Battling Rare Childhood Eye Cancer

Ruzanna Harutyunyan's picture

Retinoblastoma is a rare cancer that develops in the retina of the eye and mostly affects young children. According to the National Cancer Institute, about 300 children are diagnosed with the disease each year in the United States.

In fact, the vast majority of cases of retinoblastoma occur among young children, with almost two-thirds of all retinoblastomas occurring before the age of two years and 95 percent occurring before the age of five years. Now, Jefferson Hospital for Neuroscience (JHN) and Wills Eye Institute are offering these young patients a new, targeted treatment option that can save their life and help save their sight and eyes.


Intra-arterial chemotherapy is a novel technique that delivers high doses of chemotherapy directly into the artery that leads to the affected eye. This therapy allows for a repeated delivery of the chemotherapeutic agents, with the aim of destroying the retinoblastoma cells, while minimizing side effects associated with more traditional intravenous (IV) chemotherapy.

For most retinoblastoma patients, chemotherapy is delivered either through an IV, where it travels throughout the body and successfully eradicates the cancer but can cause side effects like hair loss or more serious effects like deafness, kidney failure or leukemia. Unfortunately, surgical removal of the affected eye (enucleation) is often necessary for children with advanced retinoblastoma to ensure safe life prognosis.

But more recently, intra-arterial chemotherapy has been pioneered by the Philadelphia team at Jefferson Hospital for Neuroscience (JHN) and Wills Eye Institute. Carol Shields, M.D., an eye cancer specialist at Wills Eye Institute, has teamed up with Robert Rosenwasser, M.D., chair of the Department of Neurological Surgery at Jefferson Medical College of Thomas Jefferson University, to treat these patients with this new, targeted therapy. First the child is evaluated by Dr. Shields and the diagnosis of retinoblastoma is established and evaluation of blood flow to the eye is studied. Next, Dr. Rosenwasser inserts a long, thin tube known as a microcatheter into an artery in the child's body and threads it up until it reaches the artery leading to the eye containing the retinoblastoma cancer. Chemotherapy agents are then injected into the tube and delivered directly into the eye. Later, reassessment with laser treatment to the cancer is performed by Dr. Shields.

“Intra-arterial chemotherapy is a promising new technique for treatment of selective cases of retinoblastoma and allows for a higher local dose of chemotherapy to the eye with minimal side effects to the body as compared to the more traditional intravenous method,” said Dr. Carol Shields. “This high dose of chemotherapy delivered to the eye accelerates regression of the tumor, without adversely affecting other healthy organs. While the long term outcome of these patients is still unknown, this new delivery system may reduce the need for the surgical removal of some patients’ eyes.”