Subhadhirasakul S.Sujjitjoon J.Yenchitsomanus P.t.Jarutatsanangkoon K.Loon N.W.Sanpakit K.Buaboonnam J.Takpradit C.Yuti P.Sawasdee N.Luangwattananun P.Sangkitporn S.Limmahachai A.Atchaneeyasakul L.o.Mahidol University2026-04-172026-04-172026-01-01Pediatric Blood and Cancer (2026)15455009https://repository.li.mahidol.ac.th/handle/123456789/116240Effective 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.MedicineArticleSCOPUS10.1002/1545-5017.703342-s2.0-10503541038215455017