Intravitreal GD2-Specific Chimeric Antigen Receptor T-Cell Therapy for Refractory Retinoblastoma

Area of Science:

• Oncology
• Immunology
• Ophthalmology

Background:

• Advanced retinoblastoma (RB) presents limited treatment options.
• GD2-specific chimeric antigen receptor (CAR) T cells demonstrate antitumor efficacy with low toxicity.
• Previous evaluation of CAR T-cells in RB was lacking.

Purpose of the Study:

• To evaluate the feasibility and safety of intravitreal GD2-CAR T-cell therapy in a patient with refractory retinoblastoma.
• To assess the therapeutic effect and immune response induced by GD2-CAR T-cell injections in the eye.

Main Methods:

• A single patient with refractory retinoblastoma received two intravitreal injections of GD2-CAR T cells under compassionate use.
• Ocular inflammation, intraocular pressure, and tumor response were monitored.
• Histopathological analysis was performed after enucleation.

Main Results:

• Intravitreal GD2-CAR T-cell therapy was feasible and generally well-tolerated.
• Localized ocular inflammation occurred post-injection, subsiding and recurring.
• Histopathology revealed significant peritumoral lymphocytic infiltration and tumor regression.
• Enucleation was required due to elevated intraocular pressure.

Conclusions:

• Intravitreal GD2-CAR T-cell therapy can induce localized immune activation and tumor regression in retinoblastoma.
• The treatment demonstrated feasibility and tolerability, supporting further investigation.
• Potential side effects such as ocular inflammation and increased intraocular pressure require careful monitoring.