Nine cancer centers share $8.9 million grant to improve treatment for neuroblastoma, a cancer that strikes children

June 11, 2000

With an $8.9 million grant from the National Cancer Institute, nine institutions 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 grant to develop those new therapies is Robert C. Seeger, MD, of Childrens Hospital Los Angeles and the University of Southern California. UCSF pediatric oncologist Katherine K. Matthay, MD, is principal investigator of the clinical consortium, New Approaches to Neuroblastoma Therapy (NANT), which aims to 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 the Children's Medical Center at UCSF, 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," said Matthay. "That is why we formed this new consortium."

Seeger, who is professor of hematology/oncology and deputy director of research at Childrens 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 Childrens Hospital Los Angeles (CHLA), the Indiana University 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 be applicable to other childhood and adult malignancies as well.
The nine NANT institutions are: Childrens Hospital Los Angeles/USC; the Children's Medical Center/UCSF; Lucile Packard Children's Hospital/Stanford University; the University of Wisconsin; the University of Michigan; Cincinnati Children's Hospital; the University of Minnesota; the Indiana University, and Children's Hospital of Philadelphia/University of Pennsylvania.

The Children's Medical Center at UCSF has been providing 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.

Reporter's Note: Matthay is on sabbatical but may be reached by phone or e-mail: For details contact Janet Basu at 415-476-2557 or

Seeger will be available via a satellite press conference on July 13 from 8:30 to 10 a.m. For details contact Steve Rutledge at CHLA, 323-669-4121

For more information on clinical trials for neuroblastoma at UCSF, consult and

For more information about NANT consult

For more information about children's cancer centers and clinical trials, consult the National Cancer Institute website at:

Katherine K. Matthay, MD -- background

Katherine K. Matthay, MD is UCSF professor in residence of pediatrics, chief of pediatric oncology and director of the pediatric oncology program at the Children's Medical Center at UCSF. Her work focuses on clinical research using cell-specific treatments, particularly in treatment of neuroblastoma.

She is chair of the Neuroblastoma Strategy Group in the Children's Cancer Group, a National Cancer Institute - directed organization of university and affiliate medical centers dedicated to clinical trials for the study and therapy of childhood cancer. She is principal investigator of a nine-university consortium, New Approaches to Neuroblastoma Therapy (NANT), which aims to bring to the bedside the most promising strategies to improve the outcome of children with this disease.

At UCSF, Matthay and her research group study treatments for children with progressive neuroblastoma, including the use of [131 I]-MIBG, targeted radiotherapy. She led the nationwide Children's Cancer Group Phase III clinical trials that in 1999 reported the success of autologous bone marrow transplantation plus 13-cis retinoic acid as adjuvant therapies with a three-fold improvement in disease free survival rates for children with high-risk neuroblastoma.

Matthay continues to participate in research on new methods of bone marrow transplant using purged marrow or peripheral blood stem cells. She participates in other innovative Phase I pilot studies for neuroblastoma treatment and in collaborative studies with basic research laboratories on the molecular genetics and immunology of neuroblastoma biology.

Matthay earned her degree in medicine from the University of Pennsylvania. She served as a fellow and postgraduate research fellow in pediatric hematology-oncology at UCSF and joined the UCSF faculty in 1980.

For more information consult

University of California - San Francisco

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