A new therapy for type 1 diabetes? A promising treatment for deadly viruses? Does a common treatment for children with severe infection actually cause more harm than good? These are important questions being investigated by pediatric researchers at Mattel Children's Hospital UCLA.
They are also the compelling projects that earned top accolades at the sixth annual Today's and Tomorrow's Children Fund (TTCF) Faculty Presentation Awards on May 16 which honored the researchers and awarded a total of $348,300 to support their studies.
Dr. Kuk-Wha Lee, (Santa Monica), assistant professor of pediatric endocrinology, won the grand prize of $150,000 for her research in finding a new treatment for type 1 diabetes. Each day, approximately 40 children nationwide are diagnosed with this disease that causes the body's immune system to attack and destroy the insulin-producing cells of the pancreas. It leads to a dependence on injected or pumped insulin for life and carries the constant threat of devastating complications. Lee and fellow investigators have discovered a unique protein called Humanin (HN) that protects nerve cells, blood-derived cells and muscle cells against stresses that harm the body. They propose that HN will protect insulin-producing cells from dying in the process that leads to type 1 diabetes. The award enables Lee and her colleagues to move their research project forward which has the potential for HN to be rapidly translated into a clinical trial in newly diagnosed children with this disease.
Dr. Paul Krogstad, (Culver City), professor of pediatric infectious diseases, earned an award of $110,300 for his work in the discovery and development of drugs to treat enterovirus infections in children. The enteroviruses are a group of more than 100 readily transmitted, seasonal viruses that usually produce common cold symptoms, hand-foot-and-mouth diseases, and other mild conditions in adolescents and adults. For newborns, infants, and young children, however, enteroviruses can be deadly. In this vulnerable population, they frequently produce central nervous system infections, hepatitis, and other life-threatening illnesses. There are no medications to treat enterovirus infections. Krogstand and his colleagues are studying the chemistry of enteroviruses, searching for potential therapeutic targets. Specifically, they have identified a little-used experimental antibiotic that dramatically reduces the growth of enteroviruses in cells and aim to confirm their initial studies and identify other compounds as well. Support from the TTCF will provide the critical initial push to garner the larger-scale funding needed to identify candidate medications to be tested in clinical trials.
Dr. Yonca Bulut, (Beverly Hills), associate clinical professor of pediatric critical care, was awarded $88,000 for her research to investigate whether iron supplementation worsens infections in children. Sepsis, a severe form of childhood infection, is a major cause of hospitalization and affects more than 40,000 children annually with a 10 percent mortality rate, especially among infants. When a child is hospitalized with a severe infection, iron supplementation is common practice to prevent anemia. However, iron is also an essential nutrient for bacteria, which in turn could be more harmful than helpful. Bulut and her colleagues' ultimate goal is to examine the safety of iron supplementation in healthy and ill children. Support from TTCF will allow them to obtain critical pre-clinical data to lay the framework for human studies.
The Today's and Tomorrow's Children Fund was born out of the belief that, by pooling their donations, a group of committed individuals can award a major gift every year to make an important difference in the work of talented UCLA pediatrics faculty members whose cutting edge research benefits the boys and girls at Mattel Children's Hospital UCLA. Organized in 2006, the group continues to grow each year and currently includes 64 volunteer members from the community. UCLA faculty members are selected to present their research projects to the group members, who then select the winning presentations. Ultimately, the group hopes to expand its membership and to provide an annual award of $1 million to one or more pediatric researchers.
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