Investigators in The Washington University NF Center were awarded a 2014 Innovation Grant from Alex’s Lemonade Stand Foundation (ALSF). This grant to David H. Gutmann, MD, PhD, will support our efforts to develop personalized (precision) medicine strategies to optimize pediatric brain tumor management.
Brain tumors (low-grade gliomas) arising in children do not represent a single disease, but rather comprise a heterogeneous collection of distinct cancer subtypes. Our ability to more effectively manage children with brain tumors is limited by an incomplete understanding of the factors that contribute to this heterogeneity. In the common cancer predisposition syndrome, neurofibromatosis type 1 (NF1), preliminary studies have suggested that the specific NF1 genetic mutation may be an underappreciated contributor to low-grade glioma formation, progression, and response to therapy.
Grant funds received by the Washington University NF Center will be used to define the cause-and-effect relationship between the specific germline NF1 genetic mutation and low-grade glioma behavior by generating NF1-patient stem cell lines and mouse strains harboring different germline NF1 gene mutations. These proposed studies will establish a new conceptual framework for developing and testing therapies tailored to specific children as an initial step towards personalized treatments for pediatric low-grade glioma.
Alex’s Lemonade Stand Foundation (ALSF) emerged from the front yard lemonade stand of cancer patient Alexandra “Alex” Scott (1996-2004). In 2000, 4-year-old Alex announced that she wanted to hold a lemonade stand to raise money to help find a cure for all children with cancer. Since Alex held that first stand, the Foundation bearing her name has evolved into a national fundraising movement, complete with thousands of supporters across the country carrying on her legacy of hope. To date, Alex’s Lemonade Stand Foundation, a registered 501(c)3 charity, has raised more than $80 million toward fulfilling Alex’s dream of finding a cure, funding over 450 pediatric cancer research projects nationally. For more information on Alex’s Lemonade Stand Foundation, visit AlexsLemonade.org.