$100,000 awarded to UC Davis stem cell researcher

Investigate molecular causes of brain tumors in children

Sacramento, Calif.- Paul Knoepfler, an associate professor of cell biology and human anatomy at UC Davis School of Medicine, has received a $100,000 grant from the St. Baldrick’s Foundation to investigate the molecular causes of brain tumors in children.

The foundation, perhaps best known for its signature head-shaving fundraising events, is a nonprofit organization dedicated to supporting childhood cancer research.

Knoepfler is exploring the hypothesis that a certain factor — the oncogene N-Myc – causes a deadly brain cancer as well as other childhood tumors by locking in the abnormal development of certain neural stem cells. His research is focusing on a possible aberrant mechanism that leads to medulloblastomas, the most common form of malignant brain tumors in children. The tumors are more prevalent in children under the age of 5, and are found more often in males than females.

“The problem in medulloblastomas is that for some reason the neural stem cells appear to have an accelerator, but no brake,” said Knoepfler, who also holds a position at the Institute for Pediatric Regenerative Medicine at Shriners Hospital for Children-Northern California. “Once a normal stem cell mutates into a cancer stem cell, it continues to rapidly grow and develop in its cancerous form. Our preliminary data point to N-Myc doing double duty as a ‘molecular villain,’ punching the accelerator and releasing the brakes in these cells, thus driving the formation of tumors. I’m hoping to come up with new treatments by identifying a way to harness these troubling cellular activities.”

According to National Cancer Institute, medulloblastomas, although rare, are a common brain tumor in children and responsible for up to 25 percent of all pediatric brain cancers. Nationally, about 500 cases are diagnosed annually. They usually occur in children before the age of 10. The tumors arise in the part of the brain (cerebellum) that controls balance and other complex motor functions.

Knoepfler, who is a cancer survivor himself, 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.

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. St. Baldrick’s funds are granted to some of the most brilliant childhood cancer research experts in the world and to younger professionals who will be the experts of tomorrow. Funds awarded also enable hundreds of local institutions to participate in national pediatric cancer clinical trials, a child’s best hope for a cure. Since the foundation’s first grants as an independent charity in 2005, St. Baldrick’s has funded more than $76 million in childhood cancer research. For more information please call 1.888.899.BALD or visit www.StBaldricks.org.