Novel Treatment of Retinoblastoma

The aim of this project is to advance the development of a new drug product and drug delivery device for the treatment of Retinoblastoma, a childhood cancer of the eye. This type of ocular cancer affects children globally and often results in loss of vision or spreading of life-threatening cancers to other parts of the body. Visual impairment and blindness reduce quality of life and cause considerable economic burdens for affected children, their families, and caregivers. The ocular drug delivery device is designed to precisely deliver drugs adjacent to the site of the cancer in the eye. This is done in a less invasive manner than existing treatments. It causes significantly less physical trauma to the patient and mitigates the potential to spread the cancers around the patient's body. The drug product aims to deliver safe and robust localised treatment while reducing side effects that come from systemic and intra-arterial treatments. The critical project outcome is the safe and effective treatment of children with Retinoblastoma measured by reduction and eradication of the cancers and reduced rates of recurrence and side effects over the current standard of care. Demonstration of the device's clinical effectiveness includes completion of a safe injection procedure and successful delivery of a localised treatment adjacent to the tumour in the eye of Retinoblastoma patients. Fabrication of the device and drug uses highly specialised production mechanisms. The combination of these innovative components along with clinical data will expand an already strong intellectual property position. Current UK Innovate funding is advancing this project to first clinical trials, with the expectation that its successful completion will one day become a treatment for children with significant unmet needs.