The Children's Hospital Enrolls First Child in Trial for Rare Cancer
from The Children's Hospital (TCH) News, February 2006
The Children’s Hospital was the first in the world to enroll a pediatric patient with a rare type of leukemia in a new clinical drug trial, said Lia Gore, MD, director of Children’s Experimental Therapeutics Program.
In fact, of the five children in the world who are enrolled in the trial, two are at Children’s. The protocol uses the drug forodesine, which is used to treat leukemia and lymphoma in adults, to treat B-cell lineage leukemia in children.
“This is the first time this drug has been used to treat this type of leukemia in children,” Dr. Gore said.
The trial is one of 14 currently underway at Children’s for patients who have undergone chemotherapy to treat their cancers but did not respond well to treatment. For example, Dr. Gore said, nationwide, 2,500 children a year are diagnosed with Acute Lymphoblastic Leukemia (ALL), the most common of all pediatric cancers. Eighty percent are cured the first time around with chemotherapy; the rest relapsed. Patients who relapse have several options, including seeking more treatment.
Children’s sees roughly 30 to 40 pediatric patients a year – from as far away as the Czech Republic – who have relapsed and are seeking further treatment. Of that number, 20 to 30 enter a clinical trial.
The reasons for the low numbers of patients who enroll in these experimental trials, Dr. Gore said, are many.
“Sometimes patients’ bodies are too worn out for another round of drugs,” she said. “Also, for many years, these types of trials weren’t offered, so patients may not know they exist.” In fact, she said, just three years ago, there were no such “second-round” trials. And no wonder: It takes a principal investigator 200 to 300 hours just to write a trial. It takes another 6 to 18 months for each trial to get approval.
This particular trial – piloted at Children’s – has now expanded to 10 other institutions across the country to ensure that enough patients are enrolled to get meaningful conclusions. It is one of several trials of a new generation of “sophisticated science” drugs.
“Typically, children are treated with toxic, aggressive chemotherapy,” Dr. Gore said. “The chemotherapy is a combination of several aggressive inpatient treatments that have a lot of side effects. With some of these new drugs, kids are treated as outpatients. They keep all their hair. They don’t have side effects. There’s no way you would think they were sick. The disease is controlled, and, at the same time, the child has a great quality of life.
“Most chemotherapy is a bazooka-gun theory. It kills all cells, including healthy ones, which is why patients have so many side effects. These molecular-targeted therapies can target the cancer cells but not the healthy cells.”
The addition of such trials – half of which Dr. Gore has written – has positioned Children’s as a player among the world’s best cancer centers, she said.
“We are truly a comprehensive cancer center, and we’re helping to develop the next generation of leukemia trials and new approaches to cancer therapy and pediatric cancer research,” she said.
