Translational Research: A Rochester Tradition
George Hoyt Whipple, the founding dean of the University of Rochester School of Medicine and Dentistry, set the stage for translational research at Rochester. Whipple devoted much of his research energy to anemia and the physiology of the liver. His work, along with others, led to the use of raw liver as a treatment for pernicious anemia. A classic example of basic research transformed into a patient-focused treatment, it also resulted in a shared Nobel Prize in 1934 for Whipple.
Since then, research from Rochester has created new treatments that have changed the world in many ways. Here are a few examples.
Lung Surfactant
Medical Center researchers were the first to administer lung surfactant to premature infants, dramatically improving the survival rates of babies born more than 12 weeks premature from 50 to 90 percent. In 1981, Robert Notter, M.D., Ph.D., and other Rochester scientists studied a preparation called Calf Lung Surfactant Extract as a potential clinical surfactant. Medical Center physicians started treating premature infants and other newborns with lung development problems in 1983 with surfactant made in Notter's lab. The researchers published clinical papers documenting the efficacy of the surfactant extract in premature infants in 1985. The extract was used to treat patients into the 1990s. Today, various surfactants save lives around the world.
Haemophilus Influenza b (Hib) Vaccine
URMC scientists developed the Haemophilus influenza b (Hib) vaccine, virtually eradicating a leading cause of meningitis in preschoolers. Three Rochester pediatric researchers – the late David Smith, M.D. (M'58), Porter Anderson, Ph.D., and Richard Insel, M.D. – were among the first to develop and test a "conjugate" vaccine, a method to make a vaccine more effective by linking it to a protein that incites a more powerful immune system response to fight an infection.
The team first used the technology to tackle bacteria known as Haemophilus influenza type b (Hib). Smith eventually founded a company called Praxis to develop a vaccine. The Hib vaccine was approved by the federal Food and Drug Administration in 1990, then the first vaccine in 20 years to be recommended by the FDA for universal use in children. It has virtually wiped out infection by Hib bacteria, which was a leading cause of meningitis in children under the age of 5. Another vaccine based on the technology, Prevnar, was introduced in 2000. It prevents invasive infections by pneumococcal bacteria, which cause meningitis, ear infections, pneumonia, and other diseases. In the developing world, about one million people die each year of infections caused by the bacteria. Today, the David H. Smith Center for Vaccine Biology and Immunology builds on the heritage of such strong basic science research to help design the next generation of vaccines.
Cervical Cancer Vaccine
In June 2006, the FDA approved a vaccine with a Rochester lineage that can prevent a high percentage of cervical cancer. The vaccine utilizes 20 years of research by a trio of University virologists – Richard Reichman, M.D., William Bonnez, M.D., and Robert Rose, Ph.D. The vaccine targets a group of viruses known as human papillomaviruses (HPV), which cause 12,000 cases of cervical cancer in women in the United States annually.
Virus-like particles, or VLPs, are key to the vaccine. The Rochester virologists began studying how a person's immune system fights HPV infection, eventually focusing on the actual viral particle that causes the disease. After finding that the body produces antibodies that could neutralize the virus, they learned how to make harmless virus-like particles to trigger the same immune response. They accomplished this by putting an HPV gene into insect cells using a virus called baculovirus, which infects insects; the HPV gene then produces particles that mimic the shape of real HPV particles. In early 1997, the trio began one of the world's first tests in humans of a vaccine to prevent HPV infection that was based on the patented Rochester technology. The study found the vaccine was safe and triggered an immune response. The University has licensing agreements with major drug makers for the vaccine.
LASIK Surgery
Customized LASIK surgery grew out of laboratory work in the early 1990s by a team led by David Williams, Ph.D., director of the University's Center for Visual Science. The team discovered how to use a laser beam to take extraordinary images of the inner human eye, enabling the mapping of dozens of defects or aberrations of the eye that were previously unknown. Scott MacRae, M.D., surgeon and professor of ophthalmology, applied the technology in his pioneering development of customized LASIK surgery, which dramatically enhances vision.
Disparities and Vaccinations
In an important "bench to community" study – the branch of translational research that evaluates the benefits and effectiveness of treatments – Peter Szilagyi, M.D., M.P.H., professor of pediatrics, found that a coordinated city-wide program that involves careful tracking of children's immunizations, and use of outreach workers to call on those families whose children fall behind on their shots and help overcome barriers to care, can dramatically increase the number of children who are vaccinated – as well as increase the likelihood of those children returning for regular, preventive checkups. The intervention boosted immunization rates among children in the city of Rochester to a level nearly identical to suburban children. The program is considered a national model, and has been adopted by other cities and urban areas.
Gabapentin and Hot Flashes
Rochester researchers have investigated new therapies for hot flashes for several years. Thomas J. Guttuso Jr., M.D., a former neurologist at Strong Memorial Hospital, first observed the unlikely connection between the seizure/migraine medication and hot flashes back in 1999. A female patient who was prescribed gabapentin for headaches told Guttuso that it did a better job at taming her hot flashes. This information led Guttuso to investigate further. His study was the first, randomized, placebo-controlled clinical trial to confirm the observation that gabapentin relieves hot flashes.
This year, in the July issue of Obstetrics and Gynecology journal, Rochester researchers reported that gabapentin is as effective as estrogen, which used to be the gold standard treatment for menopause symptoms. Sireesha Y. Reddy, M.D., assistant professor of obstetrics and gynecology at the University, was the lead author of the study, the first to compare gabapentin and estrogen. The University holds a patent on the use of gabapentin for hot flashes.


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