肿瘤细胞治疗研究
英文原题：Intravitreal GD2-Specific Chimeric Antigen Receptor T-Cell Therapy for Refractory Retinoblastoma.
Intravitreal GD2-Specific Chimeric Antigen Receptor T-Cell Therapy for Refractory Retinoblastoma.
6周时眼内压升高需行眼球摘除术。
晚期、难治性视网膜母细胞瘤（RB）的有效治疗仍然有限。GD2 特异性嵌合抗原受体（CAR）T 细胞显示出强效抗肿瘤活性且毒性极小，但此前尚未在 RB 中评估。在同情用药下，一名难治性 RB 患者接受了两次玻璃体内 GD2-CAR T 细胞注射。1 周内肿瘤周围出现局限性眼部炎症，随后消退，并在第二次注射后复发。眼压升高导致 6 周时需行眼球摘除术。组织病理学显示肿瘤周围密集淋巴细胞浸润，未累及前节。玻璃体内 GD2-CAR T 细胞治疗可行、耐受良好，并诱导了局部免疫激活和肿瘤消退，支持进一步研究。
Effective treatments for advanced, treatment-resistant retinoblastoma (RB) remain limited. GD2-specific chimeric antigen receptor (CAR) T cells show potent antitumor activity with minimal toxicity but have not previously been evaluated in RB. Under compassionate use, a patient with refractory RB received two intravitreal GD2-CAR T-cell injections. Localized ocular inflammation developed around the tumor within 1 week, subsided, and recurred after the second injection. Elevated intraocular pressure required enucleation at 6 weeks. Histopathology demonstrated dense peritumoral lymphocytic infiltration without anterior segment involvement. Intravitreal GD2-CAR T-cell therapy was feasible, well tolerated, and induced localized immune activation and tumor regression, supporting further investigation.
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