Phase I clinical trials offer new avenue for pediatric cancer patients

July 17, 2007

Patients who suffer from recurrence of childhood cancer now have another option. The New Therapies for Pediatric Cancer Program at Comer Children's Hospital makes Phase I clinical trials available to youths with certain refractory or relapsed cancer.

"Ultimately, these trials will deliver new pediatric cancer treatments more quickly," said Mark Ratain, MD, Leon O. Jacobson Professor of Medicine and director of the advanced solid tumor clinic at the University of Chicago.

The New Therapies Program works closely with the adult cancer research program--monitoring the effectiveness the pharmacokinetics and toxicity of the drugs to determine the best dosages for young patients.

"By working so closely with the University of Chicago's well-established adult cancer treatment programs, we can fast track the access children have to new agents," said Susan Cohn, MD, director of the program and section chief of clinical sciences at the University of Chicago Institute for Molecular Pediatric Sciences.

Although researchers have made outstanding advances in the last four decades, 30 percent of children with cancer die from the disease. In the past, children have not had access to cancer drugs in Phase I clinical trials. This new program begins with candidates ages 14 and older or weighing at least 45 kilograms.

"Within the program, scientists from varied backgrounds are thinking collaboratively about curing childhood cancer," said John Cunningham, section chief of hematology/oncology at the University of Chicago Comer Children's Hospital. "We're able to test new drugs and deliver them to the pediatric population several years faster than we could before."

Through this collaboration, physicians and scientists compare pediatric and adult populations and observe similarities and differences. With carefully designed Phase I clinical trials, they begin with small, effective doses and gradually increase to determine the correct dosage and to observe the toxicity of new therapies.

"Patients in Phase I clinical studies are the first to potentially benefit from a new treatment," said Cohn, a leading expert in the study and treatment of neuroblastoma, a malignant solid tumor that forms in the nervous system outside the brain. "They also receive close monitoring by experts in the field."

These test are particularly important to patients with high-risk neuroblastoma. One of the deadliest pediatric cancers, neuroblastoma has a cure rate of only 30 percent.

Physicians who treat neuroblastoma must take many factors into account: location of the tumor, its size, whether or not the patient was treated with other therapies in the past and the type of therapies. Each treatment plan is tailored to the patient's individual circumstances.

Numerous Phase I trials for pediatric patients currently are open. For patients with refractory or relapsed neuroblastoma, UCMC researchers are working as part of the New Approaches to Neuroblastoma Treatment (NANT) consortium, a network of 13 hospitals conducting studies to test therapies for this deadly disease. The University of Chicago Comer Children's Hospital is the only Chicago institution in NANT.

The other two trials study drugs to treat solid tumors. These studies began with the adult population and have been expanded to offer the agents to adolescents.

"My hope is that after we demonstrate drug safety for the teen population, we can use these medications for a younger population," Cohn said. "We are fortunate to have such a well-developed, well-respected adult program. There can be great synergy in building pediatric and adult programs together."

The National Cancer Institute sponsors NANT. Other medical institutions participating in the NANT study include C.S. Mott Children's Hospital at the University of Michigan, Children's Healthcare of Atlanta, Children's Hospital and Regional Medical Center in Seattle, Children's Hospital Boston's Dana-Farber Cancer Institute, Children's Hospital Los Angeles, Children's Hospital Medical Center in Cincinnati, Children's Hospital of Philadelphia, Lucile Packard Children's Hospital, Riley Hospital for Children at Indiana University, Texas Children's Cancer Center at Baylor College of Medicine, University of California at San Francisco, University of Wisconsin Comprehensive Cancer Center.

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