New Investigational Treatment Approach for Rare Childhood Eye Cancer

Recently, David H. Abramson, Chief of the Ophthalmic Oncology Service at Memorial Sloan-Kettering, and Pierre Gobin, a NewYork-Presbyterian Hospital radiologist, have introduced a new treatment for certain patients in which high doses of chemotherapy are injected into the artery that leads directly to the affected eye, in a process known as intra-arterial chemotherapy. (For most retinoblastoma patients, chemotherapy is delivered either intravenously into the bloodstream, where it travels throughout the body, or periocularly into the area around the eye.)

The new approach, which was first used in spring 2006, seeks to allow safe, repeated intra-arterial delivery of the chemotherapeutic agent melphalan (Alkeran®) to the eye, with the aim of destroying the retinoblastoma cells, while minimizing side effects and eventually restoring vision in the affected eye. In the procedure, a long, thin tube known as a microcatheter is inserted into an artery in the child's groin and threaded through the body until it reaches the artery leading to the eye containing the retinoblastoma tumor. Melphalan is injected into the tube and delivered directly into the eye.

While standard intravenous chemotherapy can also affect other non-cancerous tissues, the intra-arterial method allows higher doses to be delivered precisely to the tumor, sparing healthy tissue. Most patients in the trial require two to three treatments of melphalan, which is normally not used intravenously to treat children because of its toxic effect on a child's bone marrow. "This approach allows us to give powerful medications to the tumor without delivering toxic doses to the patient," Dr. Abramson notes.

To date, ten patients have received the procedure, which Dr. Abramson and Dr. Gobin are calling chemosurgery, reflecting the technique's surgically precise use of chemotherapy. Early results suggest that it may be more effective than standard chemotherapy, while also helping to avoid some of the side effects of intravenous chemotherapy. Dr. Abramson cautions that further testing is required before any conclusions can be drawn, though the initial findings are grounds for guarded optimism.

"If these trials are successful, intra-arterial chemotherapy may replace enucleation for the majority of retinoblastoma cases," Dr. Abramson says. "That would be a revolutionary improvement in the treatment of this disease."