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Oxford-Harrington Rare Disease Scholar Award
Development of a Novel Biologic for Rheumatoid Arthritis
2016 Harrington Scholar-Innovator
Nunzio Bottini, MD, PhD, Associate Professor of Cellular Biology at the La Jolla Institute for Allergy and Immunology and consulting rheumatologist at the University of California, San Diego (UCSD), was born curious. It's part of the makeup of a physician-scientist, he says.
“Physician-scientists view disease as a problem that should be solved,” Dr. Bottini explains. “Curiosity tells you there is a better way to treat these problems. As a scientist you can contribute to that.” In Dr. Bottini's case, curiosity has defined his career.
After completing his MD and PhD at the University of Rome in his native Italy, Dr. Bottini received postdoctoral research training in biochemistry and signal transduction at the Sanford-Burnham Medical Research Institute in La Jolla, California. That experience ignited his interest in immunology, and he spent his first years in the United States in the laboratory. Relocating to La Jolla Institute in 2009, Dr. Bottini's interest in immunology led him to rheumatology and autoimmune diseases, and he moved into the clinical sphere as well. With a keen understanding of signaling mechanisms in immune cells, he enjoys putting that training into practice with his patients.
“Rheumatology is very brainy in terms of how you proceed,” he notes. “On the clinical side, there is a lot of thinking and science involved in a diagnosis. In terms of science in the lab, there is a lot you can do.” In rheumatoid arthritis, immune cells move into the joint and release antibodies that irritate local joint lining cells, which amplifies pain and joint damage.
For about one-third of patients, the drugs used to suppress the immune system are not effective, Dr. Bottini says. “We want to develop a first-in-class drug for rheumatoid arthritis that complements current immune-targeted medications and does not increase the risk of infection,” he states. He already has defined a protein agent that blocks the destructive process of local cells in the joints.
With assistance from Harrington Discovery Institute experts, Dr. Bottini anticipates moving the protein through preclinical development to ensure its safety and determine how long it persists in the bloodstream. The critical stage for his project was a laboratory experiment that proved the protein's effectiveness. Dr. Bottini and the other scientists in his lab were anxious as they waited for the results that week, he remembers.
“Most of the time, we are wrong. We work for that two percent of the time that we get exciting data. That is the payoff for being curious.”