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A Victory Over Kid's Cancer

Treatment with Immunotherapy Helps the Body's Own Immune System Finish Off Deadly Tumors

No medical problem is graver than cancer in children. But more youngsters are now surviving a deadly malignancy called neuroblastoma, thanks to a new immunotherapy treatment that a doctor at Maria Fareri Children's Hospital at Westchester Medical Center helped to pioneer. 

Neuroblastoma is the most common cancer during the first year of life, and it's responsible for 15 percent of all infants' cancer deaths. About 40 percent of neuroblastoma cases aren't discovered until they reach an advanced stage, and the cancer progresses very rapidly.

"It's a very bad disease," says M. Fevzi Ozkaynak, M.D., a pediatric hematologist and oncologist who is Director of the Bone-Marrow Transplant Program at the children's hospital. 

Diagnosis of advanced cancer

As vice chairman of a key clinical study of immunotherapy, Dr. Ozkaynak provided treatment that helped save the life of a neuroblastoma patient—8-year-old Brian Mullin of Stony Point. Today Brian is an active boy who loves baseball, pro wrestling and Star Wars. But when he was 18 months old, his parents noticed a bump on the right side of his head. Soon afterward, his left eye began to bulge, "like he was making goo-goo eyes at us," says his father, Chris.

An ophthalmologist found a mass growing behind his eye and suggested the Mullins see Dr. Ozkaynak. "He did a biopsy of the lump and said it was definitely malignant and most likely neuroblastoma," recalls Mullin. "And he said it was important that we act immediately." Brian was admitted to Westchester, underwent tests to confirm the diagnosis and started chemotherapy within two days.

"It came at us out of the blue," adds the boy's father.  "The scary part is when they tell you what might happen." 

Intensive surgery

Neuroblastoma is a cancer that forms in nerve tissue. The cause is a genetic mutation in the DNA, but what produces that mutation is unclear; it's not inherited from the parents. The cancer usually starts in the adrenal glands, which sit atop the kidneys, but may also originate in the neck, chest or spinal cord.

Neuroblastoma sometimes begins before a child is born, so that by the time symptoms develop, it has usually spread to other parts of the body. That was the case with Brian, whose cancer had reached stage 4, the most advanced stage.

If the disease is found early, in stage 1, a surgeon can remove the tumor and "that can be the end of treatment," says Dr. Ozkaynak.

Brian was not so lucky. He underwent a brutal regimen that began with the removal of some of his bone-marrow stem cells. "The bone marrow creates new red and white blood cells, but the chemotherapy treatment can wipe out the bone marrow," says the doctor. To protect them, the stem cells were frozen and held while Brian received three chemotherapy treatments. The treatments shrank the lesions on his head and behind his eye, along with others on his ribs and spine. He then had surgery to remove the main tumor on his adrenal gland.

Once recovered from surgery, Brian received a week of extremely high-dose chemotherapy. Then Dr. Ozkaynak thawed the stem cells and transfused them back into Brian's bone marrow so new blood cells could be grown. Brian was kept in a sterile isolation unit while his immune system recovered.

Brian's mother, Eileen, stayed with him throughout the treatment while Chris was home with their daughter, Linda, now 12. "Eileen had to wear gloves and masks the whole time, and she is very claustrophobic, so it was hard for her," says Mullin. "But if Brian was there, so was she."

New, experimental treatment

A month after this treatment, Brian started radiation therapy, which targeted remaining lesions in his stomach, chest and throat. During this time, Dr. Ozkaynak began administering the experimental treatment, immunotherapy, as part of a clinical study sponsored by the national Children's Oncology Group, an entity within the National Cancer Institute.

The study investigated whether introducing natural agents to help the body's immune system fight the cancer itself would benefit patients. Researchers across the country, including Dr. Ozkaynak, injected three substances into the subjects. The first was an antibody that attaches itself to the surface of the neuroblastoma cell and, in essence, provokes the body's immune system to attack it. The other two were natural hormones called cytokines, which boost the immune system's strength.

Brian was one of 226 patients in the study. After two years, 66 percent of those who underwent immunotherapy were alive with no sign of cancer, compared with 46 percent of those who didn't have the therapy.

The combined radiation and antibody therapy was rough, Mullin says. "It was bad. Brian couldn't eat or go to the bathroom. He lost so much weight, we couldn't believe how tiny he was. We thought about stopping. But we decided to give it one more day, then one more day, and eventually he got a little better."

A bright future

Today, Dr. Ozkaynak even uses a term for Brian's status that cancer specialists use very cautiously. "We wait five years from the initial diagnosis," he explains. "But Brian has passed that mark, so we can say he's cured."

Brian is still checked yearly, but he's no longer on medication. The only residual effect is that, because the chemotherapy may have affected his growth plates—the areas of tissue near the ends of bones where growth occurs—he may not grow as tall as Chris, who is 6'3". Says the relieved dad: "That's a price we'll gladly pay."


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