UCSF establishes Pediatric Brain

Building on its foundation as a leading site for pediatric brain tumor research and care, UCSF has established a Pediatric Brain Tumor Institute devoted to understanding and developing new treatments for childhood brain tumors.

The Institute’s research will focus on the biology of pediatric brain tumors, which is not as well understood as that of brain tumors in adults. Because of this lack of knowledge, new therapies have been slow to develop, said Mitchel S. Berger, MD, chair of the Department of Neurological Surgery and Kathleen M. Plant distinguished professor, who will head the Institute.

“This Institute aims to address the challenge of understanding the biology of pediatric brain tumors and to provide new treatments to children with brain tumors by using innovative, biologically based strategies,” Berger said.

Sam Hawgood, MD, chair of the Department of Pediatrics and medical director for UCSF Children’s Hospital, said the Institute will keep UCSF Children’s Hospital at the forefront of innovative pediatric brain tumor care.  “The Institute will integrate advances in tumor genetics and stem cell biology with the well-established clinical programs in childhood cancer that we already provide.”

Childhood brain tumors are rare, but the mortality rate associated with them has surpassed acute lymphoblastic leukemia, and brain tumors are now the most common cause of cancer death among children. While approximately 60 percent of children with brain tumors survive at least five years, this figure has improved only slightly in the past quarter century.

The Institute is funded by a $300,000 grant over three years from the Pediatric Brain Tumor Foundation of the United States, a nonprofit organization devoted to finding causes and cures for childhood brain tumors, and matching funds from the UCSF Department of Neurological Surgery.

“As we move into this exciting phase of development, we are grateful for the opportunity to collaborate with the Pediatric Brain Tumor Foundation in expanding our pediatric brain tumor research and treatment program and making the future of brain tumor therapy for children more hopeful,” Berger said.

The Institute initially will focus on five main pediatric brain tumor projects, with emphasis on pediatric brainstem glioma and medulloblastoma, led by Berger as principal investigator. Department of Neurological Surgery investigators also will collaborate with other institutions, including the Pediatric Brain Tumor Foundation Institute at Duke University.

Brainstem gliomas are tumors located in the area that connects the spinal cord to the brain just above the back of the neck and may grow rapidly or slowly, depending on the grade of the tumor. Rapidly growing diffuse brainstem gliomas carry a very poor prognosis, and patients with this tumor type rarely survive longer than one year after being diagnosed. The first project will study the origin of pediatric brainstem gliomas by examining central nervous system development and neural stem cells. Another project will examine how to better deliver drugs to this area of the brain by studying convection-enhanced and intra-nasal delivery of therapeutic agents into the rodent brainstem.

A third project will investigate the role of genetics in the formation of pediatric medulloblastoma, specifically by examining the MYCN gene, which is involved in cell regulation. Medulloblastomas represent 15 to 20 percent of all pediatric brain tumors. They originate in the cerebellum and are fast-growing, invasive tumors that frequently spread to other parts of the central nervous system via the spinal fluid.

A fourth project will aim to develop a new therapy for pediatric brain tumors by targeting genes that regulate cell death. In cancer, alterations in these genes can cause them to function improperly, leading to a decrease in cell death and growth of a tumor. Investigators will develop short interfering RNA - a molecule that can interfere with the expression of genes ― that can specifically inhibit these genes and induce the death of tumor cells. In the final project, investigators will implant tumor cells derived from samples of human pediatric brain tumors into mice. This will create a much needed animal model of pediatric brain tumors that can be used to test new therapies and provide a renewable resource for other studies and the greater neuro-oncology community. 

In 1999 UCSF was chosen to join the National Cancer Institute-funded Pediatric Brain Tumor Consortium.  The UCSF team is committed to rapidly moving promising research into clinical trials and to creating an environment that focuses on the specific needs of children and their families who suffer from the effects of this devastating disease.

UCSF is a leading university that consistently defines health care worldwide by conducting advanced biomedical research, educating graduate students in the life sciences, and providing complex patient care.

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