UC Davis stem cell scientist Paul Knoepfler has been awarded $250,000 for glioma research from an organization begun 15 years ago by a 5-year-old girl with a deadly form of childhood cancer.
The two-year grant from Alex’s Lemonade Stand Foundation (ALSF) will allow Knoepfler to study a family of pediatric gliomas with certain genetic mutations that that are especially lethal. The goal of the research is to catalyze development of innovative treatments by pinning down how changes in gene expression promote childhood tumor development.
“We are honored to have received this ALSF funding and excited at this opportunity to make a difference in this important area of childhood cancer,” said Knoepfler, associate professor of cell biology and human anatomy, and scientific member of the UC Davis Comprehensive Cancer Center. “Our team includes both cancer center researchers and physicians working together toward the goal of making a difference for the children battling these often fatal brain tumors.”
Knoepfler explained that pediatric gliomas are different than those that occur in adults, but their treatment is largely based on adult glioma therapies, which he says needs to change.
“There is an urgent need for new treatments for childhood gliomas that are designed specifically for these rather than adult gliomas,” he said.
Unlike adult tumors, childhood gliomas often have distinct mutations present in a gene-making protein called H3.3. As a result, the H3.3 mutations represent a possible new therapeutic target for children with gliomas.
“The goal of this ALSF grant is to determine how the H3.3 mutations cause cancer with the more specific objective of catalyzing new targeted treatments,” Knoepfler said. “The research will involve cutting-edge gene editing and next-generation sequencing technologies.”
The ALSF was established by Alexandra Scott of Manchester, Conn. Diagnosed at age 2 with neuroblastoma, she was later determined to help other children with cancer and started a lemonade stand to raise money. Alex passed away at age 8, but her effort continued to grow and has since raised more than $100 million for pediatric cancer research through the Foundation bearing her name, funding more than 500 research projects nationally. For more information on Alex’s Lemonade Stand Foundation, visit AlexsLemonade.org.