New treatment for retinoblastoma expected to save more eyes from cancer

A new technique has been developed to treat the eye cancer retinoblastoma and improve the odds for preventing eye loss, blindness, or death in children with advanced forms of the disease.

Treatments for retinoblastoma have progressed dramatically in recent years. One is a procedure called ophthalmic artery infusion chemotherapy. A tiny catheter is inserted into an artery that provides blood flow (and chemotherapy) directly to the eye and tumor. Originally introduced in the late 1980s, direct ophthalmic artery infusion significantly increases treatment effectiveness while reducing side effects.

Unfortunately, many children with retinoblastoma are not good candidates for conventional ophthalmic artery infusion. This applies in to particular younger, smaller patients with advanced disease, according to Todd Abruzzo, MD, director of Interventional Neuroradiology at Cincinnati Children's Hospital Medical Center in Ohio.

“The catheters are so large compared to the smaller arteries of the child that they restrict blood flow to the eye, causing back pressure that pushes blood flow and chemotherapy away from the eye and tumor,” Abruzzo said. “Unfortunately, for too many of these children there is no option other than enucleation, or loss of the eye. You can imagine what that means for a child.”

Four-year-old Khloe Cline is one of these children. Her case was especially challenging because she had advanced cancer in both eyes (bilateral retinoblastoma). When she developed retinoblastoma at age 18 months, doctors in her hometown of Indianapolis, Indiana, identified 11 tumors in both eyes.

After an unsuccessful attempt with conventional chemotherapy, Khloe's physicians referred her to Cincinnati Children's, where researchers have been testing an innovation to ophthalmic artery infusion chemotherapy.

Working with physicians in the Cancer and Blood Diseases Institute (CBDI) at Cincinnati Children's, Abruzzo developed a double-catheter infusion technique that involves inflating a tiny balloon in the external carotid artery to prevent backpressure and ensure blood and chemotherapy flow to the eye and tumor. He further improved the technique by administering verapamil, a drug that increases the flow of chemotherapy to the tumor and helps block the tumor's ability to pump chemotherapy away before it does its job.

The result is a safe, effective, and reproducible method for delivering chemotherapy treatment to the eye, especially in patients with advanced retinoblastoma who are not candidates for conventional infusion therapy, according to the study (Journal of Neurointerventional Surgery. 2014; doi:10.1136/neurintsurg-2014-011295). Of 19 eyes (17 patients) treated in the study, 11 eyes were saved. 

After undergoing a series of treatments at Cincinnati Children's with the new procedure, Khloe's mother, Alicia Gray, credits the physicians with saving her daughter's eyes and allowing her to retain functional eyesight. And although Khloe's eyes have been scarred and her vision is not perfect, she carries on like any normal energetic 4-year-old, with a penchant for learning and a strong desire to be a doctor when she grows up.

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