Intravitreal GD2-Specific Chimeric Antigen Receptor T-Cell Therapy for Refractory Retinoblastoma
Issued Date
2026-01-01
Resource Type
ISSN
15455009
eISSN
15455017
Scopus ID
2-s2.0-105035410382
Journal Title
Pediatric Blood and Cancer
Rights Holder(s)
SCOPUS
Bibliographic Citation
Pediatric Blood and Cancer (2026)
Suggested Citation
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. Intravitreal GD2-Specific Chimeric Antigen Receptor T-Cell Therapy for Refractory Retinoblastoma. Pediatric Blood and Cancer (2026). doi:10.1002/1545-5017.70334 Retrieved from: https://repository.li.mahidol.ac.th/handle/123456789/116240
Title
Intravitreal GD2-Specific Chimeric Antigen Receptor T-Cell Therapy for Refractory Retinoblastoma
Author's Affiliation
Corresponding Author(s)
Other Contributor(s)
Abstract
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.