Bright Futures
05/17/2010
Find the best young researchers. Support them generously. And watch the breakthroughs happen for kids. That’s the formula behind the Faculty Scholars Program funded by the Children’s Discovery Institute

Could a child with scoliosis or muscular dystrophy be treated with genetically targeted therapy? Does congenital heart disease arise from genetic defects in heart-cell metabolism? Are there better ways to boost a child’s immune system? The Faculty Scholars want to know. 

The Children’s Discovery Institute funds six Faculty Scholars. Brilliant scientists at the beginning of their careers, the Scholars’ fresh thinking holds tremendous promise for children’s health. Thanks to donor-funded grants, these rising stars can set up laboratories at Washington University School of Medicine and forge ahead with innovative research.

“The start of a scientist’s career is a crucial time,” said S. Celeste Morley, MD, PhD, Instructor in the Division of Pediatric Infectious Diseases and one of the newest Faculty Scholars. “You’re making the transition from training, and missteps can happen that might derail new scientific ideas. Support from the Children’s Discovery Institute helps nurture and protect these new ideas.” 

Powerful pediatric research 

Children’s immune response is the focus of the two newest scholars, Dr. Morley and Megan Cooper, MD, PhD, Fellow in the Division of Pediatric Rheumatology.
 
Dr. Cooper has already published an article that showed, for the first time, “memory” in a type of white blood cell called the natural-killer cell, which is involved in the body’s innate immunity. “Memory in a cell,” said Dr. Cooper, “is what boosts your immune system after an exposure— it’s why a vaccine works.” Dr. Cooper will use her funding to study memory-like responses in natural-killer cells. Her research may suggest ways to strengthen the immune system in premature infants, children with HIV infection, or children with juvenile rheumatoid arthritis.
 
A scientist since the 7th grade, Dr. Morley attended a regional public science high school, conducting DNA research as a teenager. In medical school, she began her study of the T- lymphocyte, a white blood cell that moves through the body to fight bacteria, viruses, and cancer cells. As a Faculty Scholar, Dr. Morley will work to understand how the deficiency of a protein, L-plastin, hampers children’s T-cellmediated immunity. Ultimately, her work may improve the diagnosis and care of children with immune deficiencies or cancer, while identifying new targets for drug therapy to boost T-cell function.  

For Faculty Scholar Jennifer Duncan, MD, Assistant Professor of Pediatrics and specialist in critical care medicine, understanding the cellular basis of congenital heart disease is the driving force for her work. “I study mitochondria,” Dr. Duncan declared, “the powerhouse inside the cytoplasm of every cell. In heart cells, mitochondria have to work hard to keep the heart pumping.” Dr. Duncan analyzes the connection between heart failure in children and developmental events that could disrupt the metabolism of mitochondria. 

Robert Baloh, MD, PhD, Assistant Professor of Neurology, and Christina Gurnett, MD, PhD, Assistant Professor of Neurology, Pediatrics, and Orthopedic Surgery are Faculty Scholars who focus on musculoskeletal and neuromuscular diseases. 

Dr. Gurnett studies the genetic basis of conditions like clubfoot and scoliosis. Her funding has enabled her to develop a DNA data bank of musculoskeletal diseases. “What we’re after,” said Dr. Gurnett, “is genetic information that might point to gene therapies or to dietary or environmental changes that treat or prevent skeletal disorders.” 

Like Dr. Gurnett, Dr. Baloh has used his funding to generate a DNA data bank, this one for neuromuscular diseases (NMDs) like muscular dystrophy. He has also begun work on a project that uses a patient’s skin cells to generate muscle or nerve cells, which are used to evaluate potential therapies. “It’s a painstaking process,” Dr. Baloh said, “but we are definitely moving toward treatment for these currently untreatable diseases. In the meantime, the genetic information we discover is profoundly reassuring to families, who want to know why their child has an NMD.” 

Finding advanced treatment for malaria—a disease that kills hundreds of thousands of children annually—is the goal of Faculty Scholar Audrey Odom, MD, PhD. “Malaria caused by the parasite Plasmodium falciparum is not amenable to vaccines,” noted Dr. Odom. “Immunity is short-lived, and we could conceivably wait 50 to 100 years for a vaccine. So new drug treatment is vital, especially since the organism is often resistant to the drugs we have.” Dr. Odom is closing in on the biosynthetic pathways that sustain the parasite’s life, which could be interrupted by novel drugs. 

Funding that fuels careers— and the community 

“Being a Faculty Scholar has allowed me to get my lab off the ground,” said Dr. Baloh. He echoed a sentiment voiced by all the Scholars—gratitude to the Children’s Discovery Institute for helping launch a productive research career. “When you leave training, someone has to take a chance on you. It means a lot to get this support.”

Faculty Scholars use their funding efficiently, and often locally. They buy equipment ranging from centrifuges to filing systems. They hire research assistants in the St. Louis community. Scholars report that this funding also enables them to increase their collaboration with other scientists, locally, nationally, and in some cases, globally. Such collaboration only bolsters the Institute’s reputation as an influential center for pediatric research. 

Finally, the Faculty Scholars program safeguards the community’s most precious resource—the health of our children, now and in the future.