Research That Rocked
Many scientific discoveries and innovations that changed the course of medicine originated at Cincinnati Children’s. Here’s a sampling.
Information and documents for this article came from the Cincinnati Children’s Archives. Founded in 1980 and housed in the historic Mitchell-Nelson Library (R3), the Archives’ mission is to support, chronicle, and promote the ongoing mission and heritage of Cincinnati Children’s.
- Bubble-defoam oxygenator heart-lung machine—Leland Clark, PhD, director of the Division of Neurophysiology, invented the technology that allowed the Cardiology team at Cincinnati Children’s to perform the first open-heart surgery in 1952. The machine took unoxygenated blood from the heart, oxygenated it and returned it to the body.
- Oral polio vaccine—Albert Sabin, MD, developed a live-virus oral vaccine against polio that was famously distributed to thousands of families on “Sabin Sunday,” April 24, 1960. Since then, this devastating disease has nearly been eliminated worldwide. For his work, he received the Presidential Medal of Freedom and the Daniel Drake Award, which is the highest honor the UC College of Medicine gives.
- PKU screening—Helen Berry, MS, did pioneering research on phenylketonuria (PKU), a genetic inability to metabolize an amino acid in protein, resulting in severe mental retardation. Berry helped develop a test to detect PKU and was a proponent of early screening. She spent years developing a dietary supplement that made it possible for PKU patients to eat a less restrictive diet, the first major improvement in treatment of PKU in 30 years.
- Sickle cell screening—Marilyn Gaston, MD, began the sickle cell disease program at Cincinnati Children’s in 1972. Later she became assistant surgeon general and rear admiral in the U.S. Public Health Service. In 1986, she published a sickle cell study that led to nationwide newborn screening.
- Human surfactant—In 1988, Jeffrey Whitsett, MD, who co-directs the Perinatal Institute, announced that he and his research team had identified and cloned two proteins essential to the production of human surfactant, a substance that keeps lungs pliable so they can easily expand and contract as one breathes. Whitsett’s discovery revolutionized care for premature newborns around the world.
- Rotavirus vaccine—Richard Ward, PhD, and David Bernstein, MD, developed and conducted early clinical trials of a successful rotavirus vaccine. It was first licensed in Mexico in 2004, and it received FDA approval in 2008 for use in the United States. It is now used worldwide. The disease, which is usually not fatal in the U.S., previously caused 500,000 deaths per year in undeveloped countries.
- Gene therapy for sickle cell anemia—Punam Malik, MD, a physician-scientist in the Cancer and Blood Diseases Institute, has done promising research on a new treatment for sickle cell anemia that reverses symptoms of the disease. Preliminary data from a pilot Phase 1-2 clinical trial was presented at the American Society of Hematology’s annual meeting in December 2018.
- Stem cell and organoid medicine—Jim Wells, PhD, graduate student Stephen Trisno, and other scientists at Cincinnati Children’s Center for Stem Cell and Organoid Medicine (CuSTOM) have been working to bioengineer the entire human gastrointestinal system in a laboratory, using pluripotent stem cells (PSCs). Their efforts are leading to new personalized diagnostic methods and focused in part on developing regenerative tissue therapies to treat or cure GI disorders. Also collaborating on this study are the divisions of Developmental Biology; Oncology; Allergy and Immunology, and Endocrinology at Cincinnati Children’s, as well as the Gladstone Institutes in San Francisco.