Intraocular Chemotherapy for Retinoblastoma

This trial explores the safety and effectiveness of adding melphalan, a chemotherapy injected directly into the eye, to the standard chemotherapy for retinoblastoma, a rare eye cancer. The focus is on cases where the cancer has spread into the eye's fluid, making treatment more challenging. Researchers hope melphalan can kill cancer cells more effectively. Suitable candidates have newly diagnosed retinoblastoma in one or both eyes with specific cancer spread patterns. As a Phase 2 trial, the research measures how well the treatment works in an initial, smaller group, contributing to advancements in retinoblastoma care.

The trial protocol does not specify whether you need to stop taking your current medications. However, it does mention that patients must not have had any prior anti-cancer therapy other than specific eye treatments. It's best to discuss your current medications with the trial team.

Research shows that injecting melphalan directly into the eye is generally well-tolerated for treating retinoblastoma, a type of eye cancer. Studies have found this method to be safe for patients. These studies also report no major differences in eye safety or survival rates with varying doses of melphalan. Thus, melphalan presents a safe option for patients with this eye cancer.¹²³⁴⁵

Unlike the standard treatments for retinoblastoma, which often involve systemic chemotherapy, melphalan is delivered directly into the eye through intraocular chemotherapy. This targeted delivery method allows for higher concentrations of the drug to reach the tumor with potentially fewer systemic side effects. Researchers are excited about melphalan because it offers a promising way to treat eye tumors more effectively and safely, potentially preserving vision and improving quality of life for patients.

Research has shown that injecting melphalan directly into the eye can effectively treat retinoblastoma, a type of eye cancer. In this trial, participants will receive melphalan as part of the treatment arm. This method helps control tumors, especially those in the vitreous humor, the jelly-like fluid inside the eye. Delivering melphalan through the main artery of the eye, known as intra-arterial chemotherapy, has resulted in better chances of saving the eye compared to the usual method of administering chemotherapy through a vein. Melphalan damages the DNA of cancer cells, stopping them from growing and dividing. This targeted treatment may lead to better outcomes for retinoblastoma, particularly in challenging cases with cancer cells in the vitreous humor.⁶⁷⁸⁹¹⁰