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Newly approved drug boosts pediatric cancer survival rates


Jack is being treated with a newly approved medication that has shown to boost survival rates by 50 percent. This medication -- Unituxin -- was available to Upstate pediatric patients during the clinical trial phase. (PHOTO COURTESY OF THE BRICKMAN-DONNELLY FAMILY)

When Jessica Brickman of Camillus put her baby, Jack, to sleep that Friday last fall, the 9-month-old was just as jolly and happy as always. The little guy awoke with black circles around his eyes, clearly not feeling well, and throwing up.

Emergency pediatricians at the Upstate Golisano Children‘s Hospital explored whether Jack had fallen or been injured. A computerized tomography scan revealed tumors on his skull, and one of the doctors prepared Brickman and Jack‘s father, Robert Donnelly, for the news they would hear later that day.

Jack had an advanced form of neuroblastoma, the most common cancer in infancy, which affects about 700 babies in the United States each year. Pediatric oncologists at Upstate cared for 15 children with the diagnosis between January 2010 and February 2015.

Jack‘s tumors were classified as high risk, unusual for an infant under 18 months of age. It would require aggressive treatment in order to improve his chance for a cure. Babies with low-risk tumors have high cure rates.

Pediatric oncologist Irene Cherrick, MD (PHOTO BY CINDY BELL)

Pediatric oncologist Irene Cherrick, MD (PHOTO BY CINDY BELL)

Treatment started the next day for Jack. He was hospitalized for 10 days, receiving the first of six rounds of chemotherapy. He went home on his big sister, Alyssa‘s, second birthday. After additional chemotherapy, Jack had stem cells removed and frozen to be returned to his body later. He underwent surgery to remove his tumors. Then his parents took him to Philadelphia for three weeks for specialized radiation therapy before returning to Upstate for the stem cell transplant. Beginning the week of Memorial Day, Jack started immunotherapy.

The boy, now 18 months old, receives a continuous infusion of a drug called Unituxin, which will help his body‘s immune system find and destroy any remaining cancer cells. The infusion lasts from 10 to 20 hours over four consecutive days, and it‘s painful. But Unituxin has become part of the standard treatment for neuroblastoma since researchers showed that children with high-risk tumors who received the drug had survival rates of 66 percent, compared with rates of 46 percent among babies who did not.

As soon as researchers realized how effective the drug was, the international trial involving 1,200 children was halted, and all of the children received Unituxin.

“Unituxin basically increased the cure rate by 50 percent. That‘s huge,” said Upstate‘s Irene Cherrick, MD, one of the pediatric oncologists who celebrated the approval of the drug in March. It‘s only the third drug approved by the FDA specifically for the treatment of cancer in children. The medication has been available to patients at Upstate Golisano Children‘s Hospital through the clinical trial.

As revolutionary as the drug is, it does not replace the treatment regimen for neuroblastoma – and it does not cure everyone.

Jack‘s parents remain hopeful. “If it works the way it‘s supposed to,” Brickman said, “it will make him better in the long run.”

This article appears in the summer 2015 issue of Cancer Care magazine.