UCSF Turns to Targeted Treatments to Boost Brain Tumor Survival in Children

By Suzanne Leigh, UCSF Science Cafe | May 18, 2010

Targeted therapies tailored to the unique biology of brain tumors may offer renewed hope to children diagnosed with the number one cause of cancer death, according to faculty at UCSF Children's Hospital.

An estimated 4,030 new cases of brain and central nervous system tumors in children are expected to be diagnosed this year, according to the Central Brain Tumor Registry of the United States. And 32 percent of children die within 10 years of diagnosis, a rate that is unacceptably high, say the doctors who treat them.

Speaking at a reception hosted by the Pediatric Brain Tumor Consortium (PBTC) Foundation in San Francisco on May 6, UCSF radiation oncologist Daphne Haas-Kogan, MD, said that a new clinical trial is pending approval to test a targeted treatment for children with low-grade glioma, slow-growing tumors arising from glial cells, which can be resistant to surgery, radiation and chemotherapy.

This new generation of cancer treatments, which offers the promise of "personalized medicine," differs from standard chemotherapeutic agents, which have been prescribed for decades and work by indiscriminately destroying dividing cells, healthy and malignant alike. In contrast, the targeted drugs are designed to strike the pathways of aberrant genes in specific cancer cells.

The trial, which is funded by the PBTC, a network created by the National Cancer Institute in 1999 dedicated to developing advanced treatments for young patients, follows the discovery of genetic abnormalities in pediatric brain tumors. These include abnormal activation of certain pathways and a particular genetic aberration in a protein called BRAF, the focus of research carried out by Haas-Kogan, C. David James, PhD, and colleagues.

"This is the first real breakthrough in understanding the biology of this glioma," said pediatric neuro-oncologist Anu Banerjee, MD, whose research has included the direct delivery of new drugs to tumor in patients with high-grade glioma. "Hopefully it will illuminate new treatment opportunities that would be more effective and less morbid than current treatment of radiation and chemotherapy."

Read more at Suzanne Leigh, UCSF Science Cafe