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New Approach for Treating Retinoblastoma

Novel therapy may ease stress of treatment for most common eye cancer in children

October 9, 2014 | Jessica Pasley

Jude Kee, mother Amy, and grandmother Ann Westerbeck

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.

Conventional therapy for patients diagnosed with retinoblastoma, the most common ocular cancer in children, includes systemic chemotherapy, external beam radiation and/or surgical removal of the eye.

Doctors at Vanderbilt University Medical Center are on track to radically change the way the disease is treated using an emerging therapeutic approach called intra-arterial chemotherapy, or IA chemo.

The procedure, performed on an outpatient basis, delivers chemotherapy directly to the tumor via the ophthalmic artery, using a catheter that is inserted into the groin and threaded to the eye under X-ray guidance.

In this way, 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.”

Eighteen-month-old Jude Kee, Vanderbilt’s first patient, is showing signs of improvement. After two treatments in both eyes, administered four weeks apart, the tumors are shrinking — indicators of the success of the novel therapy.

Initially concerned that Jude had lazy eye, his mother took him to the pediatrician who referred them to a specialist. She soon discovered that her son had bilateral retinoblastoma (tumors were present in both eyes).

Upon seeing Daniels, Jude’s parents were given the choice of following the traditional treatment route or becoming trailblazers for future pediatric patients at Vanderbilt.

Amy Kee was familiar with the newest therapeutic option via a family friend dealing with the same diagnosis. She was aware that Daniels had come to Vanderbilt to offer IA Chemo.

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

Anthony Daniels, M.D., and  Joshua Carlson, M.D.

Anthony Daniels, M.D., right, examines Jude Kee’s eyes while Joshua Carlson, M.D., watches the monitor.

“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.”

Vanderbilt, the only center offering IA chemo in the region, is one of just a few centers in the country offering the novel therapy.

Daniels, along with neurointerventionalist Michael Froehler, M.D., Ph.D., are able to offer the full spectrum of treatment for patients.

Patients are referred to Daniels, who makes the initial diagnosis and develops a treatment plan based on the classification of the tumor as well as the specific needs of the child.

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.

Daniels and his team work with pediatric oncologists at Monroe Carell Jr Children’s Hospital at Vanderbilt to ensure the patients are closely monitored post treatment.

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 Children’s Hospital, Froehler 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.

Jude’s next examination and IA treatment are scheduled for Oct. 20 and 23.

Photo(s) By: Photos by Anne Rayner