Virus-based treatment for retinoblastoma shows promising results

A new potential therapy for retinoblastoma has had positive effects in mouse models and a pilot clinical trial involving two patients


A potential treatment for retinoblastoma has shown promising results in mouse models and a pilot clinical trial.

The research, which was described in Science Translational Medicine, involved harnessing a virus that infects and kills cells with the dysfunctional genetic pathway that leads to retinoblastoma.

The treatment, named VCN-01, was found to be safe in rabbits. Mice with retinoblastoma who were injected with the therapy experienced curtailed tumour growth compared to mice treated with chemotherapy.

The new treatment also prevented metastasis and extended the time to enucleation.

In two paediatric patients with retinoblastoma, VCN-01 replicated within tumour cells without causing systemic inflammation.

The authors highlighted that these results suggest the virus-based treatment warrants further investigation.