Cora Tucker was 2 1/2 when she was diagnosed in March 2011 with high-risk neuroblastoma, a rare cancer of the nervous system that typically occurs in children younger than 5.
Her parents were given a choice. She could have the standard therapy – surgery, chemotherapy, radiation and a stem cell transplant. Or they could enroll her in a clinical trial that added a new drug to that regimen.
They chose the trial, available through Dr. Andrew Gilman at Levine Children’s Hospital in Charlotte. Cora’s treatment lasted a year and a half.
“It was tough, but she responded to everything they gave her,” Candice Tucker said of her daughter, now 6. “She’s currently clean. She’s a survivor.”
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The drug showed such significant benefit that the clinical trial, with 226 patients from multiple U.S. medical centers, was stopped early. In March, the Food and Drug Administration approved the drug, Unituxin, for treatment of high-risk neuroblastoma.
Gilman, director of the pediatric blood and marrow transplant program at Levine Children’s, had been working toward this day since the mid-1990s when, at the University of Iowa, he designed the initial trial of the antibody that would become Unituxin.
For the latest trial, he was co-principal investigator with a San Diego doctor, and Gilman enrolled about 10 patients from the Charlotte area. He had also been principal investigator for an earlier study to determine a safe dose of the drug.
To see it coming to fruition is very exciting.
Dr. Andrew Gilman, researcher at Levine Children’s Hospital
Neuroblastoma is a rare cancer that forms from immature nerve cells, usually in the adrenal glands but sometimes in the abdomen, chest or nerve tissue near the spine. About a third of patients are diagnosed early enough to have tumors removed surgically, followed by chemotherapy. They have a “high survival rate,” Gilman said.
But the cancer is often diagnosed late because symptoms are subtle. A growing tumor can lead to a distended stomach. Or tumors in the chest could cause coughing and wheezing.
Being diagnosed with “high-risk” neuroblastoma means the cancer has metastasized, or spread to other organs. Standard treatment is aggressive, and many patients have a recurrence anyway. The tumors can be difficult to remove surgically because they might have wrapped around the spinal cord or large blood vessels.
Thirty years ago, the number of children who survived high-risk neuroblastoma was zero, Gilman said. Over the years, with improvements in therapy, the survival rate reached 40 percent. But the Unituxin trial showed that adding this drug improved survival even more.
Patients enrolled in the trial were randomly assigned to get the standard therapy or the standard plus Unituxin. Four years later, 59 percent survived in the former group. But 74 percent survived in the group that got Unituxin.
The new drug is a targeted immunotherapy, one of many such drugs that have come to market in recent years. It stimulates patients’ immune systems to destroy cancer cells without harming healthy cells nearby.
“This whole notion that the immune system would be used to kill cancer cells has existed for decades,” Gilman said. But it took years for researchers to learn enough about the workings of the immune system to make therapies possible.
“People have been dedicating their careers to this,” Gilman said. “To see it coming to fruition is very exciting.”