Novel therapy eases stress of retinoblastoma treatment

From the Winter 2015 edition of Vanderbilt Medicine Magazine

Amy Kee plays with her son, Jude, as his grandmother, Ann Westerbeck, looks on. Jude is undergoing a new therapy at Vanderbilt to treat his retinoblastoma.   Photo by Anne Rayner.
Amy Kee plays with her son, Jude, as his grandmother, Ann Westerbeck, looks on. Jude is undergoing a new therapy at Vanderbilt to treat his retinoblastoma. Photo by Anne Rayner.

Doctors at Vanderbilt are on track to radically change the way retinoblastoma is treated using an approach that delivers chemotherapy directly to the tumor via the ophthalmic artery.

This novel approach, known as IA chemo, uses a catheter that is inserted into the groin and threaded to the eye under X-ray guidance. IA chemo is able to limit the adverse effects typically associated with systemic chemotherapy.

“It is transformative,” said Anthony Daniels, M.D., assistant professor of Ophthalmology and Visual Sciences at the Vanderbilt Eye Institute. “We are able to tailor this treatment to each individual patient and each tumor. It is our expectation that this will become the primary modality of treatment of retinoblastoma at Vanderbilt.”

Vanderbilt’s first patient was 18-month-old Jude Kee, who had bilateral retinoblastoma (tumors in both eyes). After two treatments, administered four weeks apart, his tumors are shrinking—indicators of the success of the novel therapy.

Daniels gave Jude’s parents the choice of following the traditional treatment route which includes systemic chemotherapy, external beam radiation and/or surgical removal of the eye or becoming trailblazers for future pediatric patients at Vanderbilt.

“We feel very fortunate to have this treatment available at Vanderbilt,” said Jude’s mother, Amy Kee. “It is phenomenal. Jude has not been limited whatsoever and that is a huge blessing.

“We will come out on the other side of this just fine. He is able to go to school and be normal. We have not had any worries about his immune system being suppressed or any of the other issues typically associated with systemic chemo.

“I have read about the effects of chemo. I have read the stories about children having their eye or eyes removed. I know that we are extremely fortunate to have Dr. Daniels right here offering IA.”

“We feel very fortunate to have this treatment available at Vanderbilt. It is phenomenal. Jude has not been limited whatsoever and that is a huge blessing.”

Vanderbilt is one of just a few centers in the country offering the novel therapy.

While traditional chemotherapy (systemic delivery) has its uses in certain circumstances, the only patients who are not considered candidates for IA chemo are those whose tumors are so small that they can be treated with laser or cryotherapy alone, or if the tumor has destroyed the eye, requiring removal.

On average, patients undergo at least three treatments to fully destroy the tumor.

“The other amazing thing about IA chemotherapy is that it has the ability to prevent new retinoblastoma tumors from forming elsewhere in the eye,” Daniels said. “Historically, we would kill the tumor, only to have new tumors pop up elsewhere in the eye. With IA chemotherapy the patient almost never gets another new tumor in the eye because any early cancer cells that are present are killed before they can grow.”

Performed in the angiography suite at Monroe Carell Jr. Children’s Hospital, Michael Froehler, M.D., Ph.D., uses the same technique used to treat brain aneurysms.

He inserts a tiny catheter into the patient’s groin and threads it to the arterial branch leading to the tumor in the eye. He then injects the chemotherapy cocktail prescribed by Daniels.

The technique allows for a high concentration of chemotherapy to kill the tumor while limiting the toxic effects on the rest of the body.

“The endovascular approach allows the treatment to be specifically tailored to each unique tumor and eye within the same patient,” Froehler said. “Sometimes the arterial supply to one eye is different from the other, and frequently the tumor on one side is bigger or more severe than the other.

“By recognizing these differences, we can individualize the catheter placement and IA chemo regimen to optimize treatment effect.”

The Kees are hopeful that IA chemo will open a new door for children diagnosed with retinoblastoma.

“We have had huge success so far,” Kee said. “I am hopeful that we will not need to do any other kinds of treatments.

“I just keep going back to the fact that if I had not noticed that he had lazy eye, we may not have caught it in time. Not only would the possibility of removing his eye been much greater, but the cancer could have gone to his brain.

“This treatment has made a huge, huge difference. The entire center of one of his eyes has opened up and they think that he may be able to see a little bit out of that eye now,” Kee said.