Newswise — PHILADELPHIA - The American College of Physicians, the largest medical specialty organization in the United States, presented Daniel J. Rader, MD, with its Award for Outstanding Work in Science as Related to Medicine at the organization’s annual scientific conference in San Diego. The award recognizes “outstanding work in science (non-clinical or clinical, biochemical, biological, physical, or social) as related to medicine.”

Rader is the Seymour Gray Professor of Molecular Medicine and chair of the department of Genetics in the Perelman School of Medicine at the University of Pennsylvania.

He is an internationally recognized authority in the genetics and physiology of lipoprotein metabolism and atherosclerosis (hardening of the arteries). Lipoproteins transport fats such as cholesterol and triglycerides throughout the body for energy and storage, then finally to the liver for elimination. Rader uses human genetics to identify new genes and pathways involved in regulating lipoprotein metabolism and influencing atherosclerosis and then uses model systems to understand the mechanisms by which they act. This approach of ‘functional genomics’ provides new insights into human biology and also identifies new potential targets for therapeutic intervention.

Rader and colleagues helped identify the molecular defect in a rare genetic disorder that causes extremely low levels of low-density lipoproteins, or LDL ("bad cholesterol"causing  plaque that can clog arteries, potentially leading to heart attack, stroke, and peripheral artery disease). This discovery resulted in the development of inhibitors of this pathway which reduced levels of LDL.  Rader then spent a decade converting one such potential inhibitor, which had been abandoned by its pharmaceutical developer, into a treatment for the orphan disease homozygous familial hypercholesterolemia (HoFH), which is characterized by extremely high levels of LDL and heart disease in children. This led to FDA and European approval of lomitapide, the first effective medication for the treatment of HoFH.

Rader also has a special interest in the metabolism of high-density lipoprotein (HDL—"good cholesterol") and its relationship to atherosclerosis. High HDL cholesterol levels are widely associated with a lower risk of heart disease. But Rader has refined this knowledge to show that the ability of HDL to extract cholesterol from cells is a better predictor of coronary disease than simple HDL cholesterol blood levels. While high HDL levels may indicate an efficient purging of excess fats, in some cases they may reflect the liver's inability to push out cholesterol for excretion from the body, leading to a build-up in the blood. Rader and his team have helped to identify and understand specific genes that affect HDL function and its relationship to coronary disease.

Rader has authored over 450 scientific articles and received many research awards including the Burroughs Wellcome Fund Clinical Scientist Award, Bristol Myers Squibb Cardiovascular Research Award, Doris Duke Charitable Foundation Distinguished Clinical Investigator Award, AHA Jeffrey M. Hoeg Award for Basic Science & Clinical Research, AHA Clinical Research Prize, and CRF Distinguished Clinical Research Award. He is an elected member of the American Society for Clinical Investigation, Association of American Physicians, and National Academy of Medicine.

At the Perelman School of Medicine, he has received the William Osler Patient Oriented Research Award, Donald B. Martin Outstanding Teacher Award, and the Outstanding Faculty Award from the Department of Medicine. He has been named to Philadelphia magazine’s “Top Docs” honor roll every year since 2002.

Rader earned his medical degree at the Medical College of Pennsylvania, followed by an internship and residency at Yale-New Haven Hospital and a post-doctoral fellowship at the National Institutes of Health.