UCSF Part of New Consortium on Children's Cancer

July 12, 2000
News Office: Janet Basu (415) 502-6397

With an $8.9 million grant from the National Cancer Institute, nine institutions -- including UCSF -- have joined forces to develop and test new treatments for neuroblastoma, an aggressive cancer that only strikes children.

The principal investigator of the NCI project program to develop those new therapies is Dr. Robert C. Seeger of Children's Hospital Los Angeles and the University of Southern California. Dr. Katherine K. Matthay, a pediatric oncologist at UCSF Medical Center, is principal investigator of the clinical consortium, New Approaches to Neuroblastoma Therapy (NANT), which will bring to the bedside the most promising strategies to improve the outcome of children with this disease.

Neuroblastoma is the most common cancer among infants and the second most common solid tumor malignancy in children under five. At the time they are diagnosed, 45 percent of affected children have high-risk disease, with the cancer often spreading throughout the body. A nationwide Phase III clinical trial led by Matthay, who is director of pediatric clinical oncology at UCSF Children's Health Services, last year showed a three-fold improvement in disease-free survival from high-risk neuroblastoma due to a new combination of therapies. But even with this improvement, only 40 percent of children with the most dangerous form of neuroblastoma survive for five years without a recurrence.

"In spite of the best therapies we have available, neuroblastoma recurs in many children affected by it, and those children have a poor prognosis," Matthay said. "That is why we formed this new consortium."

Seeger, a professor of hematology/oncology and deputy director of research at Children's Hospital Los Angeles and the Department of Pediatrics at the Keck School of Medicine at the University of Southern California, said that the NCI project program grant will allow investigators at the nine institutions to share research information and potentially speed the development of strategies that will enhance the effectiveness of current therapies. "Our goal is to improve disease-free survival for 90 percent or more of children with neuroblastoma," Seeger said.

Four different laboratory-based projects will investigate the promise of different strategies to stop tumors and their metastases, by targeting drugs to neuroblastoma tumors and by enhancing the drugs' effectiveness at killing even those cancer cells that are resistant to other drugs. Those projects will be led by investigators at Children's Hospital Los Angeles (CHLA), the University of Indiana and the University of Southern California.

The fifth major project of the study, led by Matthay, will translate the most promising of these strategies into small-scale Phase I - II clinical trials, offered to children in the context of the best current treatment for their stage of neuroblastoma. If these steps show that a strategy can be delivered safely and show evidence that it will be effective, the method will be offered to the nationwide NCI-sponsored children's cancer research networks for large-scale Phase III clinical trials.

Such large-scale nationwide trials, with many institutions working together, are credited with major advances against childhood cancer in the past 25 years. Some children's cancers have high cure rates and treatments for others have improved. Seeger, Matthay and their colleagues expect that the NANT project will speed the process of offering treatments for this particularly recalcitrant form of cancer and may apply to other childhood and adult malignancies as well.

The nine institutions participating in this study are: Children's Hospital Los Angeles/USC; Children's Health Services at UCSF Medical Center; Lucile Packard Children's Hospital/Stanford University; the University of Wisconsin; the University of Michigan; Cincinnati Children's Hospital; the University of Minnesota; the University of Indiana; and Children's Hospital of Philadelphia/University of Pennsylvania.

Children's Health Services at UCSF Medical Center has provided specialized care to children since 1913. Children come to UCSF from throughout the western United States and around the world for expert treatment of cancer; heart defects, other congenital anomalies and other major diseases.