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.
Subhadhirasakul et al. (2026) studied this question.