NEWS | August 21, 2012

St. Baldrick's Foundation awards $100,000 to UC Davis scientist


For the second consecutive year, Paul Knoepfler, associate professor of cell biology and human anatomy at UC Davis School of Medicine, has been awarded a $100,000 grant from the St. Baldrick's Foundation to pursue his novel research into the molecular causes of brain tumors in children.

St. Baldrick's, which is known for its annual head-shaving event fundraisers, is a nonprofit organization dedicated to supporting research into childhood cancers.

Paul Knoepfler © UC Regents
Paul Knoepfler

Knoepfler's research includes a focus on the most common type of pediatric brain tumors: medulloblastomas. The tumors are more prevalent in children under the age of 5, and are more often found in males than females. Current treatments remain limited and often have toxic side effects in young patients, including lifelong cognitive impairment. Researchers suspect that these brain tumors, as well as other kinds of pediatric cancers outside of the brain, are caused by mutations in a gene called the N-Myc. They suspect an excess of N-Myc can alter the DNA structure in the brain's normal stem cells causing them to become cancerous.

"Until we better understand N-Myc and its role in medulloblastomas, clinicians will struggle against this terrible cancer and pediatric patients will continue to suffer," said Knoepfler, who also is an associate investigator with the Institute for Pediatric Regenerative Medicine at Shriners Hospital for Children-Northern California. "Thanks to support from St. Baldrick's, I'm working on the idea that N-Myc mutations cause cancerous stem cells to form and function like bad seeds that give rise to tumors. My hope is that our findings will help provide a foundation for developing a safe and effective future treatment."

The research involves what are called primitive neuroectodermal tumors (PNETs), of which medulloblastomas are one type. Because N-Myc is implicated in all PNETs, including pediatric tumors of the brain, eye (retina) and kidney (Wilms tumor), Knoepfler's studies could have extremely important clinical implications.

Medulloblastomas, while rare, are responsible for up to 25 percent of all pediatric brain cancers, according to National Cancer Institute. About 500 cases around the nation are diagnosed annually. The tumors occur in a part of the brain (cerebellum) that controls balance and other complex motor functions.

Knoepfler specializes in regenerative medicine and cancer-related research. Some of his work focuses on understanding how stem cells are programmed and how that programming can go awry, thereby causing birth defects or cancer. He specifically has analyzed the epigenetic changes in stem cells -- the mechanisms, apart from mutations, by which environment influences gene expression and may lead to tumors or birth defects.

About the St. Baldrick's Foundation
The St. Baldrick's Foundation is a volunteer-driven charity committed to funding the most promising research to find cures for childhood cancers and give survivors long and healthy lives. The Foundation funds more in childhood cancer research grants than any organization except the U.S. government. Since the Foundation's first grants as an independent charity in 2005, St. Baldrick's has funded more than $101 million in childhood cancer research. For more information, please call 1.888.899.BALD or visit

About UC Davis School of Medicine
UC Davis School of Medicine is among the nation's leading medical schools, recognized for its research and primary-care programs. The school offers fully accredited master's degree programs in public health and in informatics, and its combined M.D.-Ph.D. program is training the next generation of physician-scientists to conduct high-impact research and translate discoveries into better clinical care. Along with being a recognized leader in medical research, the school is committed to serving underserved communities and advancing rural health. For more information, visit UC Davis School of Medicine at