distinct from that of other FOXR2-driven brain tumors, highlight the susceptibility of ventral telencephalon-derived interneurons to FOXR2-driven oncogenesis, and suggest that FOXR2-induced activation of glial programs may explain the mixed neuronal and oligodendroglial features in these tumors. More broadly, this work underscores systematic profiling of brain development as an efficient approach to orient oncogenic targeting for in vivo modeling, critical for the study of rare tumors and development of therapeutics.
    Journal
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    FOXR2 (Forkhead Box R2) • LHX6 (LIM Homeobox 6)
    18d
    ALS-associated FUS mutation reshapes the RNA and protein composition of stress granules. (PubMed, Nucleic Acids Res)
    Moreover, we show that mutant FUS, together with its protein interactors and their target RNAs, are responsible for the reshaping of the mutant SG transcriptome with alterations that can be linked to neurodegeneration. Our data describe the molecular differences between physiological and pathological SG in ALS-FUS conditions, showing how FUS mutations impact the RNA and protein composition of these condensates.
    Journal
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    FUS (FUS RNA Binding Protein)