Neuroblastoma

P2, N=20, Recruiting, Children's Hospital Medical Center, Cincinnati

Melittin may emerge as a molecule that modulates inflammation-related gene expression in neuroblastoma cells and may be associated with IL-37a-related regulatory responses. These findings suggest that melittin could act as a potential modulator of inflammation-associated signaling pathways.

This case highlights that ectopic maxillary sinus olfactory neuroblastoma can mimic neuroendocrine carcinoma on limited biopsy, emphasizing the importance of complete resection and thorough histopathological evaluation for accurate diagnosis and grading. Hyams grade I disease in atypical locations carries an excellent prognosis with combined endoscopic surgery and radiotherapy, underscoring the prognostic value of Hyams grading and the need for long-term surveillance.

In patients with atypical findings, tissue diagnosis by core needle or open surgical biopsy, followed by histopathologic and immunohistochemical evaluation, should be considered before initiating empiric chemotherapy. Early multidisciplinary assessment is essential for accurate diagnosis and appropriate management.

Receiver operating characteristic (ROC) analyses demonstrated strong discriminative performance for both the gene signature (AUC 0.87-1.00) and the associated miRNAs (AUC 0.95-1.00). Together, these findings define a novel PBMC-based gene-miRNA signature that mirrors central ALS pathology and shows high diagnostic accuracy and disease specificity, highlighting its potential as a minimally invasive biomarker for ALS.

Our study uncovered the YTHDF2/CYLD/NOX4 axis as a novel ferroptosis regulator in PD, revealing a dual epitranscriptomic-posttranslational therapeutic target for neuroprotection.

P2, N=27, Recruiting, Tianjin Medical University Cancer Institute and Hospital | Not yet recruiting --> Recruiting

Among them, compound 9 behaved as a well-balanced dual-acting inhibitor (hBChE, IC50 = 557 nM; hMAO B, IC50 = 142 nM) capable of mitigating interleukin-6 release from stimulated human microglia clone 3 (HMC3) cells in a dose-dependent manner and of counteracting 6-hydroxydopamine (6-OHDA) toxicity in SH-SY5Y neuroblastoma cell lines. Moreover, X-ray crystal structures of 9 in both hBChE and hMAO B were solved at 2.36 Å and 1.60 Å resolution, respectively.

This study identifies critical thresholds for identifying children at high risk of pulmonary complications after neuroblastoma resection, facilitating more accurate risk stratification and early intervention. These findings have important clinical implications for optimizing perioperative management and reducing postoperative morbidity.

We implemented these two xTRACeRs as Chimeric Antigen Receptor (CAR) T cells and demonstrated their potent killing efficacy and specificity. Overcoming restriction across HLA supertypes lifts a key barrier in HLA-targeted immunotherapy by expanding patient coverage.

P1/2, N=374, Active, not recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: May 2026 --> May 2027 | Trial primary completion date: May 2026 --> May 2027

The patient received Vincristine, Adriamycin, Cyclophosphamide Ifosfamide, Etoposide (VAC/IE) chemotherapy according to the European Ewing tumour Working Initiative of National Groups (EUROE.W.I.N.G.) protocol and achieved full neurological recovery. This case illustrates the diagnostic and therapeutic challenges posed by Ewing-like sarcomas in the absence of molecular confirmation. Function-preserving subtotal resection combined with multimodal chemotherapy may lead to excellent outcomes and may be preferable to radical resection when the latter carries a high neurological risk.