The past century has seen great strides in the treatment of retinoblastoma, with more than 95% of US patients now being cured of this rare childhood malignancy. Building on this success, ophthalmologists have sought to minimize the adverse effects from radiation and systemic chemotherapy, hoping not only to cure the disease but also to save the eye and preserve vision.
Radiation can achieve this end, but it also can give rise to secondary cancers. And while systemic chemotherapy—which replaced radiation in the 1990s as first-line treatment for retinoblastoma—is effective in shrinking tumors, it has many adverse effects, including hearing impairment, development of second cancers, and long-term fertility issues. Systemic chemotherapy also does not fully eradicate the tumor, necessitating additional focal techniques such as laser therapy or cryotherapy that require the patient to undergo numerous examinations, procedures, and treatments.