Intravitreal GD2‐Specific Chimeric Antigen Receptor T‐Cell Therapy for Refractory Retinoblastoma
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.
Authors
- Jatuporn Sujjitjoon (ORCID: https://orcid.org/0000-0002-5459-8031)
- La‐ongsri Atchaneeyasakul
- Pornpimon Yuti (ORCID: https://orcid.org/0000-0002-9121-5079)
- Siripakorn Sangkitporn
- Nunghathai Sawasdee (ORCID: https://orcid.org/0000-0003-1951-9948)
- Kleebsabai Sanpakit (ORCID: https://orcid.org/0000-0003-3452-1894)
- Jassada Buaboonnam (ORCID: https://orcid.org/0000-0002-5240-1071)
- Piriya Luangwattananun (ORCID: https://orcid.org/0000-0001-6160-1967)
- Arnan Limmahachai (ORCID: https://orcid.org/0000-0002-4934-6518)
- Subongkoch Subhadhirasakul
- Kridtin Jarutatsanangkoon
- Ng Wei Loon
- Chayamon Takpradit
- Pa‐thai Yenchitsomanus
Institutions
- Ministry of Public Health (TH)
- Siriraj Hospital (TH)
Publication Details
- Journal
- Pediatric Blood & Cancer
- Published
- 2026-04-11
- DOI
- https://doi.org/10.1002/1545-5017.70334
- Primary Topic
- CAR-T cell therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- National Research Council of Thailand