Novel Treatment of Retinoblastoma

该项目的目的是推进用于治疗视网膜母细胞瘤（一种儿童眼癌）的新药物产品和药物输送装置的开发。这种类型的眼癌影响全球儿童，通常导致视力丧失或危及生命的癌症扩散到身体的其他部位。视力障碍和失明会降低患者的生活质量，并给受影响的儿童、他们的家庭和护理人员带来相当大的经济负担。眼部药物输送装置旨在将药物精确输送到眼睛中癌症部位附近。这是以比现有治疗更少侵入性的方式完成的。它对患者造成的身体创伤显著减少，并减轻了癌症在患者身体周围扩散的可能性。该药物产品旨在提供安全和稳健的局部治疗，同时减少全身和动脉内治疗的副作用。关键项目成果是安全有效地治疗视网膜母细胞瘤儿童，通过减少和根除癌症以及降低复发率和副作用来衡量，超过目前的护理标准。该设备的临床有效性的证明包括完成安全的注射程序，并成功地在视网膜母细胞瘤患者的眼睛肿瘤附近进行局部治疗。该设备和药物的制造使用高度专业化的生产机制。这些创新组件与临床数据的结合沿着将扩大本已强大的知识产权地位。目前英国创新基金正在推进该项目的第一次临床试验，期望其成功完成有一天将成为一种治疗有重大未满足需求的儿童的方法。

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.