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Philadelphia, PA (December 19, 2013) – In response to cut-backs in National Institutes of Health (NIH) funding of new R01 and P01 applications, Alex’s Lemonade Stand Foundation has announced the awarding of two new Springboard Grants to researchers at leading institutions across the country. The grants, designed to jump-start new projects with high impact potential for childhood cancer research while other funding is sought, will provide $100,000 over the course of one year as researchers work to reapply for NIH funding.
The grants will extend to Stella T. Chou, MD at The Children’s Hospital of Philadelphia (CHOP), who will focus on how an extra chromosome 21 and the mutant GATA1 affects blood development creating a predisposition to leukemia in children with Down Syndrome; and Peter Murray, PhD at St. Jude Children’s Research Hospital in Memphis, TN who will examine special cells in the immune system, macrophages, and how they function in tumors. (Full lay summaries of Dr. Chou’s and Dr. Murray’s projects can be found on the following page.)
Alex’s Lemonade Stand Foundation first introduced the Springboard Grant in 2012 to advance new projects with high impact potential who had applied to the NIH for an R01 or P01 award within the last six months. To be considered for funding, the applicants must have scored within the top 20%, but outside of the agency’s fundable range. The goal of the grants is to sustain the research while scientists reevaluate and subsequently reapply for large-scale funding.
“With less than 5% of the Federal Government’s total funding for cancer research each year being dedicated to childhood cancers, Alex’s Lemonade Stand Foundation is dedicated to keeping promising research alive,” said Jay Scott, Co-Executive Director of the Foundation. “Through the Springboard Grant, we work to sustain the research of these promising investigators while they reapply for large-scale funding, ultimately resulting in better treatments and cures for all childhood cancers.”
In addition to Springboard Grants, Alex’s Lemonade Stand Foundation introduced Bridge Grants in 2012 as a new funding opportunity for researchers whose projects are in jeopardy due to the decreased funding available from the NIH. ALSF’s Bridge Grants are intended to keep scientist’s projects on track while they reapply for NIH funding to ensure novel research projects are not compromised. ALSF’s Bridge Grant awards also provide $100,000 for 12 months.
2013 Funded Springboard Grant Lay Summaries
Stella T. Chou, MD, The Children's Hospital of Philadelphia, Philadelphia, PA
Modeling Genetic Modifiers of Down Syndrome Leukemogenesis
Children with Down Syndrome (Trisomy 21) exhibit numerous blood abnormalities and a predisposition to leukemia. Neonates with Down Syndrome are often born with a pre-leukemia termed transient myeloproliferative disorder (TMD). Many subsequently develop acute megakaryoblastic leukemia (AMKL) by age 4 which is associated with significant treatment-related toxicity. Both TMD and AMKL are accompanied by mutations in a gene named GATA1 that is required for normal blood development.
My research proposal focuses on how an extra chromosome 21 and the mutant GATA1 affects blood development. We are using induced pluripotent stem cells, a renewable human cell source that we generated from blood cells of patients with Down Syndrome and TMD, to define step-by-step the pathways of aberrant blood formation and progression to leukemia associated with trisomy 21.
Peter Murray, PhD, St. Jude Children's Research Hospital, Memphis, TN
Inflammatory Signaling in the Tumor Microenvironment
We associate "inflammation" with diseases such as arthritis, inflammatory bowel diseases, bronchitis or rashes. Most of us would not consider cancer an inflammatory disease, yet pathologists first made the link between cancers and inflammatory cells a century ago. Today, there is tremendous interest in cancer and inflammation because of the possibility that inflammation is helping cancer cells survive and proliferate.
We study a special cell of the immune system called macrophages. Macrophages get their name from the Greek: "big eater," and are important for many aspects of immunity: macrophages recognize and eat bacteria and viruses as well as dead and dying cells that need to be removed. Macrophages also signal to other cells of the immune system and help organize immune responses. However, macrophages can also become uncontrolled: over-active macrophages can cause excessive inflammation, and become unresponsive to other immune cells that give a "stop" signal to block macrophages' aggressive behavior. Macrophages also invade tumors, often in large numbers.
What are the macrophages doing in tumors: are they helping the cancer cells to grow? Or, do macrophages recognize that the cancer cells are out of control and need to be stopped? The goal of this project is to understand what is special about cancer macrophages. We will focus on cancers of childhood, and use model systems that closely mimic human cancer and translate our findings to human macrophages isolated from childhood cancers.
About Childhood Cancer
Childhood cancer is a general term used to describe cancer in children occurring regularly, randomly and sparing no ethnic group, socioeconomic class, or geographic region. Childhood cancer extends to over a dozen types of cancers and a countless amount of subtypes. Just a few of these cancer types include: Ewing’s sarcoma, glioma, leukemia, lymphoma, medulloblastoma, neuroblastoma, osteosarcoma, retinoblastoma, rhabdomyosarcoma and Wilm’s tumor. In the United States, childhood cancer is the leading cause of death by disease in children under the age of 15. Every day, approximately 250 kids around the world die from cancer, accounting for 91,250 losing their lives to the disease every year.
About Alex’s Lemonade Stand Foundation
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 $65 million toward fulfilling Alex’s dream of finding a cure, funding over 350 pediatric cancer research projects nationally. For more information on Alex’s Lemonade Stand Foundation, visit AlexsLemonade.org.